Trial explorer
Every trial in the timelines, with the headline result exactly as published. Each row links to the trial page and to the milestone it belongs to. Results from different trials are not directly comparable.
Showing 1003 of 1003 trials.
| Treatment | Setting | Phase | Headline result (as published) | ||||
|---|---|---|---|---|---|---|---|
| cll-trialists-meta-analysis | Chronic lymphocytic leukaemia | 1955 | Chemotherapy | Metastatic | meta-analysis | — | OS: 44% vs 47% at 120 moMilestone: Chlorambucil: the oral alkylating-agent reference for CLL |
| myeloma-trialists-overview-1998 | Multiple myeloma | 1962 | Chemotherapy | Any setting | meta-analysis | 6,633 | ORR: 60% vs 53.2%Milestone: Melphalan, then melphalan plus prednisone, in myeloma |
| DFCI 05-001 | Childhood acute lymphoblastic leukemia (ALL) | 1967 | Chemotherapy | Any setting | 3 | 463 | Asparaginase-related toxicity: 28% vs 26%Milestone: Asparaginase: from 1967 leukemia reports to a core ALL drug |
| EBCTCG | Breast cancer | 1971 | Endocrine, Chemotherapy, Radiation | Adjuvant, Metastatic | meta-analysis | — | OS: 61.4% vs 50.5% at 120 moMilestone: Tamoxifen: from ICI 46474 to adjuvant standard |
| EBCTCG | Breast cancer | 1971 | Endocrine | Adjuvant, Metastatic | meta-analysis | 21,457 | Any first recurrence: HR 0.53Milestone: Tamoxifen: from ICI 46474 to adjuvant standard |
| HIP⚑ needs review | Breast cancer | 1971 | Detection & prevention | Screening | — | — | No results recordedMilestone: First screening RCTs: HIP (1971) and Swedish counties (1985) |
| ici46474-christie-1971 | Breast cancer | 1971 | Endocrine | Adjuvant, Metastatic | — | 46 | Clinical response rate: see trialMilestone: Tamoxifen: from ICI 46474 to adjuvant standard |
| NSABP B-14 | Breast cancer | 1971 | Endocrine | Adjuvant, Metastatic | — | 2,644 | OS: 93% vs 92% at 48 moMilestone: Tamoxifen: from ICI 46474 to adjuvant standard |
| swedish-county-screening-trial | Breast cancer | 1971 | Detection & prevention | Screening | — | 162,981 | No results recordedMilestone: First screening RCTs: HIP (1971) and Swedish counties (1985) |
| bonadonna-abvd-vs-mopp | Hodgkin lymphoma | 1975 | Chemotherapy | Locally advanced, Metastatic | — | 60 | CR rate: 75% vs 76%Milestone: ABVD versus MOPP: preliminary randomised results (Bonadonna) |
| dartmouth-vs-dacarbazine-1999 | Melanoma | 1975 | Chemotherapy | Metastatic | 3 | 240 | ORR: 18.5% vs 10.2%Milestone: Dacarbazine: the reference single agent in metastatic melanoma |
| PEPI | Endometrial cancer | 1975 | Understanding, Detection & prevention | Prevention | — | 596 | Simple (cystic) hyperplasia: 27.7% vs 0.8%Milestone: Two 1975 studies link estrogen use to endometrial cancer |
| samuels-1975-bleomycin-combinations | Testicular germ cell tumors | 1975 | Chemotherapy | Metastatic | — | 90 | ORR: see trialMilestone: Bleomycin combinations for testicular cancer (1975) |
| samuels-1975-continuous-bleomycin | Testicular germ cell tumors | 1975 | Chemotherapy | Metastatic | — | 23 | Complete response rate: see trialMilestone: Bleomycin combinations for testicular cancer (1975) |
| temozolomide-vs-dacarbazine-2000 | Melanoma | 1975 | Chemotherapy | Metastatic | 3 | 305 | OS: median 7.7 vs 6.4 monthsMilestone: Dacarbazine: the reference single agent in metastatic melanoma |
| milan-cmf-adjuvant | Breast cancer | 1976 | Chemotherapy | Adjuvant | — | 386 | Treatment failure: 5.3% vs 24% at 27 moMilestone: Adjuvant CMF chemotherapy after mastectomy (Milan, 1976) |
| NWTS-1 | Neuroblastoma and Wilms tumour | 1976 | Chemotherapy, Radiation, Surgery, Understanding | Any setting | — | 606 | No results recordedMilestone: NWTS-1: randomised trial of radiotherapy and chemotherapy |
| einhorn-1977-pvb-series | Testicular germ cell tumors | 1977 | Chemotherapy | Metastatic | — | 50 | Complete response rate: see trialMilestone: PVB: high remission rates in disseminated testis cancer (1977) |
| NWTS-5 | Neuroblastoma and Wilms tumour | 1978 | Understanding, Chemotherapy, Radiation, Surgery | Any setting | 3 | 2,021 | No results recordedMilestone: NWTS-1 histology: anaplastic and sarcomatous tumours fare worse |
| walker-1978-bcnu-radiotherapy-anaplastic-glioma | Glioblastoma | 1978 | Radiation, Chemotherapy | Adjuvant | — | 303 | No results recordedMilestone: Randomised trials of postoperative radiotherapy and nitrosoureas |
| goldman-1986-bmt-cml | Chronic myeloid leukemia | 1979 | Cell therapy & transplant | Any setting | observational | 70 | OS: see trialMilestone: Marrow transplant in CML: identical twins, then matched siblings |
| koreth-2009-allo-sct-meta | Acute myeloid leukaemia (including APL) | 1979 | Cell therapy & transplant | Any setting | meta-analysis | 6,007 | RFS: HR 0.80 (95% CI 0.74–0.86)Milestone: Allogeneic transplant in first remission of AML |
| zittoun-1995-eortc-gimema | Acute myeloid leukaemia (including APL) | 1979 | Cell therapy & transplant | Any setting | — | 623 | DFS: 55% vs 30% at 48 moMilestone: Allogeneic transplant in first remission of AML |
| aalders-stage-i-pelvic-radiotherapy | Endometrial cancer | 1980 | Radiation | Adjuvant | — | 540 | Vaginal and pelvic recurrence: 1.9% vs 6.9%Milestone: Adjuvant pelvic radiotherapy: less recurrence, no survival gain shown |
| ASTEC/EN.5 | Endometrial cancer | 1980 | Radiation | Adjuvant | — | 905 | OS: 84% vs 84% at 60 mo, HR 1.05 (95% CI 0.75–1.48)Milestone: Adjuvant pelvic radiotherapy: less recurrence, no survival gain shown |
| GOG-99 | Endometrial cancer | 1980 | Radiation | Adjuvant | 3 | 448 | Cumulative incidence of recurrence: 3% vs 12% at 24 moMilestone: Adjuvant pelvic radiotherapy: less recurrence, no survival gain shown |
| PORTEC-1 | Endometrial cancer | 1980 | Radiation | Adjuvant | — | 715 | Locoregional recurrence: 4% vs 14% at 60 moMilestone: Adjuvant pelvic radiotherapy: less recurrence, no survival gain shown |
| calgb-aml-anthracycline-1982 | Acute myeloid leukaemia (including APL) | 1981 | Chemotherapy | Any setting | — | — | Complete remission rate: 72% vs 59%Milestone: Cytarabine with an anthracycline: CALGB trials in AML, 1981-82 |
| IESS-I | Sarcoma and GIST | 1981 | Chemotherapy, Radiation, Surgery | Localised | — | 342 | RFS: 60% vs 24% at 60 moMilestone: Intergroup Ewing's Sarcoma Studies: multimodal therapy |
| IESS-II | Sarcoma and GIST | 1981 | Chemotherapy, Radiation, Surgery | Localised | — | 214 | DFS: 68% vs 48% at 60 moMilestone: Intergroup Ewing's Sarcoma Studies: multimodal therapy |
| Milan I | Breast cancer | 1981 | Surgery, Radiation | Localised | — | 701 | Local recurrence: 8.8% vs 2.3%Milestone: Breast conservation vs mastectomy: Milan I, NSABP B-04 and B-06 |
| NSABP B-04 | Breast cancer | 1981 | Surgery, Radiation | Localised | — | 1,665 | OS: HR 1.08 (95% CI 0.91–1.28)Milestone: Breast conservation vs mastectomy: Milan I, NSABP B-04 and B-06 |
| NSABP B-06 | Breast cancer | 1981 | Surgery, Radiation | Localised | — | — | IBTR: see trialMilestone: Breast conservation vs mastectomy: Milan I, NSABP B-04 and B-06 |
| HERO | Prostate cancer | 1982 | Endocrine | Metastatic | 3 | 930 | Sustained castration rate: 96.7% vs 88.8%Milestone: LHRH agonists and medical castration (Tolis, 1982) |
| limb-sparing-vs-amputation-rct | Sarcoma and GIST | 1982 | Surgery, Radiation, Chemotherapy | Localised | — | 43 | DFS: 71% vs 78% at 60 moMilestone: Randomised trial: limb-sparing surgery plus radiotherapy vs amputation |
| rosen-preop-chemo-series | Sarcoma and GIST | 1982 | Chemotherapy, Surgery | Neoadjuvant | — | 57 | Continuous disease-free rate: see trialMilestone: Preoperative chemotherapy tailors osteosarcoma treatment |
| siop-1977-1979 | Neuroblastoma and Wilms tumour | 1983 | Chemotherapy, Radiation, Surgery | Neoadjuvant | — | 164 | No results recordedMilestone: SIOP and UK trials of preoperative chemotherapy before nephrectomy |
| UKW3 | Neuroblastoma and Wilms tumour | 1983 | Chemotherapy, Radiation, Surgery | Neoadjuvant | — | 205 | Stage III at nephrectomy: 9.8% vs 29.8%Milestone: SIOP and UK trials of preoperative chemotherapy before nephrectomy |
| mvac-sternberg-1985-series | Bladder cancer | 1985 | Chemotherapy | Metastatic | — | 25 | Complete response rate: see trialMilestone: MVAC: combination chemotherapy for advanced urothelial cancer |
| mvac-vs-cisplatin-intergroup | Bladder cancer | 1985 | Chemotherapy | Metastatic | — | 269 | OS: median 12.5 vs 8.2 monthsMilestone: MVAC: combination chemotherapy for advanced urothelial cancer |
| link-osteosarcoma-adjuvant-1986 | Sarcoma and GIST | 1986 | Chemotherapy | Adjuvant | — | 36 | RFS: 66% vs 17% at 24 moMilestone: Randomised trials: adjuvant chemotherapy for osteosarcoma |
| GOG-33⚑ needs review | Endometrial cancer | 1987 | Understanding, Surgery | Localised | observational | 621 | Disease outside the uterus at surgery: see trialMilestone: GOG-33: surgical-pathologic spread in stage I disease |
| williams-1987-bep-vs-pvb | Testicular germ cell tumors | 1987 | Chemotherapy | Metastatic | — | 261 | Disease-free status after treatment: 83% vs 74%Milestone: BEP versus PVB: etoposide replaces vinblastine |
| williams-1987-stage-ii-adjuvant-vs-observation | Testicular germ cell tumors | 1987 | Chemotherapy, Surgery | Adjuvant | — | 195 | Relapse rate: 6% vs 49%Milestone: Adjuvant chemotherapy vs observation after RPLND, stage II |
| huang-atra-apl-1988 | Acute myeloid leukaemia (including APL) | 1988 | Targeted | Any setting | — | 24 | Complete remission rate: see trialMilestone: All-trans retinoic acid induces complete remission in APL |
| intergroup-melanoma-surgical-trial-2cm-vs-4cm⚑ needs review | Melanoma | 1988 | Surgery | Localised | — | 740 | Local recurrence at excision site: 0.8% vs 1.7%Milestone: Narrower excision margins: randomised trials from 1988 to 2016 |
| irs-i-rhabdomyosarcoma | Sarcoma and GIST | 1988 | Chemotherapy, Radiation, Surgery | Localised, Metastatic | — | 686 | OS: see trialMilestone: Intergroup Rhabdomyosarcoma Studies I to IV: successive protocols |
| irs-ii-rhabdomyosarcoma | Sarcoma and GIST | 1988 | Chemotherapy, Radiation, Surgery | Localised, Metastatic | — | 999 | OS: see trialMilestone: Intergroup Rhabdomyosarcoma Studies I to IV: successive protocols |
| irs-iii-rhabdomyosarcoma | Sarcoma and GIST | 1988 | Chemotherapy, Radiation, Surgery | Localised, Metastatic | — | 1,062 | PFS: see trialMilestone: Intergroup Rhabdomyosarcoma Studies I to IV: successive protocols |
| irs-iv-rhabdomyosarcoma | Sarcoma and GIST | 1988 | Chemotherapy, Radiation, Surgery | Localised, Metastatic | — | 883 | FFS: 77% vs 75% at 36 moMilestone: Intergroup Rhabdomyosarcoma Studies I to IV: successive protocols |
| nci-til-1988 | Melanoma | 1988 | Cell therapy & transplant, Immunotherapy | Metastatic | — | 20 | ORR: see trialMilestone: TIL plus IL-2 in metastatic melanoma: NCI preliminary report (1988) |
| nci-til-2011 | Melanoma | 1988 | Cell therapy & transplant, Immunotherapy | Metastatic | — | 93 | ORR: see trialMilestone: TIL plus IL-2 in metastatic melanoma: NCI preliminary report (1988) |
| swedish-melanoma-study-group-2cm-vs-5cm | Melanoma | 1988 | Surgery | Localised | — | 989 | OS: HR 0.96 (95% CI 0.75–1.24)Milestone: Narrower excision margins: randomised trials from 1988 to 2016 |
| thomas-2004-margins-1cm-vs-3cm | Melanoma | 1988 | Surgery | Localised | — | 900 | Melanoma locoregional recurrence: HR 1.26 (95% CI 1.00–1.59)Milestone: Narrower excision margins: randomised trials from 1988 to 2016 |
| veronesi-1988-margins-1cm-vs-3cm | Melanoma | 1988 | Surgery | Localised | — | — | Regional lymph node metastasis: 4.6% vs 6.5%Milestone: Narrower excision margins: randomised trials from 1988 to 2016 |
| NWTS-3 | Neuroblastoma and Wilms tumour | 1989 | Chemotherapy, Radiation | Any setting | — | 1,439 | No results recordedMilestone: NWTS-3 and NWTS-4: shorter and pulse-intensive chemotherapy |
| NWTS-4 | Neuroblastoma and Wilms tumour | 1989 | Chemotherapy, Radiation | Any setting | — | 1,687 | RFS: 91.3% vs 91.4% at 24 moMilestone: NWTS-3 and NWTS-4: shorter and pulse-intensive chemotherapy |
| sectg-bep-3-vs-4-cycles | Testicular germ cell tumors | 1989 | Chemotherapy | Metastatic | 3 | 184 | Disease-free status after treatment: 98% vs 97%Milestone: Three cycles of BEP for good-prognosis disease |
| kolb-1995-dli-ebmt⚑ needs review | Chronic myeloid leukemia | 1990 | Cell therapy & transplant | Relapsed / refractory | observational | 135 | CR: see trialMilestone: Donor leukocyte transfusions for relapse after transplant (1990) |
| moertel-levamisole-fu-adjuvant⚑ needs review | Colorectal cancer | 1990 | Chemotherapy | Adjuvant | — | 1,296 | No results recordedMilestone: Adjuvant fluorouracil plus levamisole for stage III colon cancer |
| french-cll-fludarabine-cap-chop | Chronic lymphocytic leukaemia | 1991 | Chemotherapy | Metastatic | — | 938 | ORR: 71.1% vs 71.5%Milestone: Fludarabine: 1991 US approval and a 2000 trial against chlorambucil |
| lamm-1991-bcg-vs-doxorubicin | Bladder cancer | 1991 | Immunotherapy | Localised, Adjuvant | — | 262 | DFS: 37% vs 17% at 60 moMilestone: BCG versus intravesical doxorubicin, then BCG maintenance |
| rai-2000-fludarabine-chlorambucil | Chronic lymphocytic leukaemia | 1991 | Chemotherapy | Metastatic | — | 509 | CR rate: 20% vs 4%Milestone: Fludarabine: 1991 US approval and a 2000 trial against chlorambucil |
| swog-bcg-maintenance-lamm-2000 | Bladder cancer | 1991 | Immunotherapy | Localised, Adjuvant | — | 550 | RFS: median 76.8 vs 35.7 monthsMilestone: BCG versus intravesical doxorubicin, then BCG maintenance |
| sylvester-2002-bcg-progression-meta-analysis | Bladder cancer | 1991 | Immunotherapy | Localised, Adjuvant | meta-analysis | 4,863 | Progression to stage T2 or higher: 9.8% vs 13.8%Milestone: BCG versus intravesical doxorubicin, then BCG maintenance |
| sylvester-2005-bcg-vs-chemotherapy-cis-meta-analysis | Bladder cancer | 1991 | Immunotherapy | Localised, Adjuvant | meta-analysis | 700 | Complete response in carcinoma in situ: 68.1% vs 51.5%Milestone: BCG versus intravesical doxorubicin, then BCG maintenance |
| va-laryngeal-cancer-study | Head and neck cancer | 1991 | Chemotherapy, Radiation, Surgery | Locally advanced | — | 332 | OS: 68% vs 68% at 24 moMilestone: Induction chemotherapy plus radiation preserved the larynx (VA) |
| CALGB 8251 | Hodgkin lymphoma | 1992 | Chemotherapy | Locally advanced, Metastatic | — | 361 | CR rate: 83% vs 67%Milestone: CALGB: ABVD and MOPP/ABVD versus MOPP in advanced disease |
| INT 0123 | Gastric and oesophageal cancer | 1992 | Chemotherapy, Radiation | Locally advanced | 3 | 236 | OS: median 13 vs 18.1 monthsMilestone: RTOG 85-01: chemoradiotherapy versus radiotherapy alone |
| MRC TE08 | Testicular germ cell tumors | 1992 | Surgery, Understanding | Localised | — | — | Relapse with intermediate or poor prognosis: 0.8% vs 0.6%Milestone: Surveillance after orchidectomy for stage I non-seminoma |
| mrc-interferon-vs-mpa-rcc | Kidney cancer (renal cell carcinoma) | 1992 | Immunotherapy | Metastatic | — | — | OS: median 8.5 vs 6 months, HR 0.72 (95% CI 0.55–0.94)Milestone: Cytokine therapy: high-dose interleukin-2 and interferon alfa |
| mrc-stage-i-nsgct-surveillance | Testicular germ cell tumors | 1992 | Surgery, Understanding | Localised | observational | 373 | RFR: see trialMilestone: Surveillance after orchidectomy for stage I non-seminoma |
| MSLT-I | Melanoma | 1992 | Surgery, Understanding | Localised | 3 | 2,001 | DFS: 78.3% vs 73.1% at 60 mo, HR 0.74 (95% CI 0.59–0.93)Milestone: Sentinel-node biopsy: Morton 1992 to MSLT-I (2006, 2014) |
| RTOG 85-01 | Gastric and oesophageal cancer | 1992 | Chemotherapy, Radiation | Locally advanced | 3 | 121 | OS: median 12.5 vs 8.9 monthsMilestone: RTOG 85-01: chemoradiotherapy versus radiotherapy alone |
| SCOPE1 | Gastric and oesophageal cancer | 1992 | Chemotherapy, Radiation | Locally advanced | 2/3 | 258 | Treatment failure-free at 24 weeks: 66.4% vs 76.9%Milestone: RTOG 85-01: chemoradiotherapy versus radiotherapy alone |
| bnli-beam-abmt-relapsed | Hodgkin lymphoma | 1993 | Chemotherapy, Cell therapy & transplant | Relapsed / refractory | — | 40 | No results recordedMilestone: High-dose BEAM with transplant for relapsed Hodgkin's disease |
| european-apl-91 | Acute myeloid leukaemia (including APL) | 1993 | Targeted, Chemotherapy | Any setting | — | 101 | Complete remission rate: 91% vs 81%Milestone: ATRA in randomized trials for newly diagnosed APL, 1993-1997 |
| funen-fobt-trial | Colorectal cancer | 1993 | Detection & prevention | Screening | — | — | No results recordedMilestone: Faecal occult blood screening trials and mortality |
| german-cml-hu-vs-busulfan-1993 | Chronic myeloid leukemia | 1993 | Chemotherapy | Any setting | 3 | 441 | OS: median 58.2 vs 45.4 monthsMilestone: Hydroxyurea versus busulfan: the German CML Study Group trial |
| ghsg-ebmt-beam-hsct-relapsed | Hodgkin lymphoma | 1993 | Chemotherapy, Cell therapy & transplant | Relapsed / refractory | — | 161 | FFTF: 55% vs 34% at 36 moMilestone: High-dose BEAM with transplant for relapsed Hodgkin's disease |
| minnesota-colon-cancer-control-study | Colorectal cancer | 1993 | Detection & prevention | Screening | — | 46,551 | No results recordedMilestone: Faecal occult blood screening trials and mortality |
| north-american-intergroup-apl | Acute myeloid leukaemia (including APL) | 1993 | Targeted, Chemotherapy | Any setting | — | 346 | Complete remission rate: 72% vs 69%Milestone: ATRA in randomized trials for newly diagnosed APL, 1993-1997 |
| nottingham-fobt-trial | Colorectal cancer | 1993 | Detection & prevention | Screening | — | 152,850 | No results recordedMilestone: Faecal occult blood screening trials and mortality |
| NPS | Colorectal cancer | 1993 | Detection & prevention | Prevention, Screening | observational | 1,418 | No results recordedMilestone: Polypectomy and colorectal cancer prevention (National Polyp Study) |
| swog-ecog-chop-vs-third-generation-1993 | Non-Hodgkin lymphoma (DLBCL and follicular) | 1993 | Chemotherapy | Locally advanced, Metastatic | 3 | 899 | Alive without disease at 3 years: 46% vs 41% at 36 moMilestone: CHOP vs three intensive regimens in advanced lymphoma (1993) |
| calgb-8525 | Acute myeloid leukaemia (including APL) | 1994 | Chemotherapy | Any setting | 3 | 1,088 | DFS: HR 0.67 (95% CI 0.53–0.86)Milestone: High-dose cytarabine consolidation: CALGB randomised trial, 1994 |
| french-cml-ifn-arac-1997 | Chronic myeloid leukemia | 1994 | Immunotherapy | Any setting | 3 | — | OS: 85.7% vs 79.1% at 36 moMilestone: Randomized trials of interferon alfa in CML, 1994 to 1997 |
| german-cml-ifn-vs-bu-hu-1994 | Chronic myeloid leukemia | 1994 | Immunotherapy | Any setting | 3 | 513 | OS: median 5.5 vs 3.8 yearsMilestone: Randomized trials of interferon alfa in CML, 1994 to 1997 |
| icsg-ifn-vs-chemo-1994 | Chronic myeloid leukemia | 1994 | Immunotherapy | Any setting | 3 | 322 | OS: median 72 vs 52 monthsMilestone: Randomized trials of interferon alfa in CML, 1994 to 1997 |
| ecog-1995-bleomycin-omission | Testicular germ cell tumors | 1995 | Chemotherapy | Metastatic | — | 178 | Disease-free status after treatment: 88% vs 94%Milestone: Is bleomycin needed? EP versus BEP in good-prognosis disease |
| eortc-1997-bleomycin-omission | Testicular germ cell tumors | 1995 | Chemotherapy | Metastatic | — | 419 | Complete response rate: 87% vs 95%Milestone: Is bleomycin needed? EP versus BEP in good-prognosis disease |
| GETUG T93BP | Testicular germ cell tumors | 1995 | Chemotherapy | Metastatic | — | 257 | EFS: 86% vs 91% at 48 moMilestone: Is bleomycin needed? EP versus BEP in good-prognosis disease |
| NSCLC Collaborative Group meta-analysis⚑ needs review | Lung cancer | 1995 | Chemotherapy | Any setting | meta-analysis | 9,387 | OS: HR 0.87Milestone: NSCLC chemotherapy meta-analysis of 52 trials (BMJ 1995) |
| PARMA | Non-Hodgkin lymphoma (DLBCL and follicular) | 1995 | Cell therapy & transplant, Chemotherapy | Relapsed / refractory | — | 109 | EFS: 46% vs 12% at 60 moMilestone: PARMA: autologous transplant vs salvage chemotherapy in relapse (1995) |
| AUO AH 01/94 | Testicular germ cell tumors | 1996 | Chemotherapy | Adjuvant | 3 | 382 | RFS: 99.46% vs 91.87% at 24 mo, HR 7.937 (95% CI 1.808–34.480)Milestone: Adjuvant BEP for high-risk stage I non-seminoma |
| ECOG E1684 | Melanoma | 1996 | Immunotherapy | Adjuvant | — | 287 | RFS: median 1.7 vs 1 yearsMilestone: Adjuvant interferon: E1684 (1996) to pegylated interferon (2011) |
| EORTC 18991 | Melanoma | 1996 | Immunotherapy | Adjuvant | 3 | 1,256 | RFS: 45.6% vs 38.9% at 48 mo, HR 0.82 (95% CI 0.71–0.96)Milestone: Adjuvant interferon: E1684 (1996) to pegylated interferon (2011) |
| gemcitabine-vs-fluorouracil-burris | Pancreatic cancer | 1996 | Chemotherapy | Locally advanced, Metastatic | — | 126 | Clinical benefit response: 23.8% vs 4.8%Milestone: Gemcitabine versus fluorouracil in advanced pancreatic cancer |
| GOG-111 | Ovarian cancer | 1996 | Chemotherapy | Locally advanced, Metastatic | 3 | 410 | PFS: median 18 vs 13 monthsMilestone: Paclitaxel with platinum in first line: GOG-111, OV-10, ICON3 |
| ICON3 | Ovarian cancer | 1996 | Chemotherapy | Locally advanced, Metastatic | — | 2,074 | OS: median 36.1 vs 35.4 months, HR 0.98 (95% CI 0.87–1.10)Milestone: Paclitaxel with platinum in first line: GOG-111, OV-10, ICON3 |
| IFM 90 | Multiple myeloma | 1996 | Cell therapy & transplant, Chemotherapy | Any setting | — | 200 | ORR: 81% vs 57%Milestone: Autologous transplantation trials: IFM 90, MRC VII, IFM 94 |
| IFM 94 | Multiple myeloma | 1996 | Cell therapy & transplant, Chemotherapy | Any setting | — | 399 | CR or VGPR rate: 50% vs 42%Milestone: Autologous transplantation trials: IFM 90, MRC VII, IFM 94 |
| MRC Myeloma VII | Multiple myeloma | 1996 | Cell therapy & transplant, Chemotherapy | Any setting | — | 407 | CR rate: 44% vs 8%Milestone: Autologous transplantation trials: IFM 90, MRC VII, IFM 94 |
| mrc-adjuvant-bep-high-risk-stage-i-nsgct | Testicular germ cell tumors | 1996 | Chemotherapy | Adjuvant | observational | 114 | No results recordedMilestone: Adjuvant BEP for high-risk stage I non-seminoma |
| OV-10 | Ovarian cancer | 1996 | Chemotherapy | Locally advanced, Metastatic | 3 | 680 | PFS: median 15.5 vs 11.5 monthsMilestone: Paclitaxel with platinum in first line: GOG-111, OV-10, ICON3 |
| SWENOTECA CS1 NSGCT | Testicular germ cell tumors | 1996 | Chemotherapy | Adjuvant | observational | 745 | Relapse rate: 3.2% vs 41.7%Milestone: Adjuvant BEP for high-risk stage I non-seminoma |
| dfci-95-096 | Prostate cancer | 1997 | Radiation, Endocrine | Locally advanced, Localised | 3 | 206 | All-cause mortality: HR 1.8 (95% CI 1.1–2.9)Milestone: Radiotherapy plus androgen suppression (EORTC 22863) |
| EORTC 22863 | Prostate cancer | 1997 | Radiation, Endocrine | Locally advanced, Localised | 3 | 415 | OS: 79% vs 62% at 60 moMilestone: Radiotherapy plus androgen suppression (EORTC 22863) |
| ide-c2b8-pivotal-relapsed-indolent | Non-Hodgkin lymphoma (DLBCL and follicular) | 1997 | Immunotherapy | Relapsed / refractory | — | 166 | ORR: see trialMilestone: Rituximab for relapsed indolent lymphoma: FDA approval and JCO report |
| igcccg-1997 | Testicular germ cell tumors | 1997 | Understanding, Chemotherapy | Metastatic | observational | — | OS: 79% vs 91% at 60 moMilestone: IGCCCG prognostic classification of metastatic germ-cell cancer |
| mrc-eortc-1997-bep-vs-ceb | Testicular germ cell tumors | 1997 | Chemotherapy | Metastatic | — | 598 | Complete response rate: 87.3% vs 94.4%Milestone: Carboplatin in place of cisplatin was inferior at trial doses |
| shen-ato-relapsed-apl-1997 | Acute myeloid leukaemia (including APL) | 1997 | Targeted | Relapsed / refractory | — | 15 | Complete remission rate: see trialMilestone: Arsenic trioxide induces remission in relapsed APL |
| swedish-rectal-cancer-trial⚑ needs review | Colorectal cancer | 1997 | Radiation, Surgery | Neoadjuvant | — | 1,168 | Local recurrence: 11% vs 27%Milestone: Swedish Rectal Cancer Trial: short-course preoperative radiotherapy |
| us-multicenter-ato-relapsed-apl | Acute myeloid leukaemia (including APL) | 1997 | Targeted | Relapsed / refractory | — | 40 | Complete remission rate: see trialMilestone: Arsenic trioxide induces remission in relapsed APL |
| augmented-post-induction-slow-responders | Childhood acute lymphoblastic leukemia (ALL) | 1998 | Chemotherapy | Any setting | — | 311 | EFS: 75% vs 55% at 60 moMilestone: Augmented post-induction therapy for slow early responders |
| CALGB 8461 | Acute myeloid leukaemia (including APL) | 1998 | Understanding | Any setting | observational | 1,213 | Complete remission rate: 88% vs 67%Milestone: Diagnostic cytogenetics defines AML prognostic groups |
| gleeson-2024-tip-long-term | Testicular germ cell tumors | 1998 | Chemotherapy | Relapsed / refractory | observational | 104 | PFS: see trialMilestone: Ifosfamide-based salvage: VeIP, then TIP |
| kondagunta-2005-tip | Testicular germ cell tumors | 1998 | Chemotherapy | Relapsed / refractory | — | 46 | Complete response rate: see trialMilestone: Ifosfamide-based salvage: VeIP, then TIP |
| loehrer-1998-veip | Testicular germ cell tumors | 1998 | Chemotherapy | Relapsed / refractory | — | 135 | Disease-free status after treatment: see trialMilestone: Ifosfamide-based salvage: VeIP, then TIP |
| slamon-2001-trastuzumab-mbc | Breast cancer | 1998 | Targeted | Metastatic | — | 469 | TTP: median 7.4 vs 4.6 monthsMilestone: Trastuzumab approved for HER2-overexpressing metastatic breast cancer |
| abc-meta-analysis-neoadjuvant⚑ needs review | Bladder cancer | 1999 | Chemotherapy, Surgery | Neoadjuvant | meta-analysis | 2,688 | OS: 50% vs 45% at 60 mo, HR 0.87 (95% CI 0.78–0.98)Milestone: Neoadjuvant cisplatin-based chemotherapy before cystectomy |
| BA06 30894⚑ needs review | Bladder cancer | 1999 | Chemotherapy, Surgery | Neoadjuvant | 3 | 976 | OS: median 44 vs 37.5 monthsMilestone: Neoadjuvant cisplatin-based chemotherapy before cystectomy |
| CCG-3891 | Neuroblastoma and Wilms tumour | 1999 | Cell therapy & transplant, Chemotherapy, Radiation, Targeted | Any setting | — | 379 | EFS: 34% vs 22% at 36 moMilestone: Transplant and isotretinoin in high-risk neuroblastoma (CCG-3891) |
| CONVERT | Lung cancer | 1999 | Radiation, Chemotherapy | Locally advanced | 3 | 547 | OS: median 25 vs 30 months, HR 1.18 (95% CI 0.95–1.45)Milestone: Limited-stage SCLC: twice-daily thoracic radiotherapy (Turrisi 1999) |
| EORTC 22993-08993 | Lung cancer | 1999 | Radiation | Maintenance | 3 | — | Time to symptomatic brain metastases: HR 0.27 (95% CI 0.16–0.44)Milestone: Prophylactic cranial irradiation in SCLC (1999 and 2007) |
| GOG 120 | Cervical cancer | 1999 | Chemotherapy, Radiation | Locally advanced | — | 526 | No results recordedMilestone: Concurrent cisplatin-based chemoradiation: the 1999 trials |
| GOG 123 | Cervical cancer | 1999 | Chemotherapy, Radiation | Locally advanced | — | — | No results recordedMilestone: Concurrent cisplatin-based chemoradiation: the 1999 trials |
| GOG 85 | Cervical cancer | 1999 | Chemotherapy, Radiation | Locally advanced | — | 388 | No results recordedMilestone: Concurrent cisplatin-based chemoradiation: the 1999 trials |
| GOG-81 | Endometrial cancer | 1999 | Endocrine, Targeted | Metastatic | — | 299 | ORR: 15% vs 25%Milestone: Hormonal therapy: progestins to letrozole combinations |
| grossman-neoadjuvant-mvac⚑ needs review | Bladder cancer | 1999 | Chemotherapy, Surgery | Neoadjuvant | — | 317 | OS: median 77 vs 46 monthsMilestone: Neoadjuvant cisplatin-based chemotherapy before cystectomy |
| IFM 99-06 | Multiple myeloma | 1999 | Targeted, Immunotherapy | Relapsed / refractory | — | 447 | OS: median 51.6 vs 33.2 months, HR 0.59 (95% CI 0.46–0.81)Milestone: Thalidomide shows activity in refractory myeloma |
| MRC TE10 | Testicular germ cell tumors | 1999 | Radiation | Adjuvant | — | 478 | RFR: 96% vs 96.6% at 36 moMilestone: Smaller radiotherapy fields and lower doses for stage I seminoma |
| MRC TE18 / EORTC 30942⚑ needs review | Testicular germ cell tumors | 1999 | Radiation | Adjuvant | — | 625 | Relapse rate: HR 1.11Milestone: Smaller radiotherapy fields and lower doses for stage I seminoma |
| NCIC cervix trial | Cervical cancer | 1999 | Chemotherapy, Radiation | Locally advanced | 3 | 259 | OS: 69% vs 66% at 36 moMilestone: Concurrent cisplatin-based chemoradiation: the 1999 trials |
| PALEO | Endometrial cancer | 1999 | Endocrine, Targeted | Metastatic | 2 | 77 | PFS: HR 0.57 (95% CI 0.32–0.99)Milestone: Hormonal therapy: progestins to letrozole combinations |
| pci-overview-1999 | Lung cancer | 1999 | Radiation | Maintenance | meta-analysis | 987 | OS: 20.7% vs 15.3% at 36 moMilestone: Prophylactic cranial irradiation in SCLC (1999 and 2007) |
| RTOG 90-01 | Cervical cancer | 1999 | Chemotherapy, Radiation | Locally advanced | — | 403 | OS: 73% vs 58% at 60 moMilestone: Concurrent cisplatin-based chemoradiation: the 1999 trials |
| singhal-thalidomide-1999 | Multiple myeloma | 1999 | Targeted, Immunotherapy | Relapsed / refractory | — | 84 | ORR: see trialMilestone: Thalidomide shows activity in refractory myeloma |
| slomovitz-everolimus-letrozole | Endometrial cancer | 1999 | Endocrine, Targeted | Metastatic | 2 | 38 | CBR: see trialMilestone: Hormonal therapy: progestins to letrozole combinations |
| thalidomide-dexamethasone-vs-placebo-dexamethasone | Multiple myeloma | 1999 | Targeted, Immunotherapy | Relapsed / refractory | — | 470 | ORR: 63% vs 46%Milestone: Thalidomide shows activity in refractory myeloma |
| turrisi-1999-twice-daily-rt⚑ needs review | Lung cancer | 1999 | Radiation, Chemotherapy | Locally advanced | — | 417 | OS: median 23 vs 19 monthsMilestone: Limited-stage SCLC: twice-daily thoracic radiotherapy (Turrisi 1999) |
| Bezwoda high-risk primary HDC study (reviewed on site) | Breast cancer | 2000 | Chemotherapy, Cell therapy & transplant | Metastatic, Adjuvant | — | — | Results not shown (integrity flag)Milestone: High-dose chemotherapy with stem-cell rescue: a reversal |
| Bezwoda metastatic HDC study (retracted) | Breast cancer | 2000 | Chemotherapy, Cell therapy & transplant | Metastatic, Adjuvant | — | 90 | Results not shown (integrity flag)Milestone: High-dose chemotherapy with stem-cell rescue: a reversal |
| degramont-lv5fu2-oxaliplatin | Colorectal cancer | 2000 | Chemotherapy | Metastatic | 3 | 420 | PFS: median 9 vs 6.2 monthsMilestone: Oxaliplatin added to infusional fluorouracil in advanced CRC |
| douillard-irinotecan-5fu-lv | Colorectal cancer | 2000 | Chemotherapy | Metastatic | — | 387 | ORR: 35% vs 22%Milestone: Irinotecan with fluorouracil as first-line therapy for metastatic CRC |
| gc-vs-mvac-urothelial | Bladder cancer | 2000 | Chemotherapy | Metastatic | 3 | 405 | OS: HR 1.04 (95% CI 0.82–1.32)Milestone: Gemcitabine-cisplatin compared with MVAC in advanced bladder cancer |
| giacchetti-oxaliplatin-chronomodulated | Colorectal cancer | 2000 | Chemotherapy | Metastatic | 3 | 200 | ORR: see trialMilestone: Oxaliplatin added to infusional fluorouracil in advanced CRC |
| irinotecan-study-group-ifl | Colorectal cancer | 2000 | Chemotherapy | Metastatic | — | 683 | OS: median 14.8 vs 12.6 monthsMilestone: Irinotecan with fluorouracil as first-line therapy for metastatic CRC |
| JCOG0607 | Gastric and oesophageal cancer | 2000 | Surgery | Localised | — | 470 | OS: see trialMilestone: Endoscopic resection for early gastric cancer: Gotoda 2000 to JCOG0607 |
| MACH-NC | Head and neck cancer | 2000 | Chemotherapy, Radiation | Locally advanced, Adjuvant | meta-analysis | — | OS: HR 0.90 (95% CI 0.85–0.94)Milestone: MACH-NC: chemotherapy added to locoregional treatment (2000) |
| Netherlands high-risk adjuvant HDC trial | Breast cancer | 2000 | Chemotherapy, Cell therapy & transplant | Metastatic, Adjuvant | 3 | 885 | RFS: 65% vs 59% at 60 mo, HR 0.83 (95% CI 0.66–1.03)Milestone: High-dose chemotherapy with stem-cell rescue: a reversal |
| Philadelphia BMT Group | Breast cancer | 2000 | Chemotherapy, Cell therapy & transplant | Metastatic, Adjuvant | 3 | 199 | OS: 32% vs 38% at 36 moMilestone: High-dose chemotherapy with stem-cell rescue: a reversal |
| ARTIST | Gastric and oesophageal cancer | 2001 | Chemotherapy, Radiation | Adjuvant | 3 | 458 | DFS: HR 0.6865 (95% CI 0.4735–0.9952)Milestone: INT-0116 adjuvant chemoradiotherapy, then ARTIST and CRITICS |
| CAM307 | Chronic lymphocytic leukaemia | 2001 | Immunotherapy | Relapsed / refractory, Metastatic | 3 | 297 | PFS: HR 0.58Milestone: Alemtuzumab: accelerated approval, then CAM307 against chlorambucil |
| campath-cll-fludarabine-failure | Chronic lymphocytic leukaemia | 2001 | Immunotherapy | Relapsed / refractory, Metastatic | 2 | 93 | ORR: see trialMilestone: Alemtuzumab: accelerated approval, then CAM307 against chlorambucil |
| CRITICS | Gastric and oesophageal cancer | 2001 | Chemotherapy, Radiation | Adjuvant | 3 | 788 | OS: median 37 vs 43 months, HR 1.01 (95% CI 0.84–1.22)Milestone: INT-0116 adjuvant chemoradiotherapy, then ARTIST and CRITICS |
| dutch-tme-trial | Colorectal cancer | 2001 | Radiation, Surgery | Neoadjuvant | — | 1,861 | OS: 82% vs 81.8% at 24 moMilestone: Dutch TME trial: preoperative radiotherapy plus standardised TME |
| ESPAC-1 | Pancreatic cancer | 2001 | Chemotherapy, Radiation | Adjuvant | — | 541 | OS: median 19.7 vs 14 months, HR 0.66 (95% CI 0.52–0.83)Milestone: ESPAC-1: adjuvant chemotherapy and chemoradiotherapy compared |
| imatinib-gist-phase2-400-vs-600 | Sarcoma and GIST | 2001 | Targeted | Metastatic | 2 | 147 | ORR: see trialMilestone: Imatinib in GIST: case report, phase 2 and FDA approval |
| imatinib-late-chronic-phase-phase2⚑ needs review | Chronic myeloid leukemia | 2001 | Targeted | Any setting, Relapsed / refractory | 2 | 532 | MCyR: see trialMilestone: STI571/imatinib: first clinical results and FDA approval |
| INT-0116 | Gastric and oesophageal cancer | 2001 | Chemotherapy, Radiation | Adjuvant | 3 | 556 | OS: median 36 vs 27 months, HR 1.35 (95% CI 1.09–1.66)Milestone: INT-0116 adjuvant chemoradiotherapy, then ARTIST and CRITICS |
| nephrectomy-ifn-eortc-mickisch-2001 | Kidney cancer (renal cell carcinoma) | 2001 | Surgery, Immunotherapy | Metastatic | — | 85 | TTP: median 5 vs 3 months, HR 0.60 (95% CI 0.36–0.97)Milestone: Nephrectomy before interferon alfa: two randomised trials (2001) |
| nephrectomy-ifn-flanigan-2001 | Kidney cancer (renal cell carcinoma) | 2001 | Surgery, Immunotherapy | Metastatic | — | — | OS: median 11.1 vs 8.1 monthsMilestone: Nephrectomy before interferon alfa: two randomised trials (2001) |
| SEMPET | Testicular germ cell tumors | 2001 | Understanding | Metastatic | observational | 51 | Specificity: see trialMilestone: FDG-PET to predict viable tumour in residual seminoma masses |
| sti571-phase1-blast-crisis | Chronic myeloid leukemia | 2001 | Targeted | Any setting, Relapsed / refractory | — | 58 | Hematologic response: see trialMilestone: STI571/imatinib: first clinical results and FDA approval |
| sti571-phase1-chronic-phase | Chronic myeloid leukemia | 2001 | Targeted | Any setting, Relapsed / refractory | 1 | 83 | MCyR: see trialMilestone: STI571/imatinib: first clinical results and FDA approval |
| ATAC | Breast cancer | 2002 | Endocrine | Adjuvant | — | 9,366 | DFS: 89.4% vs 87.4% at 36 mo, HR 0.83 (95% CI 0.71–0.96)Milestone: Aromatase inhibitors versus tamoxifen (ATAC, BIG 1-98) |
| BIG 1-98 | Breast cancer | 2002 | Endocrine | Adjuvant | 3 | 8,010 | DFS: 84% vs 81.4% at 60 mo, HR 0.81 (95% CI 0.70–0.93)Milestone: Aromatase inhibitors versus tamoxifen (ATAC, BIG 1-98) |
| bristow-2002-cytoreduction-meta-analysis | Ovarian cancer | 2002 | Surgery | Locally advanced | meta-analysis | 6,885 | OS: median 33.9 vs 22.7 monthsMilestone: Cytoreduction and residual disease: maximal surgery linked to survival |
| EBCTCG | Breast cancer | 2002 | Endocrine | Adjuvant | meta-analysis | 31,920 | Breast cancer mortality: 12.1% vs 14.2%, HR 0.85 (95% CI 0.75–0.96)Milestone: Aromatase inhibitors versus tamoxifen (ATAC, BIG 1-98) |
| IRIS⚑ needs review | Chronic myeloid leukemia | 2002 | Targeted | Any setting | 3 | 1,106 | MCyR: 87.1% vs 34.7% at 18 moMilestone: IRIS: imatinib versus interferon plus cytarabine, first line |
| llovet-2002-tace-rct | Hepatocellular carcinoma | 2002 | Surgery, Chemotherapy | Locally advanced | — | 112 | OS: 75% vs 63% at 12 mo, HR 0.47 (95% CI 0.25–0.91)Milestone: Two randomised trials of chemoembolisation for unresectable HCC |
| LNH-98.5 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2002 | Immunotherapy, Chemotherapy | Locally advanced, Metastatic | — | 399 | CR rate: 76% vs 63%Milestone: R-CHOP vs CHOP in older DLBCL: GELA LNH98-5 (2002) |
| lo-2002-tace-rct | Hepatocellular carcinoma | 2002 | Surgery, Chemotherapy | Locally advanced | — | 80 | OS: 57% vs 32% at 12 moMilestone: Two randomised trials of chemoembolisation for unresectable HCC |
| PIVOT | Prostate cancer | 2002 | Surgery | Localised | 3 | 731 | All-cause mortality: 47% vs 49.9%, HR 0.88 (95% CI 0.71–1.08)Milestone: Prostatectomy vs watchful waiting or observation (SPCG-4, PIVOT) |
| SPCG-4 | Prostate cancer | 2002 | Surgery | Localised | — | 695 | Prostate cancer mortality: 4.6% vs 8.9%, HR 0.50 (95% CI 0.27–0.91)Milestone: Prostatectomy vs watchful waiting or observation (SPCG-4, PIVOT) |
| SWENOTECA CS1 seminoma | Testicular germ cell tumors | 2002 | Understanding, Surgery | Localised, Adjuvant | observational | 897 | Relapse rate: HR 1.9Milestone: Surveillance and risk-adapted management of stage I seminoma |
| warde-2002-pooled-surveillance⚑ needs review | Testicular germ cell tumors | 2002 | Understanding, Surgery | Localised, Adjuvant | observational | 638 | RFR: see trialMilestone: Surveillance and risk-adapted management of stage I seminoma |
| ACOSOG Z0011 | Breast cancer | 2003 | Surgery, Understanding | Localised | 3 | 891 | OS: 92.5% vs 91.8% at 60 mo, HR 0.79Milestone: Sentinel-node biopsy and less axillary surgery (B-32, Z0011) |
| AGO-OVAR-3 | Ovarian cancer | 2003 | Chemotherapy | Locally advanced | 3 | 798 | PFS: 37.5% vs 40% at 24 moMilestone: Carboplatin versus cisplatin with paclitaxel: GOG-158, AGO-OVAR-3 |
| APEX | Multiple myeloma | 2003 | Targeted | Relapsed / refractory | 3 | 669 | TTP: median 6.22 vs 3.49 months, HR 0.55Milestone: Bortezomib in relapsed myeloma: SUMMIT phase 2 to APEX |
| ardeshna-rituximab-vs-watch-wait-fl | Non-Hodgkin lymphoma (DLBCL and follicular) | 2003 | Chemotherapy, Immunotherapy | Any setting | 3 | — | Time to new treatment: 88% vs 46% at 36 mo, HR 0.21 (95% CI 0.14–0.31)Milestone: Watch and wait vs immediate chlorambucil, low-grade lymphoma |
| fung-2015-cvd-mortality⚑ needs review | Testicular germ cell tumors | 2003 | Supportive care, Chemotherapy, Radiation | Any setting | observational | 15,006 | Cardiovascular disease mortality: HR 4.86 (95% CI 1.25–32.08)Milestone: Cardiovascular disease as a late effect of testicular cancer treatment |
| GOG-158 | Ovarian cancer | 2003 | Chemotherapy | Locally advanced | 3 | 792 | PFS: median 20.7 vs 19.4 monthsMilestone: Carboplatin versus cisplatin with paclitaxel: GOG-158, AGO-OVAR-3 |
| haugnes-2010-cvd | Testicular germ cell tumors | 2003 | Supportive care, Chemotherapy, Radiation | Any setting | observational | 990 | Atherosclerotic disease after treatment: HR 2.30 (95% CI 1.04–5.30)Milestone: Cardiovascular disease as a late effect of testicular cancer treatment |
| HD9 | Hodgkin lymphoma | 2003 | Chemotherapy | Locally advanced, Metastatic | — | 1,201 | FFTF: 76% vs 69% at 60 moMilestone: BEACOPP (GHSG HD9) versus COPP-ABVD in advanced disease |
| INT-0091 | Sarcoma and GIST | 2003 | Chemotherapy | Localised, Metastatic | — | 518 | EFS: 69% vs 54% at 60 moMilestone: INT-0091: adding ifosfamide and etoposide in Ewing sarcoma |
| IRIS molecular substudy | Chronic myeloid leukemia | 2003 | Understanding | Any setting | 3 | 1,106 | MMR: 57% vs 24%Milestone: BCR-ABL1 transcript monitoring and the international scale |
| NSABP B-32 | Breast cancer | 2003 | Surgery, Understanding | Localised | 3 | 5,611 | OS: 90.3% vs 91.8% at 96 mo, HR 1.20 (95% CI 0.96–1.50)Milestone: Sentinel-node biopsy and less axillary surgery (B-32, Z0011) |
| qidong-afp-screening-trial | Hepatocellular carcinoma | 2003 | Detection & prevention | Screening | — | 5,581 | Stage I share of liver cancers at diagnosis: 29.6% vs 6%Milestone: Two Chinese randomised surveillance trials, two answers |
| RTOG 91-11 | Head and neck cancer | 2003 | Chemotherapy, Radiation | Locally advanced | 3 | 547 | Larynx preservation: 88% vs 75% at 24 moMilestone: RTOG 91-11: concurrent cisplatin for larynx preservation |
| shanghai-afp-ultrasound-screening-trial | Hepatocellular carcinoma | 2003 | Detection & prevention | Screening | — | 18,816 | Resection among HCC cases: 46.5% vs 7.5%Milestone: Two Chinese randomised surveillance trials, two answers |
| SUMMIT | Multiple myeloma | 2003 | Targeted | Relapsed / refractory | 2 | 202 | ORR: see trialMilestone: Bortezomib in relapsed myeloma: SUMMIT phase 2 to APEX |
| uk-watch-wait-vs-chlorambucil-lowgrade | Non-Hodgkin lymphoma (DLBCL and follicular) | 2003 | Chemotherapy, Immunotherapy | Any setting | — | 309 | OS: median 6.7 vs 5.9 yearsMilestone: Watch and wait vs immediate chlorambucil, low-grade lymphoma |
| van-den-belt-dusebout-2006-cvd⚑ needs review | Testicular germ cell tumors | 2003 | Supportive care, Chemotherapy, Radiation | Any setting | observational | 2,512 | Myocardial infarction after treatment: HR 1.2 (95% CI 0.7–2.1)Milestone: Cardiovascular disease as a late effect of testicular cancer treatment |
| veronesi-sentinel-node-trial | Breast cancer | 2003 | Surgery, Understanding | Localised | — | 516 | No results recordedMilestone: Sentinel-node biopsy and less axillary surgery (B-32, Z0011) |
| amgen-20020408 | Colorectal cancer | 2004 | Targeted | Metastatic | 3 | 463 | PFS: HR 0.54 (95% CI 0.44–0.66)Milestone: Cetuximab and panitumumab: anti-EGFR antibodies in refractory CRC |
| ANITA | Lung cancer | 2004 | Chemotherapy | Adjuvant | — | 840 | OS: median 65.7 vs 43.7 months, HR 0.80 (95% CI 0.66–0.96)Milestone: Adjuvant cisplatin after resection: IALT, JBR.10 and LACE |
| BOND | Colorectal cancer | 2004 | Targeted | Metastatic | — | 329 | ORR: 22.9% vs 10.8%Milestone: Cetuximab and panitumumab: anti-EGFR antibodies in refractory CRC |
| CAO/ARO/AIO-94 | Colorectal cancer | 2004 | Radiation, Chemotherapy | Neoadjuvant, Locally advanced | 3 | 823 | OS: 76% vs 74% at 60 moMilestone: CAO/ARO/AIO-94: preoperative vs postoperative chemoradiotherapy |
| choi-2018-metachronous-hp | Gastric and oesophageal cancer | 2004 | Detection & prevention | Prevention | — | 470 | Metachronous gastric cancer: 7.2% vs 13.4%, HR 0.50 (95% CI 0.26–0.94)Milestone: H. pylori eradication to prevent gastric cancer: trials 2004-2024 |
| choi-2020-family-history-hp | Gastric and oesophageal cancer | 2004 | Detection & prevention | Prevention | — | 1,838 | Gastric cancer incidence: 1.2% vs 2.7%, HR 0.45 (95% CI 0.21–0.94)Milestone: H. pylori eradication to prevent gastric cancer: trials 2004-2024 |
| CO.17 | Colorectal cancer | 2004 | Targeted | Metastatic | 3 | 572 | OS: median 6.1 vs 4.6 months, HR 0.77 (95% CI 0.64–0.92)Milestone: Cetuximab and panitumumab: anti-EGFR antibodies in refractory CRC |
| CRYSTAL | Colorectal cancer | 2004 | Targeted | Metastatic | 3 | — | PFS: HR 0.85 (95% CI 0.72–0.99)Milestone: Cetuximab and panitumumab: anti-EGFR antibodies in refractory CRC |
| EORTC 22931 | Head and neck cancer | 2004 | Chemotherapy, Radiation | Adjuvant | 3 | 334 | PFS: 47% vs 36% at 60 mo, HR 0.75 (95% CI 0.56–0.99)Milestone: Adding cisplatin to postoperative radiotherapy for high-risk disease |
| eortc-62005-gist | Sarcoma and GIST | 2004 | Targeted | Metastatic | 3 | 946 | PFS: HR 0.82 (95% CI 0.69–0.98)Milestone: Imatinib 400 mg vs 800 mg daily in advanced GIST |
| GOG-107 | Endometrial cancer | 2004 | Chemotherapy | Metastatic | 3 | 299 | ORR: 42% vs 25%Milestone: Chemotherapy for advanced disease: GOG-107, -177 and -209 |
| GOG-169 | Cervical cancer | 2004 | Chemotherapy | Metastatic | 3 | 280 | OS: median 9.7 vs 8.8 monthsMilestone: Cisplatin-paclitaxel and other platinum doublets for advanced disease |
| GOG-177 | Endometrial cancer | 2004 | Chemotherapy | Metastatic | 3 | 273 | ORR: 57% vs 34%Milestone: Chemotherapy for advanced disease: GOG-107, -177 and -209 |
| GOG-179⚑ needs review | Cervical cancer | 2004 | Chemotherapy | Metastatic | 3 | 294 | OS: median 9.4 vs 6.5 monthsMilestone: Cisplatin-paclitaxel and other platinum doublets for advanced disease |
| GOG-204 | Cervical cancer | 2004 | Chemotherapy | Metastatic | 3 | 513 | OS: HR 1.15 (95% CI 0.79–1.67)Milestone: Cisplatin-paclitaxel and other platinum doublets for advanced disease |
| GOG-209 | Endometrial cancer | 2004 | Chemotherapy | Metastatic | 3 | 1,381 | OS: median 37 vs 41 months, HR 1.002Milestone: Chemotherapy for advanced disease: GOG-107, -177 and -209 |
| habr-gama-stage-0-series | Colorectal cancer | 2004 | Surgery, Radiation | Neoadjuvant, Locally advanced | observational | 265 | Clinical complete response: see trialMilestone: Watch-and-wait after chemoradiotherapy for complete clinical response |
| hurwitz-ifl-bevacizumab | Colorectal cancer | 2004 | Targeted | Metastatic | — | 813 | OS: median 20.3 vs 15.6 months, HR 0.66Milestone: Bevacizumab added to first-line irinotecan-based chemotherapy |
| IALT | Lung cancer | 2004 | Chemotherapy | Adjuvant | — | 1,867 | OS: 44.5% vs 40.4% at 60 mo, HR 0.86 (95% CI 0.76–0.98)Milestone: Adjuvant cisplatin after resection: IALT, JBR.10 and LACE |
| IWWD | Colorectal cancer | 2004 | Surgery, Radiation | Neoadjuvant, Locally advanced | observational | 880 | Cumulative incidence of local regrowth: see trialMilestone: Watch-and-wait after chemoradiotherapy for complete clinical response |
| japan-gast-metachronous-hp | Gastric and oesophageal cancer | 2004 | Detection & prevention | Prevention | — | 544 | Metachronous gastric cancer: HR 0.339 (95% CI 0.157–0.729)Milestone: H. pylori eradication to prevent gastric cancer: trials 2004-2024 |
| JBR.10 | Lung cancer | 2004 | Chemotherapy | Adjuvant | 3 | 482 | OS: median 94 vs 73 months, HR 0.69Milestone: Adjuvant cisplatin after resection: IALT, JBR.10 and LACE |
| JCOG0505 | Cervical cancer | 2004 | Chemotherapy | Metastatic | 3 | 253 | OS: median 17.5 vs 18.3 months, HR 0.994Milestone: Cisplatin-paclitaxel and other platinum doublets for advanced disease |
| LACE⚑ needs review | Lung cancer | 2004 | Chemotherapy | Adjuvant | meta-analysis | 4,584 | OS: HR 0.89 (95% CI 0.82–0.96)Milestone: Adjuvant cisplatin after resection: IALT, JBR.10 and LACE |
| lamivudine-hbv-advanced-liver-disease | Hepatocellular carcinoma | 2004 | Detection & prevention | Prevention | — | 651 | Disease progression in chronic hepatitis B: 7.8% vs 17.7%, HR 0.45Milestone: Lamivudine in advanced hepatitis B: fewer HCC, a borderline finding |
| linqu-hp-cluster | Gastric and oesophageal cancer | 2004 | Detection & prevention | Prevention | — | 180,284 | Gastric cancer incidence: HR 0.86 (95% CI 0.74–0.99)Milestone: H. pylori eradication to prevent gastric cancer: trials 2004-2024 |
| metagist-imatinib-dose | Sarcoma and GIST | 2004 | Targeted | Metastatic | meta-analysis | 1,640 | PFS: HR 0.89 (95% CI 0.79–1.00)Milestone: Imatinib 400 mg vs 800 mg daily in advanced GIST |
| MOSAIC | Colorectal cancer | 2004 | Chemotherapy | Adjuvant | 3 | 2,246 | DFS: 78.2% vs 72.9% at 36 mo, HR 0.77Milestone: MOSAIC: oxaliplatin added to adjuvant fluorouracil for colon cancer |
| NSABP C-07 | Colorectal cancer | 2004 | Chemotherapy | Adjuvant | 3 | 2,492 | DFS: 76.1% vs 71.8% at 36 mo, HR 0.80 (95% CI 0.69–0.93)Milestone: MOSAIC: oxaliplatin added to adjuvant fluorouracil for colon cancer |
| RTOG 9501 | Head and neck cancer | 2004 | Chemotherapy, Radiation | Adjuvant | 3 | 459 | Locoregional control: 82% vs 72% at 24 mo, HR 0.61 (95% CI 0.41–0.91)Milestone: Adding cisplatin to postoperative radiotherapy for high-risk disease |
| s0033-gist | Sarcoma and GIST | 2004 | Targeted | Metastatic | 3 | 746 | PFS: median 20 vs 18 monthsMilestone: Imatinib 400 mg vs 800 mg daily in advanced GIST |
| SWOG 9916 | Prostate cancer | 2004 | Chemotherapy | Metastatic | 3 | 770 | OS: median 17.5 vs 15.6 months, HR 0.80 (95% CI 0.67–0.97)Milestone: Docetaxel for castration-resistant disease (TAX 327, SWOG 9916) |
| TAX 327 | Prostate cancer | 2004 | Chemotherapy | Metastatic | 3 | 1,006 | OS: median 18.9 vs 16.5 months, HR 0.76 (95% CI 0.62–0.94)Milestone: Docetaxel for castration-resistant disease (TAX 327, SWOG 9916) |
| wong-2004-fujian-hp | Gastric and oesophageal cancer | 2004 | Detection & prevention | Prevention | — | 1,630 | No results recordedMilestone: H. pylori eradication to prevent gastric cancer: trials 2004-2024 |
| AALL0434 | Childhood acute lymphoblastic leukemia (ALL) | 2005 | Chemotherapy | Relapsed / refractory, Any setting | 3 | 1,562 | DFS: 88.2% vs 82.1% at 60 moMilestone: Nelarabine for T-cell ALL |
| b31-n9831-joint | Breast cancer | 2005 | Targeted, Chemotherapy | Adjuvant | 3 | — | DFS: HR 0.48Milestone: Adding trastuzumab to adjuvant chemotherapy (HERA, B-31/N9831) |
| eortc-26981-22981 | Glioblastoma | 2005 | Chemotherapy, Radiation | Adjuvant, Maintenance | 3 | 573 | OS: median 14.6 vs 12.1 months, HR 0.63 (95% CI 0.52–0.75)Milestone: Temozolomide added to radiotherapy for newly diagnosed glioblastoma |
| GOG-122 | Endometrial cancer | 2005 | Chemotherapy, Radiation | Adjuvant | 3 | 422 | PFS: 50% vs 38% at 60 mo, HR 0.71 (95% CI 0.55–0.91)Milestone: GOG-122: doxorubicin-cisplatin versus whole-abdominal irradiation |
| HERA | Breast cancer | 2005 | Targeted, Chemotherapy | Adjuvant | 3 | — | DFS: HR 0.54 (95% CI 0.43–0.67)Milestone: Adding trastuzumab to adjuvant chemotherapy (HERA, B-31/N9831) |
| INT-0133 | Sarcoma and GIST | 2005 | Chemotherapy, Immunotherapy | Adjuvant | — | 677 | EFS: 68% vs 71% at 36 moMilestone: INT-0133: mifamurtide added to chemotherapy; EU authorisation |
| ISEL | Lung cancer | 2005 | Targeted | Relapsed / refractory | 3 | 1,692 | OS: median 5.6 vs 5.1 months, HR 0.89 (95% CI 0.77–1.02)Milestone: Gefitinib: accelerated approval, restriction and EGFR-mutation return |
| MRC TE19 / EORTC 30982 | Testicular germ cell tumors | 2005 | Chemotherapy, Radiation | Adjuvant | 3 | 1,477 | RFR: 97.7% vs 96.7% at 24 mo, HR 1.28Milestone: TE19: single-dose carboplatin vs radiotherapy, stage I seminoma |
| ncctg-n9831 | Breast cancer | 2005 | Targeted, Chemotherapy | Adjuvant | 3 | — | No results recordedMilestone: Adding trastuzumab to adjuvant chemotherapy (HERA, B-31/N9831) |
| nelarabine-cog-phase2 | Childhood acute lymphoblastic leukemia (ALL) | 2005 | Chemotherapy | Relapsed / refractory, Any setting | 2 | 121 | CR+PR rate: see trialMilestone: Nelarabine for T-cell ALL |
| nsabp-b31 | Breast cancer | 2005 | Targeted, Chemotherapy | Adjuvant | 3 | — | No results recordedMilestone: Adding trastuzumab to adjuvant chemotherapy (HERA, B-31/N9831) |
| TARGET | Kidney cancer (renal cell carcinoma) | 2005 | Targeted | Metastatic | 3 | 903 | PFS: median 5.5 vs 2.8 months, HR 0.44 (95% CI 0.35–0.55)Milestone: Sorafenib in previously treated RCC: TARGET and FDA approval |
| travis-2005-second-cancers | Testicular germ cell tumors | 2005 | Supportive care, Radiation, Chemotherapy | Any setting | observational | 40,576 | Cumulative second solid cancer risk: 36% vs 23% at 480 moMilestone: Second cancers among 40,576 testicular cancer patients |
| ala-glioma-5-ala-fluorescence-guided-resection | Glioblastoma | 2006 | Surgery | Localised | 3 | 322 | Complete resection of contrast-enhancing tumour: 65% vs 36%Milestone: 5-ALA fluorescence-guided surgery increases complete resection |
| bonner-radiotherapy-cetuximab-hn | Head and neck cancer | 2006 | Targeted, Radiation | Locally advanced | 3 | — | Locoregional control: median 24.4 vs 14.9 months, HR 0.68Milestone: Cetuximab with radiotherapy improves locoregional control and survival |
| chen-2006-plat-resection⚑ needs review | Hepatocellular carcinoma | 2006 | Surgery | Localised | — | 180 | OS: 95.8% vs 93.3% at 12 moMilestone: Resection versus ablation for small HCC: conflicting trials |
| dasatinib-phase1-2006 | Chronic myeloid leukemia | 2006 | Targeted | Relapsed / refractory | 1 | — | MCyR: see trialMilestone: Dasatinib and nilotinib for imatinib resistance or intolerance |
| FUTURE II | Cross-cancer | 2006 | Detection & prevention | Any setting | 3 | 12,167 | No results recordedMilestone: First US-licensed HPV vaccine; later data show fewer cervical cancers |
| GOG-172 | Ovarian cancer | 2006 | Chemotherapy | Locally advanced | 3 | 429 | PFS: median 23.8 vs 18.3 monthsMilestone: Intraperitoneal chemotherapy in optimal stage III: GOG-172 |
| huang-2010-rfa-resection⚑ needs review | Hepatocellular carcinoma | 2006 | Surgery | Localised | — | 230 | OS: 86.96% vs 98.26% at 12 moMilestone: Resection versus ablation for small HCC: conflicting trials |
| lenalidomide-high-vs-low-dose-dexamethasone-ecog | Multiple myeloma | 2006 | Targeted, Immunotherapy | Relapsed / refractory | — | 445 | ORR: 68% vs 79%Milestone: Lenalidomide plus dexamethasone in relapsed myeloma: MM-009, MM-010 |
| MAGIC | Gastric and oesophageal cancer | 2006 | Chemotherapy, Surgery | Perioperative | 3 | — | OS: 36% vs 23% at 60 mo, HR 0.75 (95% CI 0.60–0.93)Milestone: MAGIC: perioperative ECF for resectable gastro-oesophageal cancer |
| MInT | Non-Hodgkin lymphoma (DLBCL and follicular) | 2006 | Immunotherapy, Chemotherapy | Locally advanced | — | 824 | EFS: 79% vs 59% at 36 moMilestone: MInT: rituximab with CHOP-like therapy, young low-risk DLBCL (2006) |
| MM-009 | Multiple myeloma | 2006 | Targeted, Immunotherapy | Relapsed / refractory | 3 | 353 | TTP: median 11.1 vs 4.7 monthsMilestone: Lenalidomide plus dexamethasone in relapsed myeloma: MM-009, MM-010 |
| MM-010 | Multiple myeloma | 2006 | Targeted, Immunotherapy | Relapsed / refractory | 3 | 351 | OS: HR 0.66Milestone: Lenalidomide plus dexamethasone in relapsed myeloma: MM-009, MM-010 |
| nilotinib-phase1-2006 | Chronic myeloid leukemia | 2006 | Targeted | Relapsed / refractory | 1 | 119 | No results recordedMilestone: Dasatinib and nilotinib for imatinib resistance or intolerance |
| REVEAL-HBV | Hepatocellular carcinoma | 2006 | Understanding | Prevention | observational | 3,653 | Incidence of hepatocellular carcinoma: 14.9% vs 1.3%Milestone: REVEAL-HBV: HCC risk rises with serum hepatitis B virus DNA level |
| sunitinib-gist-a6181004 | Sarcoma and GIST | 2006 | Targeted | Relapsed / refractory | 3 | 312 | TTP: HR 0.33Milestone: Sunitinib after imatinib failure in GIST |
| sunitinib-vs-interferon-rcc | Kidney cancer (renal cell carcinoma) | 2006 | Targeted | Metastatic | 3 | 750 | PFS: median 11 vs 5 months, HR 0.42 (95% CI 0.32–0.54)Milestone: Sunitinib versus interferon alfa as first-line therapy (2006-2007) |
| SURF⚑ needs review | Hepatocellular carcinoma | 2006 | Surgery | Localised | — | 302 | RFS: median 3.5 vs 3 years, HR 0.92 (95% CI 0.67–1.25)Milestone: Resection versus ablation for small HCC: conflicting trials |
| sylvester-2004-immediate-instillation-meta-analysis | Bladder cancer | 2006 | Understanding | Localised | meta-analysis | 1,476 | Recurrence after resection: 36.7% vs 48.4%Milestone: EORTC risk tables for non-muscle-invasive bladder cancer |
| ACTS-GC | Gastric and oesophageal cancer | 2007 | Chemotherapy | Adjuvant | 3 | — | OS: 80.1% vs 70.1% at 36 mo, HR 0.68 (95% CI 0.52–0.87)Milestone: Adjuvant S-1 (ACTS-GC) and capecitabine-oxaliplatin (CLASSIC) after D2 |
| ARCC | Kidney cancer (renal cell carcinoma) | 2007 | Targeted | Metastatic | 3 | 626 | OS: median 10.9 vs 7.3 months, HR 0.73 (95% CI 0.58–0.92)Milestone: mTOR inhibitors: temsirolimus (ARCC) and everolimus (RECORD-1) |
| asia-pacific-sorafenib-hcc | Hepatocellular carcinoma | 2007 | Targeted | Metastatic | 3 | 226 | OS: median 6.5 vs 4.2 months, HR 0.68 (95% CI 0.50–0.93)Milestone: Sorafenib: SHARP and Asia-Pacific trials in advanced HCC |
| CLASSIC | Gastric and oesophageal cancer | 2007 | Chemotherapy | Adjuvant | 3 | 1,035 | DFS: 74% vs 59% at 36 mo, HR 0.56 (95% CI 0.44–0.72)Milestone: Adjuvant S-1 (ACTS-GC) and capecitabine-oxaliplatin (CLASSIC) after D2 |
| CONKO-001 | Pancreatic cancer | 2007 | Chemotherapy | Adjuvant | 3 | 368 | DFS: median 13.4 vs 6.9 monthsMilestone: CONKO-001: adjuvant gemcitabine versus observation |
| einhorn-2007-hdct-indiana-retrospective | Testicular germ cell tumors | 2007 | Chemotherapy, Cell therapy & transplant | Relapsed / refractory | observational | 184 | No results recordedMilestone: High-dose chemotherapy with stem-cell rescue for relapsed disease |
| EORTC 30974⚑ needs review | Testicular germ cell tumors | 2007 | Chemotherapy, Cell therapy & transplant | Metastatic | 3 | 137 | Complete response rate: 44.6% vs 33.3%Milestone: First-line high-dose chemotherapy did not show benefit in trials |
| Interfant-06 | Childhood acute lymphoblastic leukemia (ALL) | 2007 | Chemotherapy, Cell therapy & transplant | Any setting | 3 | 651 | EFS: see trialMilestone: Interfant protocols for infants with ALL |
| Interfant-99 | Childhood acute lymphoblastic leukemia (ALL) | 2007 | Chemotherapy, Cell therapy & transplant | Any setting | 3 | 482 | EFS: see trialMilestone: Interfant protocols for infants with ALL |
| IT-94⚑ needs review | Testicular germ cell tumors | 2007 | Chemotherapy, Cell therapy & transplant | Relapsed / refractory | 3 | 280 | EFS: 42% vs 35% at 36 moMilestone: High-dose chemotherapy with stem-cell rescue for relapsed disease |
| keimeguibert-2007-elderly-radiotherapy-supportive-care | Glioblastoma | 2007 | Radiation, Supportive care | Adjuvant | 3 | 85 | OS: HR 0.47 (95% CI 0.29–0.76)Milestone: Radiotherapy improves survival in older people with glioblastoma |
| motzer-2007-hdct-first-line | Testicular germ cell tumors | 2007 | Chemotherapy, Cell therapy & transplant | Metastatic | 3 | 219 | Durable complete response rate: 52% vs 48%Milestone: First-line high-dose chemotherapy did not show benefit in trials |
| RECORD-1 | Kidney cancer (renal cell carcinoma) | 2007 | Targeted | Metastatic | 3 | — | PFS: median 4 vs 1.9 months, HR 0.30 (95% CI 0.22–0.40)Milestone: mTOR inhibitors: temsirolimus (ARCC) and everolimus (RECORD-1) |
| SHARP | Hepatocellular carcinoma | 2007 | Targeted | Metastatic | 3 | 602 | OS: median 10.7 vs 7.9 months, HR 0.69 (95% CI 0.55–0.87)Milestone: Sorafenib: SHARP and Asia-Pacific trials in advanced HCC |
| ANBL00P2⚑ needs review | Neuroblastoma and Wilms tumour | 2008 | Surgery | Localised | — | 87 | EFS: see trialMilestone: Low-risk neuroblastoma: observation or surgery alone |
| ASTEC⚑ needs review | Endometrial cancer | 2008 | Surgery | Localised | 3 | 1,408 | OS: HR 1.16 (95% CI 0.87–1.54)Milestone: Systematic lymphadenectomy: no survival benefit shown in two trials |
| benedetti-panici-lymphadenectomy | Endometrial cancer | 2008 | Surgery | Localised | — | 514 | DFS: 81% vs 81.7% at 60 mo, HR 1.10 (95% CI 0.70–1.71)Milestone: Systematic lymphadenectomy: no survival benefit shown in two trials |
| CCCMAC⚑ needs review | Cervical cancer | 2008 | Chemotherapy, Radiation | Locally advanced | meta-analysis | — | OS: HR 0.81Milestone: Chemoradiation individual patient data meta-analysis (2008) |
| CCLG-ENSG-5 | Neuroblastoma and Wilms tumour | 2008 | Chemotherapy, Cell therapy & transplant | Any setting | 3 | 262 | EFS: 31% vs 24.2% at 36 mo, HR 0.86 (95% CI 0.66–1.14)Milestone: European trials: rapid induction and busulfan-melphalan consolidation |
| eortc-62024-gist | Sarcoma and GIST | 2008 | Targeted, Surgery | Adjuvant | 3 | 908 | IFFS: 87% vs 84% at 60 mo, HR 0.79Milestone: Adjuvant imatinib after GIST resection: 1 year, then 3 years |
| EXTREME | Head and neck cancer | 2008 | Targeted, Chemotherapy | Metastatic | 3 | 442 | OS: median 10.1 vs 7.4 months, HR 0.80 (95% CI 0.64–0.99)Milestone: EXTREME: cetuximab added to platinum-fluorouracil, first line |
| HR-NBL1.5 | Neuroblastoma and Wilms tumour | 2008 | Chemotherapy, Cell therapy & transplant | Any setting | — | 630 | mCR rate: 35% vs 32%Milestone: European trials: rapid induction and busulfan-melphalan consolidation |
| HR-NBL1/SIOPEN | Neuroblastoma and Wilms tumour | 2008 | Chemotherapy, Cell therapy & transplant | Any setting | 3 | 598 | EFS: 50% vs 38% at 36 moMilestone: European trials: rapid induction and busulfan-melphalan consolidation |
| NB95-S and NB97 | Neuroblastoma and Wilms tumour | 2008 | Surgery | Localised | — | 340 | No results recordedMilestone: Low-risk neuroblastoma: observation or surgery alone |
| P9641 | Neuroblastoma and Wilms tumour | 2008 | Surgery | Localised | 3 | 915 | OS: see trialMilestone: Low-risk neuroblastoma: observation or surgery alone |
| REAL-2 | Gastric and oesophageal cancer | 2008 | Chemotherapy | Metastatic | 3 | 1,002 | OS: HR 0.86 (95% CI 0.80–0.99)Milestone: REAL-2: oxaliplatin and capecitabine, advanced oesophagogastric |
| ssg-xviii-aio | Sarcoma and GIST | 2008 | Targeted, Surgery | Adjuvant | 3 | 400 | RFS: 65.6% vs 47.9% at 60 mo, HR 0.46 (95% CI 0.32–0.65)Milestone: Adjuvant imatinib after GIST resection: 1 year, then 3 years |
| VISTA | Multiple myeloma | 2008 | Targeted, Chemotherapy | Any setting | 3 | 682 | TTP: median 24 vs 16.6 months, HR 0.48Milestone: VISTA: bortezomib added to melphalan-prednisone, first line |
| Z9001 | Sarcoma and GIST | 2008 | Targeted, Surgery | Adjuvant | 3 | — | RFS: 98% vs 83% at 12 mo, HR 0.35 (95% CI 0.22–0.53)Milestone: Adjuvant imatinib after GIST resection: 1 year, then 3 years |
| AALL0031 | Childhood acute lymphoblastic leukemia (ALL) | 2009 | Targeted, Chemotherapy | Any setting | — | 92 | EFS: 80% vs 35% at 36 moMilestone: Imatinib added to chemotherapy for Ph-positive ALL |
| aza-001-aml-subgroup | Acute myeloid leukaemia (including APL) | 2009 | Chemotherapy | Any setting | 3 | 113 | OS: median 24.5 vs 16 months, HR 0.47 (95% CI 0.28–0.79)Milestone: Azacitidine and decitabine in older adults with AML |
| AZA-AML-001 | Acute myeloid leukaemia (including APL) | 2009 | Chemotherapy | Any setting | 3 | 488 | OS: median 10.4 vs 6.5 months, HR 0.85 (95% CI 0.69–1.03)Milestone: Azacitidine and decitabine in older adults with AML |
| BRAIN | Glioblastoma | 2009 | Targeted | Relapsed / refractory | 2 | 167 | ORR: see trialMilestone: Bevacizumab approved for progressive glioblastoma on response rate |
| COMPARZ | Kidney cancer (renal cell carcinoma) | 2009 | Targeted | Metastatic | 3 | 1,110 | PFS: HR 1.05 (95% CI 0.90–1.22)Milestone: Pazopanib approved (2009); COMPARZ compares it with sunitinib |
| DACO-016 | Acute myeloid leukaemia (including APL) | 2009 | Chemotherapy | Any setting | 3 | 485 | OS: median 7.7 vs 5 months, HR 0.85 (95% CI 0.69–1.04)Milestone: Azacitidine and decitabine in older adults with AML |
| ERSPC | Prostate cancer | 2009 | Detection & prevention | Screening | — | 182,160 | Cumulative incidence of prostate cancer: 8.2% vs 4.8%Milestone: Prostate screening trials: ERSPC, PLCO and Goteborg (2009-2010) |
| EsPhALL2004 | Childhood acute lymphoblastic leukemia (ALL) | 2009 | Targeted, Chemotherapy | Any setting | — | 178 | DFS: 72.9% vs 61.7% at 48 mo, HR 0.63 (95% CI 0.28–1.41)Milestone: Imatinib added to chemotherapy for Ph-positive ALL |
| Göteborg-1 | Prostate cancer | 2009 | Detection & prevention | Screening | — | 20,000 | Cumulative incidence of prostate cancer: 12.7% vs 8.2%, HR 1.64 (95% CI 1.5–1.8)Milestone: Prostate screening trials: ERSPC, PLCO and Goteborg (2009-2010) |
| IPASS | Lung cancer | 2009 | Targeted | Metastatic | 3 | — | PFS: 24.9% vs 6.7% at 12 mo, HR 0.74 (95% CI 0.65–0.85)Milestone: IPASS: gefitinib vs chemotherapy, by EGFR mutation (2009) |
| klotz-active-surveillance-cohort | Prostate cancer | 2009 | Surgery, Radiation | Localised | observational | 993 | OS: see trialMilestone: Active surveillance cohort: selective delayed intervention (Klotz) |
| LACE | Endometrial cancer | 2009 | Surgery | Localised | 3 | 760 | DFS: 81.6% vs 81.3% at 54 moMilestone: LAP2 and LACE: laparoscopic vs open surgery for uterine cancer |
| LAP2⚑ needs review | Endometrial cancer | 2009 | Surgery | Localised | 3 | 2,616 | Recurrence-free interval: HR 1.14Milestone: LAP2 and LACE: laparoscopic vs open surgery for uterine cancer |
| pazopanib-vs-placebo-rcc | Kidney cancer (renal cell carcinoma) | 2009 | Targeted | Metastatic | 3 | 435 | PFS: median 9.2 vs 4.2 months, HR 0.46 (95% CI 0.34–0.62)Milestone: Pazopanib approved (2009); COMPARZ compares it with sunitinib |
| PLCO | Prostate cancer | 2009 | Detection & prevention | Screening | — | 76,693 | Men with any PSA testing during the trial: 99% vs 86%Milestone: Prostate screening trials: ERSPC, PLCO and Goteborg (2009-2010) |
| Total 16 | Childhood acute lymphoblastic leukemia (ALL) | 2009 | Chemotherapy, Radiation | Any setting | 3 | — | EFS: see trialMilestone: Treating ALL without prophylactic cranial irradiation |
| Total XV | Childhood acute lymphoblastic leukemia (ALL) | 2009 | Chemotherapy, Radiation | Any setting | 3 | — | EFS: see trialMilestone: Treating ALL without prophylactic cranial irradiation |
| A3961 | Neuroblastoma and Wilms tumour | 2010 | Chemotherapy, Surgery | Any setting | 3 | 479 | OS: see trialMilestone: Intermediate-risk neuroblastoma: reduced chemotherapy (COG A3961) |
| ALFA-0701 | Acute myeloid leukaemia (including APL) | 2010 | Antibody–drug conjugate | Any setting | 3 | 280 | EFS: 40.8% vs 17.1% at 24 mo, HR 0.58 (95% CI 0.43–0.78)Milestone: Gemtuzumab ozogamicin: approved 2000, withdrawn 2010, back 2017 |
| ALL R3 | Childhood acute lymphoblastic leukemia (ALL) | 2010 | Chemotherapy, Cell therapy & transplant, Radiation | Relapsed / refractory | — | 216 | PFS: 64.6% vs 35.9% at 36 moMilestone: Mitoxantrone in first relapse; TBI before transplant in childhood ALL |
| ANBL0032 | Neuroblastoma and Wilms tumour | 2010 | Immunotherapy | Maintenance | 3 | 226 | EFS: 66% vs 46% at 24 moMilestone: Anti-GD2 dinutuximab with cytokines after transplant (ANBL0032) |
| AVANT | Colorectal cancer | 2010 | Targeted, Chemotherapy | Adjuvant | 3 | 3,451 | DFS: HR 1.17 (95% CI 0.98–1.39)Milestone: Adjuvant bevacizumab and cetuximab trials in colon cancer |
| ca184-024 | Melanoma | 2010 | Immunotherapy | Metastatic | 3 | 502 | OS: median 11.2 vs 9.1 months, HR 0.72Milestone: Ipilimumab: anti-CTLA-4 survival trials and FDA approval (2010-2011) |
| CALGB 80303 | Pancreatic cancer | 2010 | Targeted | Locally advanced, Metastatic | 3 | 602 | OS: median 5.8 vs 5.9 monthsMilestone: Bevacizumab, cetuximab, PEGPH20 added to chemotherapy: no gain |
| CARD | Prostate cancer | 2010 | Chemotherapy | Metastatic | — | 255 | Imaging-based PFS: median 8 vs 3.7 months, HR 0.54 (95% CI 0.40–0.73)Milestone: Cabazitaxel after docetaxel (TROPIC) |
| CHORUS | Ovarian cancer | 2010 | Surgery, Chemotherapy | Neoadjuvant | 3 | 552 | OS: median 24.1 vs 22.6 months, HR 0.87 (95% CI 0.72–1.05)Milestone: Neoadjuvant chemotherapy vs primary surgery: EORTC 55971, CHORUS |
| CLL4 | Chronic lymphocytic leukaemia | 2010 | Chemotherapy, Immunotherapy | Metastatic | 3 | 375 | CR rate: 24% vs 7%Milestone: CLL8: rituximab added to fludarabine and cyclophosphamide |
| CLL8 | Chronic lymphocytic leukaemia | 2010 | Chemotherapy, Immunotherapy | Metastatic | 3 | 817 | PFS: 65% vs 45% at 36 mo, HR 0.56 (95% CI 0.46–0.69)Milestone: CLL8: rituximab added to fludarabine and cyclophosphamide |
| CORAL | Non-Hodgkin lymphoma (DLBCL and follicular) | 2010 | Chemotherapy, Immunotherapy, Cell therapy & transplant | Relapsed / refractory | 3 | 396 | Response rate: 63.5% vs 62.8%Milestone: CORAL: R-ICE vs R-DHAP before transplant in relapsed DLBCL |
| DASISION⚑ needs review | Chronic myeloid leukemia | 2010 | Targeted | Any setting | 3 | 519 | CCyR: 77% vs 66%Milestone: DASISION and ENESTnd: second-generation TKIs first line |
| ENESTnd⚑ needs review | Chronic myeloid leukemia | 2010 | Targeted | Any setting | 3 | 846 | MMR: 44% vs 22%Milestone: DASISION and ENESTnd: second-generation TKIs first line |
| EORTC 30904 | Kidney cancer (renal cell carcinoma) | 2010 | Surgery | Localised | 3 | 541 | OS: 75.7% vs 81.1% at 120 mo, HR 1.50 (95% CI 1.03–2.16)Milestone: EORTC 30904: nephron-sparing surgery versus radical nephrectomy |
| EORTC 55971 | Ovarian cancer | 2010 | Surgery, Chemotherapy | Neoadjuvant | 3 | 670 | OS: HR 0.98Milestone: Neoadjuvant chemotherapy vs primary surgery: EORTC 55971, CHORUS |
| FORUM | Childhood acute lymphoblastic leukemia (ALL) | 2010 | Chemotherapy, Cell therapy & transplant, Radiation | Relapsed / refractory | 3 | 413 | OS: 75% vs 91% at 24 moMilestone: Mitoxantrone in first relapse; TBI before transplant in childhood ALL |
| HALO 109-301 | Pancreatic cancer | 2010 | Targeted | Locally advanced, Metastatic | 3 | 492 | OS: median 11.2 vs 11.5 months, HR 1.00 (95% CI 0.80–1.27)Milestone: Bevacizumab, cetuximab, PEGPH20 added to chemotherapy: no gain |
| HD10 | Hodgkin lymphoma | 2010 | Chemotherapy, Radiation | Localised | 3 | 1,370 | FFTF: 91.1% vs 93% at 60 moMilestone: HD10: fewer cycles and lower dose in early favourable HL |
| IMPACT | Prostate cancer | 2010 | Immunotherapy, Cell therapy & transplant | Metastatic | 3 | 512 | OS: median 25.8 vs 21.7 months, HR 0.78 (95% CI 0.61–0.98)Milestone: Sipuleucel-T autologous cellular immunotherapy (IMPACT) |
| mdx010-20 | Melanoma | 2010 | Immunotherapy | Metastatic | 3 | 676 | OS: median 10 vs 6.4 months, HR 0.68Milestone: Ipilimumab: anti-CTLA-4 survival trials and FDA approval (2010-2011) |
| mylotarg-phase2-first-relapse | Acute myeloid leukaemia (including APL) | 2010 | Antibody–drug conjugate | Any setting | 2 | 142 | Overall remission rate: see trialMilestone: Gemtuzumab ozogamicin: approved 2000, withdrawn 2010, back 2017 |
| N0147 | Colorectal cancer | 2010 | Targeted, Chemotherapy | Adjuvant | 3 | 2,686 | DFS: 71.5% vs 74.6% at 36 mo, HR 1.21 (95% CI 0.98–1.49)Milestone: Adjuvant bevacizumab and cetuximab trials in colon cancer |
| NORCCAP | Colorectal cancer | 2010 | Detection & prevention | Screening | — | 100,210 | Colorectal cancer mortality: HR 0.73 (95% CI 0.56–0.94)Milestone: Flexible sigmoidoscopy screening trials |
| NSABP C-08 | Colorectal cancer | 2010 | Targeted, Chemotherapy | Adjuvant | 3 | 2,672 | DFS: 77.4% vs 75.5% at 36 mo, HR 0.89 (95% CI 0.76–1.04)Milestone: Adjuvant bevacizumab and cetuximab trials in colon cancer |
| PETACC-8 | Colorectal cancer | 2010 | Targeted, Chemotherapy | Adjuvant | 3 | 2,559 | DFS: HR 1.05 (95% CI 0.85–1.29)Milestone: Adjuvant bevacizumab and cetuximab trials in colon cancer |
| PLCO | Colorectal cancer | 2010 | Detection & prevention | Screening | — | 154,900 | No results recordedMilestone: Flexible sigmoidoscopy screening trials |
| PORTEC-2⚑ needs review | Endometrial cancer | 2010 | Radiation | Adjuvant | 3 | 427 | Vaginal recurrence: 1.8% vs 1.6% at 60 mo, HR 0.78 (95% CI 0.17–3.49)Milestone: PORTEC-2: vaginal brachytherapy in place of pelvic radiotherapy |
| PRIMA | Non-Hodgkin lymphoma (DLBCL and follicular) | 2010 | Immunotherapy | Maintenance | 3 | 1,217 | PFS: 74.9% vs 57.6%, HR 0.55 (95% CI 0.44–0.68)Milestone: PRIMA: two years of rituximab maintenance after induction (2010) |
| PROFILE 1001 | Lung cancer | 2010 | Targeted | Metastatic | 1 | 82 | ORR: see trialMilestone: Crizotinib for ALK-positive lung cancer (2010-2014) |
| PROFILE 1007 | Lung cancer | 2010 | Targeted | Metastatic | 3 | 347 | PFS: median 7.7 vs 3 months, HR 0.49 (95% CI 0.37–0.64)Milestone: Crizotinib for ALK-positive lung cancer (2010-2014) |
| PROFILE 1014 | Lung cancer | 2010 | Targeted | Metastatic | 3 | 343 | PFS: median 10.9 vs 7 months, HR 0.45 (95% CI 0.35–0.60)Milestone: Crizotinib for ALK-positive lung cancer (2010-2014) |
| PROSELICA | Prostate cancer | 2010 | Chemotherapy | Metastatic | 3 | 1,200 | OS: median 13.4 vs 14.5 months, HR 1.024Milestone: Cabazitaxel after docetaxel (TROPIC) |
| RTOG 0129 | Head and neck cancer | 2010 | Understanding | Locally advanced | observational | 323 | OS: 82.4% vs 57.1% at 36 mo, HR 0.42 (95% CI 0.27–0.66)Milestone: Tumour HPV status found to be a strong, independent prognostic factor |
| RTOG 0236 | Lung cancer | 2010 | Radiation | Localised | 2 | 59 | Primary tumour control: see trialMilestone: SBRT for medically inoperable early-stage lung cancer (RTOG 0236) |
| S0106 | Acute myeloid leukaemia (including APL) | 2010 | Antibody–drug conjugate | Any setting | 3 | 637 | DFS: HR 1.48Milestone: Gemtuzumab ozogamicin: approved 2000, withdrawn 2010, back 2017 |
| S0205 | Pancreatic cancer | 2010 | Targeted | Locally advanced, Metastatic | 3 | 745 | OS: median 6.3 vs 5.9 months, HR 1.06 (95% CI 0.91–1.23)Milestone: Bevacizumab, cetuximab, PEGPH20 added to chemotherapy: no gain |
| SCORE | Colorectal cancer | 2010 | Detection & prevention | Screening | — | — | No results recordedMilestone: Flexible sigmoidoscopy screening trials |
| SCORPION | Ovarian cancer | 2010 | Surgery, Chemotherapy | Neoadjuvant | 3 | 171 | PFS: median 14 vs 15 months, HR 1.05 (95% CI 0.77–1.44)Milestone: Neoadjuvant chemotherapy vs primary surgery: EORTC 55971, CHORUS |
| STIM | Chronic myeloid leukemia | 2010 | Targeted | Maintenance | — | 100 | MRFS: see trialMilestone: STIM: stopping imatinib after sustained molecular response |
| ToGA | Gastric and oesophageal cancer | 2010 | Targeted, Chemotherapy | Metastatic | 3 | 594 | OS: median 13.8 vs 11.1 months, HR 0.74 (95% CI 0.60–0.91)Milestone: ToGA: trastuzumab plus chemotherapy in HER2-positive gastric cancer |
| TROPIC | Prostate cancer | 2010 | Chemotherapy | Metastatic | 3 | 755 | OS: median 15.1 vs 12.7 months, HR 0.70 (95% CI 0.59–0.83)Milestone: Cabazitaxel after docetaxel (TROPIC) |
| uk-flexible-sigmoidoscopy-screening-trial | Colorectal cancer | 2010 | Detection & prevention | Screening | — | 170,432 | Colorectal cancer mortality: HR 0.69 (95% CI 0.59–0.82)Milestone: Flexible sigmoidoscopy screening trials |
| AVADO | Breast cancer | 2011 | Targeted | Metastatic | 3 | 736 | PFS: median 10.1 vs 8.2 months, HR 0.77Milestone: FDA withdraws bevacizumab for metastatic breast cancer (2011) |
| break-3 | Melanoma | 2011 | Targeted | Metastatic | 3 | 250 | PFS: median 5.1 vs 2.7 months, HR 0.30 (95% CI 0.18–0.51)Milestone: BRAF inhibition: vemurafenib (BRIM-3) and companions, 2011-2012 |
| BRIM-3 | Melanoma | 2011 | Targeted | Metastatic | 3 | 675 | OS: 84% vs 64% at 6 mo, HR 0.37 (95% CI 0.26–0.55)Milestone: BRAF inhibition: vemurafenib (BRIM-3) and companions, 2011-2012 |
| CLEOPATRA | Breast cancer | 2011 | Targeted | Metastatic | 3 | 808 | PFS: median 18.5 vs 12.4 months, HR 0.62 (95% CI 0.51–0.75)Milestone: CLEOPATRA: pertuzumab added to trastuzumab and docetaxel |
| COU-AA-301 | Prostate cancer | 2011 | Endocrine | Metastatic | 3 | 1,195 | OS: median 14.8 vs 10.9 months, HR 0.65 (95% CI 0.54–0.77)Milestone: Abiraterone after docetaxel improves survival (COU-AA-301) |
| E2100 | Breast cancer | 2011 | Targeted | Metastatic | 3 | 722 | PFS: median 11.8 vs 5.9 months, HR 0.6Milestone: FDA withdraws bevacizumab for metastatic breast cancer (2011) |
| FIRES | Endometrial cancer | 2011 | Surgery, Understanding | Localised | observational | 385 | Sentinel-node sensitivity: see trialMilestone: Sentinel node biopsy tested as an alternative to lymphadenectomy |
| GOG-0218 | Ovarian cancer | 2011 | Targeted, Chemotherapy | Locally advanced, Metastatic, Maintenance | 3 | 1,873 | PFS: median 14.1 vs 10.3 months, HR 0.717 (95% CI 0.625–0.824)Milestone: Bevacizumab added to first-line chemotherapy: GOG-218 and ICON7 |
| ICON7 | Ovarian cancer | 2011 | Targeted, Chemotherapy | Locally advanced, Metastatic, Maintenance | 3 | 1,528 | PFS: HR 0.81 (95% CI 0.70–0.94)Milestone: Bevacizumab added to first-line chemotherapy: GOG-218 and ICON7 |
| METRIC | Melanoma | 2011 | Targeted | Metastatic | 3 | 322 | PFS: median 4.8 vs 1.5 months, HR 0.45 (95% CI 0.33–0.63)Milestone: BRAF inhibition: vemurafenib (BRIM-3) and companions, 2011-2012 |
| nih-aarp-smoking-bladder | Bladder cancer | 2011 | Understanding, Detection & prevention | Prevention | observational | — | Bladder cancer incidence: HR 2.22 (95% CI 2.03–2.44)Milestone: Smoking and bladder cancer risk in a large US cohort |
| NLST | Lung cancer | 2011 | Detection & prevention | Screening | 3 | 53,454 | Positive screening test rate: 24.2% vs 6.9%Milestone: NLST: low-dose CT screening lowers lung-cancer mortality |
| PLCO | Ovarian cancer | 2011 | Detection & prevention | Screening | — | 78,216 | No results recordedMilestone: Ovarian cancer screening: PLCO and UKCTOCS find no mortality reduction |
| PRODIGE 4 / ACCORD 11 | Pancreatic cancer | 2011 | Chemotherapy | Metastatic | 2/3 | 342 | OS: median 11.1 vs 6.8 months, HR 0.57 (95% CI 0.45–0.73)Milestone: FOLFIRINOX versus gemcitabine: PRODIGE 4 / ACCORD 11 |
| RIBBON-1 | Breast cancer | 2011 | Targeted | Metastatic | 3 | 1,237 | PFS: median 8.6 vs 5.7 months, HR 0.69 (95% CI 0.56–0.84)Milestone: FDA withdraws bevacizumab for metastatic breast cancer (2011) |
| SENTI-ENDO | Endometrial cancer | 2011 | Surgery, Understanding | Localised | observational | 133 | Sentinel-node negative predictive value: see trialMilestone: Sentinel node biopsy tested as an alternative to lymphadenectomy |
| SENTOR | Endometrial cancer | 2011 | Surgery, Understanding | Localised | observational | 156 | Sentinel-node sensitivity: see trialMilestone: Sentinel node biopsy tested as an alternative to lymphadenectomy |
| SGN-35 phase 1 | Hodgkin lymphoma | 2011 | Antibody–drug conjugate | Relapsed / refractory | 1 | 45 | ORR: see trialMilestone: Brentuximab vedotin approved for relapsed Hodgkin lymphoma |
| SGN-35 pivotal phase 2 | Hodgkin lymphoma | 2011 | Antibody–drug conjugate | Relapsed / refractory | 2 | 102 | ORR: see trialMilestone: Brentuximab vedotin approved for relapsed Hodgkin lymphoma |
| TRANSCEND CLL 004 | Chronic lymphocytic leukaemia | 2011 | Cell therapy & transplant | Relapsed / refractory | 1/2 | 117 | CR/CRi rate: see trialMilestone: CAR T cells in CLL: 2011 report to 2024 liso-cel approval |
| UKCTOCS | Ovarian cancer | 2011 | Detection & prevention | Screening | — | 202,638 | Ovarian cancer mortality: 0.29% vs 0.34%Milestone: Ovarian cancer screening: PLCO and UKCTOCS find no mortality reduction |
| ZETA | Thyroid cancer | 2011 | Targeted | Metastatic | 3 | 331 | PFS: HR 0.46 (95% CI 0.31–0.69)Milestone: ZETA: vandetanib for advanced medullary thyroid cancer |
| AEWS0031 | Sarcoma and GIST | 2012 | Chemotherapy | Localised | 3 | 587 | EFS: 73% vs 65% at 60 mo, HR 0.74 (95% CI 0.54–0.99)Milestone: AEWS0031: interval-compressed chemotherapy in Ewing sarcoma |
| AFFIRM | Prostate cancer | 2012 | Endocrine | Metastatic | 3 | 1,199 | OS: median 18.4 vs 13.6 months, HR 0.63 (95% CI 0.53–0.75)Milestone: Enzalutamide after docetaxel (AFFIRM) |
| ASPIRE | Multiple myeloma | 2012 | Targeted | Relapsed / refractory | 3 | 792 | PFS: median 26.3 vs 17.6 months, HR 0.69 (95% CI 0.57–0.83)Milestone: Carfilzomib: accelerated approval to ASPIRE and ENDEAVOR |
| AURELIA | Ovarian cancer | 2012 | Targeted, Chemotherapy | Relapsed / refractory | 3 | 361 | PFS: median 6.7 vs 3.4 months, HR 0.48 (95% CI 0.38–0.60)Milestone: Bevacizumab in recurrent disease: OCEANS, AURELIA, GOG-213 |
| BC2001 | Bladder cancer | 2012 | Radiation, Chemotherapy | Localised | 3 | 458 | Locoregional disease-free survival: 67% vs 54% at 24 mo, HR 0.68 (95% CI 0.48–0.96)Milestone: BC2001: chemotherapy added to radiotherapy for bladder preservation |
| BELA | Chronic myeloid leukemia | 2012 | Targeted | Any setting, Relapsed / refractory | 3 | 502 | CCyR: 70% vs 68%Milestone: Bosutinib: from a missed first-line endpoint (BELA) to BFORE |
| BFORE | Chronic myeloid leukemia | 2012 | Targeted | Any setting, Relapsed / refractory | 3 | 536 | MMR: 47.2% vs 36.9%Milestone: Bosutinib: from a missed first-line endpoint (BELA) to BFORE |
| ca209-003 | Melanoma | 2012 | Immunotherapy | Metastatic | 1 | 296 | ORR: see trialMilestone: Anti-PD-1 in melanoma: phase 1 results to FDA approvals (2012-2014) |
| CALGB 100104 | Multiple myeloma | 2012 | Targeted, Cell therapy & transplant | Maintenance | — | 460 | TTP: median 46 vs 27 monthsMilestone: Lenalidomide maintenance after autologous transplant |
| CheckMate 037 | Melanoma | 2012 | Immunotherapy | Metastatic | 3 | 405 | ORR: 31.7% vs 10.6%Milestone: Anti-PD-1 in melanoma: phase 1 results to FDA approvals (2012-2014) |
| CNBSS | Breast cancer | 2012 | Detection & prevention, Understanding | Screening | — | 89,835 | Breast cancer mortality: HR 1.05 (95% CI 0.85–1.30)Milestone: Screening, overdiagnosis and the benefit-harm debate |
| CORRECT | Colorectal cancer | 2012 | Targeted | Metastatic, Relapsed / refractory | 3 | 760 | OS: median 6.4 vs 5 months, HR 0.77 (95% CI 0.64–0.94)Milestone: Regorafenib for previously treated metastatic CRC (CORRECT) |
| CROSS | Gastric and oesophageal cancer | 2012 | Chemotherapy, Radiation, Surgery | Neoadjuvant | — | 366 | OS: median 49.4 vs 24 months, HR 0.657 (95% CI 0.495–0.871)Milestone: CROSS: neoadjuvant chemoradiotherapy for oesophageal cancer |
| EMILIA | Breast cancer | 2012 | Antibody–drug conjugate, Targeted | Metastatic | 3 | 991 | PFS: median 9.6 vs 6.4 months, HR 0.65 (95% CI 0.55–0.77)Milestone: EMILIA: trastuzumab emtansine versus lapatinib plus capecitabine |
| ENDEAVOR | Multiple myeloma | 2012 | Targeted | Relapsed / refractory | 3 | 929 | PFS: median 18.7 vs 9.4 months, HR 0.53 (95% CI 0.44–0.65)Milestone: Carfilzomib: accelerated approval to ASPIRE and ENDEAVOR |
| ESTIMABL1 | Thyroid cancer | 2012 | Radiation, Endocrine | Adjuvant | 3 | 752 | No results recordedMilestone: HiLo and ESTIMABL1: 1.1 GBq versus 3.7 GBq radioiodine ablation |
| EXAM | Thyroid cancer | 2012 | Targeted | Metastatic | 3 | 330 | PFS: median 11.2 vs 4 months, HR 0.28 (95% CI 0.19–0.40)Milestone: EXAM and EXAMINER: cabozantinib in medullary thyroid cancer |
| EXAMINER | Thyroid cancer | 2012 | Targeted | Metastatic | — | 247 | PFS: median 11 vs 13.9 months, HR 1.24 (95% CI 0.9–1.7)Milestone: EXAM and EXAMINER: cabozantinib in medullary thyroid cancer |
| GOG-0213 | Ovarian cancer | 2012 | Targeted, Chemotherapy | Relapsed / refractory | 3 | 674 | OS: median 42.2 vs 37.3 months, HR 0.829 (95% CI 0.683–1.005)Milestone: Bevacizumab in recurrent disease: OCEANS, AURELIA, GOG-213 |
| GRID | Sarcoma and GIST | 2012 | Targeted | Relapsed / refractory | 3 | 199 | PFS: median 4.8 vs 0.9 months, HR 0.27 (95% CI 0.19–0.39)Milestone: GRID: regorafenib after imatinib and sunitinib |
| HEPATHER | Hepatocellular carcinoma | 2012 | Detection & prevention, Understanding | Prevention | observational | 9,895 | Incidence of hepatocellular carcinoma: HR 0.66 (95% CI 0.46–0.93)Milestone: Hepatitis C clearance and HCC: interferon-era SVR, then DAA debate |
| HiLo | Thyroid cancer | 2012 | Radiation, Endocrine | Adjuvant | 3 | 438 | Ablation success rate: 85% vs 88.9%Milestone: HiLo and ESTIMABL1: 1.1 GBq versus 3.7 GBq radioiodine ablation |
| IFM 2005-02 | Multiple myeloma | 2012 | Targeted, Cell therapy & transplant | Maintenance | 3 | 614 | PFS: median 41 vs 23 months, HR 0.5Milestone: Lenalidomide maintenance after autologous transplant |
| kanwal-va-daa-svr-hcc-cohort | Hepatocellular carcinoma | 2012 | Detection & prevention, Understanding | Prevention | observational | 22,500 | Incidence of hepatocellular carcinoma: HR 0.28 (95% CI 0.22–0.36)Milestone: Hepatitis C clearance and HCC: interferon-era SVR, then DAA debate |
| keynote-001-melanoma | Melanoma | 2012 | Immunotherapy | Metastatic | 1 | 135 | ORR: see trialMilestone: Anti-PD-1 in melanoma: phase 1 results to FDA approvals (2012-2014) |
| KEYNOTE-002 | Melanoma | 2012 | Immunotherapy | Metastatic | 2 | 540 | PFS: 34% vs 16% at 6 mo, HR 0.57 (95% CI 0.45–0.73)Milestone: Anti-PD-1 in melanoma: phase 1 results to FDA approvals (2012-2014) |
| lenalidomide-maintenance-meta-analysis-2017 | Multiple myeloma | 2012 | Targeted, Cell therapy & transplant | Maintenance | meta-analysis | 1,208 | PFS: median 52.8 vs 23.5 months, HR 0.48 (95% CI 0.41–0.55)Milestone: Lenalidomide maintenance after autologous transplant |
| OCEANS | Ovarian cancer | 2012 | Targeted, Chemotherapy | Relapsed / refractory | 3 | 484 | PFS: median 12.4 vs 8.4 months, HR 0.484 (95% CI 0.388–0.605)Milestone: Bevacizumab in recurrent disease: OCEANS, AURELIA, GOG-213 |
| OPTIC | Chronic myeloid leukemia | 2012 | Targeted | Relapsed / refractory | 2 | 283 | BCR-ABL1 ≤1% (IS): see trialMilestone: Ponatinib: activity against T315I (PACE) |
| PACE | Chronic myeloid leukemia | 2012 | Targeted | Relapsed / refractory | 2 | 449 | MCyR: see trialMilestone: Ponatinib: activity against T315I (PACE) |
| PALETTE | Sarcoma and GIST | 2012 | Targeted | Relapsed / refractory | 3 | 369 | PFS: median 4.6 vs 1.6 months, HR 0.31 (95% CI 0.24–0.40)Milestone: PALETTE: pazopanib after chemotherapy in non-adipocytic sarcoma |
| PX-171-003-A1 | Multiple myeloma | 2012 | Targeted | Relapsed / refractory | 2 | — | ORR: see trialMilestone: Carfilzomib: accelerated approval to ASPIRE and ENDEAVOR |
| reig-daa-prior-hcc-recurrence-cohort | Hepatocellular carcinoma | 2012 | Detection & prevention, Understanding | Prevention | observational | 58 | Radiological tumour recurrence: see trialMilestone: Hepatitis C clearance and HCC: interferon-era SVR, then DAA debate |
| SOLO2/ENGOT-Ov21 | Ovarian cancer | 2012 | Targeted | Maintenance, Relapsed / refractory | 3 | 295 | PFS: median 19.1 vs 5.5 months, HR 0.30 (95% CI 0.22–0.41)Milestone: Olaparib maintenance in platinum-sensitive relapse: Study 19 and SOLO2 |
| Study 19 | Ovarian cancer | 2012 | Targeted | Maintenance, Relapsed / refractory | 2 | 265 | PFS: median 8.4 vs 4.8 months, HR 0.35 (95% CI 0.25–0.49)Milestone: Olaparib maintenance in platinum-sensitive relapse: Study 19 and SOLO2 |
| surlemont-2021-bcg-shortage-france | Bladder cancer | 2012 | Immunotherapy, Supportive care | Localised | observational | — | Incomplete BCG induction: 20.8% vs 5.3%Milestone: BCG shortages from about 2012 limit intravesical therapy |
| vandermeer-svr-fibrosis-cohort | Hepatocellular carcinoma | 2012 | Detection & prevention, Understanding | Prevention | observational | 530 | All-cause mortality: 8.9% vs 26% at 120 mo, HR 0.26 (95% CI 0.14–0.49)Milestone: Hepatitis C clearance and HCC: interferon-era SVR, then DAA debate |
| ALSYMPCA | Prostate cancer | 2013 | Radiation | Metastatic | 3 | 921 | OS: median 14 vs 11.2 months, HR 0.70 (95% CI 0.55–0.88)Milestone: Radium-223 and survival in bone-metastatic CRPC (ALSYMPCA) |
| AML17 | Acute myeloid leukaemia (including APL) | 2013 | Targeted | Any setting | 3 | 235 | No results recordedMilestone: APL0406: ATRA plus arsenic trioxide versus ATRA plus chemotherapy |
| APL0406 | Acute myeloid leukaemia (including APL) | 2013 | Targeted | Any setting | 3 | 276 | Complete remission rate: 100% vs 95%Milestone: APL0406: ATRA plus arsenic trioxide versus ATRA plus chemotherapy |
| AQUILA | Multiple myeloma | 2013 | Immunotherapy, Targeted | Any setting | 3 | 390 | PFS: 63.1% vs 40.8% at 60 mo, HR 0.49 (95% CI 0.36–0.67)Milestone: Treating smouldering myeloma: from lenalidomide to daratumumab |
| ATHENA | Cervical cancer | 2013 | Detection & prevention | Screening | — | — | Sensitivity: 76.1% vs 47.8%Milestone: HPV testing versus cytology in screening: RCT follow-up and FDA 2014 |
| BRIGHT | Non-Hodgkin lymphoma (DLBCL and follicular) | 2013 | Chemotherapy, Immunotherapy | Locally advanced | 3 | 447 | CR rate: 31% vs 25%Milestone: StiL: bendamustine-rituximab vs R-CHOP, first-line indolent/MCL |
| cart19-penn | Childhood acute lymphoblastic leukemia (ALL) | 2013 | Cell therapy & transplant | Relapsed / refractory | — | — | Complete remission rate: see trialMilestone: CD19 CAR T cells induce remissions in children with relapsed ALL |
| CLL11 | Chronic lymphocytic leukaemia | 2013 | Immunotherapy, Chemotherapy | Metastatic | 3 | 781 | PFS: median 26.7 vs 11.1 months, HR 0.18 (95% CI 0.13–0.24)Milestone: CLL11: anti-CD20 plus chlorambucil in patients with comorbidities |
| DECISION | Thyroid cancer | 2013 | Targeted | Metastatic | 3 | 417 | PFS: median 10.8 vs 5.8 months, HR 0.59 (95% CI 0.45–0.76)Milestone: DECISION: sorafenib for radioiodine-refractory thyroid cancer |
| E3A06 | Multiple myeloma | 2013 | Immunotherapy, Targeted | Any setting | 3 | 182 | PFS: 98% vs 89% at 12 mo, HR 0.28 (95% CI 0.12–0.62)Milestone: Treating smouldering myeloma: from lenalidomide to daratumumab |
| HARMONY | Acute myeloid leukaemia (including APL) | 2013 | Targeted | Any setting | observational | 1,438 | OS: 91% vs 81% at 84 moMilestone: APL0406: ATRA plus arsenic trioxide versus ATRA plus chemotherapy |
| HPV FOCAL | Cervical cancer | 2013 | Detection & prevention | Screening | — | 19,009 | No results recordedMilestone: HPV testing versus cytology in screening: RCT follow-up and FDA 2014 |
| LNH03-6B | Non-Hodgkin lymphoma (DLBCL and follicular) | 2013 | Immunotherapy, Chemotherapy | Locally advanced | 3 | 602 | EFS: 56% vs 60% at 36 mo, HR 1.04 (95% CI 0.82–1.31)Milestone: R-CHOP-14 not better than R-CHOP-21 (two randomised trials, 2013) |
| MPACT | Pancreatic cancer | 2013 | Chemotherapy | Metastatic | 3 | 861 | OS: median 8.5 vs 6.7 months, HR 0.72 (95% CI 0.62–0.83)Milestone: MPACT: nab-paclitaxel plus gemcitabine improves survival |
| ncri-r-chop-14-vs-21 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2013 | Immunotherapy, Chemotherapy | Locally advanced | 3 | 1,080 | OS: 82.7% vs 80.8% at 24 mo, HR 0.90 (95% CI 0.70–1.15)Milestone: R-CHOP-14 not better than R-CHOP-21 (two randomised trials, 2013) |
| pcyc-1102-ibrutinib | Chronic lymphocytic leukaemia | 2013 | Targeted | Relapsed / refractory | 1/2 | 85 | ORR: see trialMilestone: Ibrutinib: BTK inhibition for relapsed CLL, approved in 2014 |
| POBASCAM | Cervical cancer | 2013 | Detection & prevention | Screening | — | — | No results recordedMilestone: HPV testing versus cytology in screening: RCT follow-up and FDA 2014 |
| QUIREDEX | Multiple myeloma | 2013 | Immunotherapy, Targeted | Any setting | 3 | 119 | TTP: HR 0.18 (95% CI 0.09–0.32)Milestone: Treating smouldering myeloma: from lenalidomide to daratumumab |
| RAINBOW | Gastric and oesophageal cancer | 2013 | Targeted | Metastatic | 3 | 665 | OS: median 9.6 vs 7.4 months, HR 0.807 (95% CI 0.678–0.962)Milestone: REGARD and RAINBOW: ramucirumab after first-line therapy |
| REGARD | Gastric and oesophageal cancer | 2013 | Targeted | Metastatic | 3 | 355 | OS: median 5.2 vs 3.8 months, HR 0.776 (95% CI 0.603–0.998)Milestone: REGARD and RAINBOW: ramucirumab after first-line therapy |
| RESONATE | Chronic lymphocytic leukaemia | 2013 | Targeted | Relapsed / refractory | 3 | 391 | PFS: HR 0.22Milestone: Ibrutinib: BTK inhibition for relapsed CLL, approved in 2014 |
| RTOG 0525 | Glioblastoma | 2013 | Chemotherapy | Maintenance | 3 | 833 | OS: median 14.9 vs 16.6 months, HR 1.03Milestone: Dose-dense temozolomide shows no survival gain (RTOG 0525) |
| StiL NHL1 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2013 | Chemotherapy, Immunotherapy | Locally advanced | 3 | 549 | PFS: median 69.5 vs 31.2 months, HR 0.58 (95% CI 0.44–0.74)Milestone: StiL: bendamustine-rituximab vs R-CHOP, first-line indolent/MCL |
| Swedescreen, POBASCAM, ARTISTIC, NTCC (pooled follow-up)⚑ needs review | Cervical cancer | 2013 | Detection & prevention | Screening | — | 176,464 | No results recordedMilestone: HPV testing versus cytology in screening: RCT follow-up and FDA 2014 |
| UKALL 2003⚑ needs review | Childhood acute lymphoblastic leukemia (ALL) | 2013 | Chemotherapy, Understanding | Any setting | — | 521 | EFS: 94.4% vs 95.5% at 60 moMilestone: MRD-guided treatment reduction for low-risk ALL (UKALL 2003) |
| ACT IV | Glioblastoma | 2014 | Targeted, Immunotherapy | Adjuvant | 3 | 745 | OS: median 20.1 vs 20 months, HR 1.01 (95% CI 0.79–1.30)Milestone: Three further add-on agents fail in randomised phase 3 trials |
| ALKA-372-001, STARTRK-1 and STARTRK-2 (integrated) | Lung cancer | 2014 | Targeted | Metastatic | 1/2 | 53 | ORR: see trialMilestone: ROS1-positive NSCLC: crizotinib to taletrectinib |
| AVAglio | Glioblastoma | 2014 | Targeted | Adjuvant | 3 | — | PFS: median 10.6 vs 6.2 months, HR 0.64 (95% CI 0.55–0.74)Milestone: Bevacizumab added to chemoradiation does not improve survival |
| CENTRIC | Glioblastoma | 2014 | Targeted, Immunotherapy | Adjuvant | 3 | 545 | OS: median 26.3 vs 26.3 months, HR 1.02 (95% CI 0.81–1.29)Milestone: Three further add-on agents fail in randomised phase 3 trials |
| CheckMate 039 | Hodgkin lymphoma | 2014 | Immunotherapy | Relapsed / refractory | 1 | 23 | ORR: see trialMilestone: PD-1 blockade for relapsed Hodgkin lymphoma |
| CheckMate 066 | Melanoma | 2014 | Immunotherapy | Metastatic | 3 | 418 | OS: 72.9% vs 42.1% at 12 mo, HR 0.42Milestone: Nivolumab monotherapy: CheckMate 066 and CheckMate 037 (2014-2015) |
| CheckMate 205 | Hodgkin lymphoma | 2014 | Immunotherapy | Relapsed / refractory | 2 | 243 | ORR: see trialMilestone: PD-1 blockade for relapsed Hodgkin lymphoma |
| COMBI-d | Melanoma | 2014 | Targeted | Metastatic | 3 | 423 | PFS: median 9.3 vs 8.8 months, HR 0.75 (95% CI 0.57–0.99)Milestone: Dabrafenib plus trametinib: COMBI-d and COMBI-v (2014) |
| COMBI-v | Melanoma | 2014 | Targeted | Metastatic | 3 | 704 | OS: 72% vs 65% at 12 mo, HR 0.69 (95% CI 0.53–0.89)Milestone: Dabrafenib plus trametinib: COMBI-d and COMBI-v (2014) |
| EORTC 62012 | Sarcoma and GIST | 2014 | Chemotherapy | Metastatic | 3 | 455 | OS: median 14.3 vs 12.8 months, HR 0.83Milestone: EORTC 62012: doxorubicin vs doxorubicin plus ifosfamide |
| FIRST | Multiple myeloma | 2014 | Targeted | Any setting | 3 | 1,623 | PFS: median 25.5 vs 21.2 months, HR 0.72Milestone: FIRST and SWOG S0777: lenalidomide-based first-line treatment |
| GETUG 13 | Testicular germ cell tumors | 2014 | Chemotherapy | Metastatic | 3 | 263 | PFS: 59% vs 48% at 36 mo, HR 0.66 (95% CI 0.44–1.00)Milestone: GETUG 13: tumour-marker decline guides dose-dense chemotherapy |
| GOG-240 | Cervical cancer | 2014 | Targeted, Chemotherapy | Metastatic | 3 | 452 | OS: median 17 vs 13.3 months, HR 0.71Milestone: GOG-240: bevacizumab added to chemotherapy (FDA 2014) |
| H10 | Hodgkin lymphoma | 2014 | Radiation, Understanding | Localised | 3 | 1,950 | PFS: 90.6% vs 77.4% at 60 mo, HR 0.42 (95% CI 0.23–0.74)Milestone: EORTC H10: PET-guided omission of radiotherapy tested |
| KEYNOTE-087 | Hodgkin lymphoma | 2014 | Immunotherapy | Relapsed / refractory | 2 | 210 | ORR: see trialMilestone: PD-1 blockade for relapsed Hodgkin lymphoma |
| MIRAGE | Glioblastoma | 2014 | Targeted, Immunotherapy | Adjuvant | 3 | 749 | OS: median 16.5 vs 17 months, HR 1.04Milestone: Three further add-on agents fail in randomised phase 3 trials |
| PROFILE 1001 (ROS1 cohort) | Lung cancer | 2014 | Targeted | Metastatic | 1 | 50 | ORR: see trialMilestone: ROS1-positive NSCLC: crizotinib to taletrectinib |
| RTOG 0825 | Glioblastoma | 2014 | Targeted | Adjuvant | 3 | 637 | OS: median 15.7 vs 16.1 months, HR 1.13Milestone: Bevacizumab added to chemoradiation does not improve survival |
| SWOG S0777 | Multiple myeloma | 2014 | Targeted | Any setting | 3 | 525 | PFS: median 43 vs 30 months, HR 0.712Milestone: FIRST and SWOG S0777: lenalidomide-based first-line treatment |
| TRIDENT-1 | Lung cancer | 2014 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: ROS1-positive NSCLC: crizotinib to taletrectinib |
| TRUST-I and TRUST-II (pooled) | Lung cancer | 2014 | Targeted | Metastatic | 2 | 273 | ORR: see trialMilestone: ROS1-positive NSCLC: crizotinib to taletrectinib |
| A041202 | Chronic lymphocytic leukaemia | 2015 | Targeted | Metastatic | 3 | 547 | PFS: 87% vs 74% at 24 mo, HR 0.39 (95% CI 0.26–0.58)Milestone: Ibrutinib as first-line therapy: RESONATE-2, A041202, E1912 |
| AETHERA | Hodgkin lymphoma | 2015 | Antibody–drug conjugate | Relapsed / refractory, Maintenance | 3 | 329 | PFS: median 42.9 vs 24.1 months, HR 0.57 (95% CI 0.40–0.81)Milestone: AETHERA: brentuximab vedotin consolidation after ASCT |
| CABOSUN | Kidney cancer (renal cell carcinoma) | 2015 | Targeted | Metastatic | 2 | 157 | PFS: median 8.2 vs 5.6 months, HR 0.66 (95% CI 0.46–0.95)Milestone: Cabozantinib: METEOR (after VEGF therapy) and CABOSUN (first line) |
| CASTOR | Multiple myeloma | 2015 | Immunotherapy | Relapsed / refractory | 3 | 498 | PFS: 60.7% vs 26.9% at 12 mo, HR 0.39 (95% CI 0.28–0.53)Milestone: Daratumumab: anti-CD38 antibody from single agent to CASTOR/POLLUX |
| CHAARTED | Prostate cancer | 2015 | Chemotherapy, Endocrine | Metastatic | 3 | 790 | OS: median 57.6 vs 44 months, HR 0.61 (95% CI 0.47–0.80)Milestone: Docetaxel with ADT in hormone-sensitive disease (CHAARTED, STAMPEDE) |
| CheckMate 017 | Lung cancer | 2015 | Immunotherapy | Metastatic | 3 | 272 | OS: median 9.2 vs 6 months, HR 0.59 (95% CI 0.44–0.79)Milestone: PD-(L)1 blockade after chemotherapy: CheckMate, KEYNOTE-010, OAK |
| CheckMate 025 | Kidney cancer (renal cell carcinoma) | 2015 | Immunotherapy | Metastatic | 3 | 821 | OS: median 25 vs 19.6 months, HR 0.73Milestone: CheckMate 025: nivolumab versus everolimus in treated RCC (2015) |
| CheckMate 057 | Lung cancer | 2015 | Immunotherapy | Metastatic | 3 | 582 | OS: median 12.2 vs 9.4 months, HR 0.73Milestone: PD-(L)1 blockade after chemotherapy: CheckMate, KEYNOTE-010, OAK |
| CheckMate 067 | Melanoma | 2015 | Immunotherapy | Metastatic | 3 | 945 | PFS: median 11.5 vs 2.9 months, HR 0.42Milestone: Nivolumab plus ipilimumab: CheckMate 069/067 and the 10-year follow-up |
| CheckMate 069 | Melanoma | 2015 | Immunotherapy | Metastatic | 2 | 142 | PFS: HR 0.40 (95% CI 0.23–0.68)Milestone: Nivolumab plus ipilimumab: CheckMate 069/067 and the 10-year follow-up |
| CheckMate 142 | Colorectal cancer | 2015 | Immunotherapy | Metastatic | 2 | — | ORR: see trialMilestone: PD-1 blockade in mismatch repair-deficient colorectal cancer |
| E1912 | Chronic lymphocytic leukaemia | 2015 | Targeted | Metastatic | 3 | 529 | PFS: 89.4% vs 72.9% at 36 mo, HR 0.35 (95% CI 0.22–0.56)Milestone: Ibrutinib as first-line therapy: RESONATE-2, A041202, E1912 |
| EF-14 | Glioblastoma | 2015 | Targeted | Maintenance | 3 | 695 | PFS: median 7.1 vs 4 months, HR 0.62Milestone: Tumour treating fields added to maintenance temozolomide (EF-14) |
| EORTC 18071 | Melanoma | 2015 | Immunotherapy | Adjuvant | 3 | 951 | RFS: median 26.1 vs 17.1 months, HR 0.75 (95% CI 0.64–0.90)Milestone: Adjuvant ipilimumab 10 mg/kg: EORTC 18071 and FDA approval (2015) |
| ET743-SAR-3007 | Sarcoma and GIST | 2015 | Chemotherapy | Relapsed / refractory | 3 | 518 | OS: median 12.4 vs 12.9 months, HR 0.87Milestone: Trabectedin vs dacarbazine in liposarcoma and leiomyosarcoma |
| FLAIR | Chronic lymphocytic leukaemia | 2015 | Targeted | Metastatic | 3 | 771 | PFS: HR 0.44 (95% CI 0.32–0.60)Milestone: Ibrutinib as first-line therapy: RESONATE-2, A041202, E1912 |
| GEN501 | Multiple myeloma | 2015 | Immunotherapy | Relapsed / refractory | 1/2 | — | ORR: see trialMilestone: Daratumumab: anti-CD38 antibody from single agent to CASTOR/POLLUX |
| GETUG-AFU 15 | Prostate cancer | 2015 | Chemotherapy, Endocrine | Metastatic | 3 | 385 | OS: median 58.9 vs 54.2 months, HR 1.01 (95% CI 0.75–1.36)Milestone: Docetaxel with ADT in hormone-sensitive disease (CHAARTED, STAMPEDE) |
| KEYNOTE-006 | Melanoma | 2015 | Immunotherapy | Metastatic | 3 | 834 | PFS: 47.3% vs 26.5% at 6 mo, HR 0.58 (95% CI 0.46–0.72)Milestone: KEYNOTE-006: pembrolizumab versus ipilimumab (2015, 10-year follow-up) |
| KEYNOTE-010 | Lung cancer | 2015 | Immunotherapy | Metastatic | 2/3 | 1,034 | OS: median 10.4 vs 8.5 months, HR 0.71 (95% CI 0.58–0.88)Milestone: PD-(L)1 blockade after chemotherapy: CheckMate, KEYNOTE-010, OAK |
| KEYNOTE-016 | Colorectal cancer | 2015 | Immunotherapy | Metastatic | 2 | 41 | PFS: median 2.2 months, HR 0.1Milestone: PD-1 blockade in mismatch repair-deficient colorectal cancer |
| METEOR | Kidney cancer (renal cell carcinoma) | 2015 | Targeted | Metastatic | 3 | 658 | PFS: median 7.4 vs 3.8 months, HR 0.58 (95% CI 0.45–0.75)Milestone: Cabozantinib: METEOR (after VEGF therapy) and CABOSUN (first line) |
| MONALEESA-2 | Breast cancer | 2015 | Targeted, Endocrine | Metastatic | 3 | 668 | PFS: 63% vs 42.2% at 18 mo, HR 0.56 (95% CI 0.43–0.72)Milestone: CDK4/6 inhibitors added to first-line endocrine therapy |
| MONARCH 3⚑ needs review | Breast cancer | 2015 | Targeted, Endocrine | Metastatic | 3 | 493 | PFS: HR 0.54 (95% CI 0.41–0.72)Milestone: CDK4/6 inhibitors added to first-line endocrine therapy |
| MURANO | Chronic lymphocytic leukaemia | 2015 | Targeted | Relapsed / refractory | 3 | 389 | PFS: 84.9% vs 36.3% at 24 mo, HR 0.17 (95% CI 0.11–0.25)Milestone: Venetoclax: BCL2 inhibition, from phase 1 to MURANO |
| OAK | Lung cancer | 2015 | Immunotherapy | Metastatic | 3 | 1,225 | OS: median 13.8 vs 9.6 months, HR 0.73 (95% CI 0.62–0.87)Milestone: PD-(L)1 blockade after chemotherapy: CheckMate, KEYNOTE-010, OAK |
| OPTiM | Melanoma | 2015 | Immunotherapy | Metastatic, Locally advanced | 3 | 436 | OS: median 23.3 vs 18.9 months, HR 0.79 (95% CI 0.62–1.00)Milestone: Talimogene laherparepvec (T-VEC): OPTiM trial and approval (2015) |
| PALOMA-2 | Breast cancer | 2015 | Targeted, Endocrine | Metastatic | 3 | 666 | PFS: median 24.8 vs 14.5 months, HR 0.58 (95% CI 0.46–0.72)Milestone: CDK4/6 inhibitors added to first-line endocrine therapy |
| POLLUX | Multiple myeloma | 2015 | Immunotherapy | Relapsed / refractory | 3 | 569 | PFS: 83.2% vs 60.1% at 12 mo, HR 0.37 (95% CI 0.27–0.52)Milestone: Daratumumab: anti-CD38 antibody from single agent to CASTOR/POLLUX |
| PORTEC-3 | Endometrial cancer | 2015 | Understanding, Chemotherapy, Radiation | Adjuvant | 3 | 686 | OS: 81.8% vs 76.7% at 60 mo, HR 0.76 (95% CI 0.54–1.06)Milestone: ProMisE and PORTEC-3: molecular classes in high-risk disease |
| PRIME II | Breast cancer | 2015 | Radiation, Surgery | Adjuvant | 3 | 1,326 | IBTR: 4.1% vs 1.3%, HR 5.19 (95% CI 1.99–13.52)Milestone: PRIME II: omitting radiotherapy in low-risk older women |
| RAPID | Hodgkin lymphoma | 2015 | Radiation, Understanding | Localised | 3 | 602 | PFS: 90.8% vs 94.6% at 36 moMilestone: RAPID: omitting radiotherapy after a negative PET |
| RECOURSE | Colorectal cancer | 2015 | Chemotherapy, Targeted | Metastatic, Relapsed / refractory | 3 | 800 | OS: median 7.1 vs 5.3 months, HR 0.68 (95% CI 0.58–0.81)Milestone: Trifluridine-tipiracil (TAS-102): RECOURSE and SUNLIGHT |
| RESONATE-2 | Chronic lymphocytic leukaemia | 2015 | Targeted | Metastatic | 3 | 269 | PFS: HR 0.16Milestone: Ibrutinib as first-line therapy: RESONATE-2, A041202, E1912 |
| sasaki-2015-mdacc-cohort | Chronic myeloid leukemia | 2015 | Understanding | Any setting | observational | 483 | 5-year relative survival: see trialMilestone: Life expectancy in CML approaches that of the general population |
| SELECT | Thyroid cancer | 2015 | Targeted | Metastatic | 3 | 392 | PFS: median 18.3 vs 3.6 months, HR 0.21Milestone: SELECT: lenvatinib for radioiodine-refractory thyroid cancer |
| SIOP WT 2001 | Neuroblastoma and Wilms tumour | 2015 | Chemotherapy | Adjuvant | 3 | 583 | EFS: 88.2% vs 92.6% at 24 moMilestone: SIOP WT 2001: doxorubicin omitted in intermediate-risk stage II-III |
| SIRIUS | Multiple myeloma | 2015 | Immunotherapy | Relapsed / refractory | 2 | — | ORR: see trialMilestone: Daratumumab: anti-CD38 antibody from single agent to CASTOR/POLLUX |
| STAMPEDE | Prostate cancer | 2015 | Chemotherapy, Endocrine | Metastatic | 2/3 | 2,962 | OS: median 81 vs 71 months, HR 0.78 (95% CI 0.66–0.93)Milestone: Docetaxel with ADT in hormone-sensitive disease (CHAARTED, STAMPEDE) |
| STORM | Hepatocellular carcinoma | 2015 | Targeted | Adjuvant | 3 | 1,114 | RFS: median 33.3 vs 33.7 months, HR 0.940 (95% CI 0.780–1.134)Milestone: STORM: adjuvant sorafenib did not improve recurrence-free survival |
| SUNLIGHT | Colorectal cancer | 2015 | Chemotherapy, Targeted | Metastatic, Relapsed / refractory | 3 | 492 | OS: median 10.8 vs 7.5 months, HR 0.61 (95% CI 0.49–0.77)Milestone: Trifluridine-tipiracil (TAS-102): RECOURSE and SUNLIGHT |
| tata-elective-vs-therapeutic-neck-dissection | Head and neck cancer | 2015 | Surgery | Localised | — | 596 | OS: 80% vs 67.5% at 36 mo, HR 0.64 (95% CI 0.45–0.92)Milestone: Elective vs therapeutic neck dissection in early oral cancer (Tata) |
| venetoclax-del17p-m13-982 | Chronic lymphocytic leukaemia | 2015 | Targeted | Relapsed / refractory | 2 | 158 | ORR: see trialMilestone: Venetoclax: BCL2 inhibition, from phase 1 to MURANO |
| venetoclax-phase1-cll | Chronic lymphocytic leukaemia | 2015 | Targeted | Relapsed / refractory | 1 | 116 | ORR: see trialMilestone: Venetoclax: BCL2 inhibition, from phase 1 to MURANO |
| AALL0232 | Childhood acute lymphoblastic leukemia (ALL) | 2016 | Chemotherapy | Any setting | 3 | 3,154 | EFS: 82% vs 75.4% at 60 moMilestone: Dexamethasone and high-dose methotrexate in high-risk B-ALL |
| aml17-aml19-mrd-monitoring | Acute myeloid leukaemia (including APL) | 2016 | Understanding | Any setting | 3 | 637 | OS: 70% vs 73% at 36 mo, HR 1.11 (95% CI 0.83–1.49)Milestone: Measurable residual disease in AML: prognostic value and monitoring |
| aml17-npm1-mrd | Acute myeloid leukaemia (including APL) | 2016 | Understanding | Any setting | observational | 346 | Relapse risk: 82% vs 30% at 36 mo, HR 4.80 (95% CI 2.95–7.80)Milestone: Measurable residual disease in AML: prognostic value and monitoring |
| AREN0532 | Neuroblastoma and Wilms tumour | 2016 | Chemotherapy, Radiation, Surgery | Any setting | 3 | 116 | EFS: see trialMilestone: COG AREN0532 and AREN0533: risk-adapted Wilms therapy |
| AREN0533 | Neuroblastoma and Wilms tumour | 2016 | Chemotherapy, Radiation, Surgery | Any setting | 3 | 292 | EFS: see trialMilestone: COG AREN0532 and AREN0533: risk-adapted Wilms therapy |
| ARIEL3 | Ovarian cancer | 2016 | Targeted | Maintenance, Relapsed / refractory | 3 | 564 | PFS: median 16.6 vs 5.4 months, HR 0.23 (95% CI 0.16–0.34)Milestone: Niraparib and rucaparib maintenance, relapsed disease: NOVA, ARIEL3 |
| ASSURE | Kidney cancer (renal cell carcinoma) | 2016 | Targeted | Adjuvant | 3 | 1,943 | DFS: median 5.8 vs 6.6 years, HR 1.02Milestone: Adjuvant VEGF-TKIs: ASSURE, S-TRAC, PROTECT and SORCE |
| blinatumomab-pediatric-phase1-2 | Childhood acute lymphoblastic leukemia (ALL) | 2016 | Immunotherapy | Relapsed / refractory | 1/2 | 93 | Complete remission rate: see trialMilestone: Blinatumomab for relapsed or refractory paediatric B-ALL |
| CheckMate 141 | Head and neck cancer | 2016 | Immunotherapy | Metastatic | 3 | 361 | OS: median 7.5 vs 5.1 months, HR 0.7Milestone: CheckMate 141: nivolumab versus standard therapy after platinum |
| CHHiP | Prostate cancer | 2016 | Radiation | Localised | 3 | 3,216 | BCF-free survival: 90.6% vs 88.3% at 60 mo, HR 0.84Milestone: Shorter radiotherapy: hypofractionation and SBRT (CHHiP, PACE-B) |
| DeCOG-SLT | Melanoma | 2016 | Surgery | Localised, Locally advanced | 3 | 483 | DMFS: 77% vs 74.9% at 36 moMilestone: Completion node dissection vs observation: DeCOG-SLT and MSLT-II |
| DESTINY-CRC01 | Colorectal cancer | 2016 | Targeted, Antibody–drug conjugate | Metastatic, Relapsed / refractory | 2 | 78 | ORR: see trialMilestone: HER2-targeted therapy for HER2-positive metastatic CRC |
| E1308 | Head and neck cancer | 2016 | Radiation, Surgery, Chemotherapy | Locally advanced, Adjuvant | 2 | 90 | PFS: see trialMilestone: Reduced-dose radiotherapy tested for good-risk HPV-positive oropharynx |
| E3311 | Head and neck cancer | 2016 | Radiation, Surgery, Chemotherapy | Locally advanced, Adjuvant | 2 | 495 | PFS: 96.9% vs 96% at 24 moMilestone: Reduced-dose radiotherapy tested for good-risk HPV-positive oropharynx |
| EURAMOS-1 | Sarcoma and GIST | 2016 | Chemotherapy, Immunotherapy | Adjuvant | 3 | 2,260 | EFS: HR 0.98 (95% CI 0.78–1.23)Milestone: EURAMOS-1: response-adapted escalation did not improve EFS |
| GOG-0262 | Ovarian cancer | 2016 | Chemotherapy | Locally advanced, Metastatic | 3 | 692 | PFS: median 14.7 vs 14 months, HR 0.89 (95% CI 0.74–1.06)Milestone: Weekly paclitaxel: JGOG 3016 gain not seen in GOG-262 or ICON8 |
| gp-vs-fp-npc⚑ needs review | Head and neck cancer | 2016 | Chemotherapy | Metastatic | 3 | 362 | PFS: median 7 vs 5.6 months, HR 0.55 (95% CI 0.44–0.68)Milestone: Gemcitabine-cisplatin versus fluorouracil-cisplatin in metastatic NPC |
| HERACLES | Colorectal cancer | 2016 | Targeted, Antibody–drug conjugate | Metastatic, Relapsed / refractory | 2 | 27 | ORR: see trialMilestone: HER2-targeted therapy for HER2-positive metastatic CRC |
| HYPO-RT-PC | Prostate cancer | 2016 | Radiation | Localised | 3 | 1,200 | BCF-free survival: 84% vs 84% at 60 mo, HR 1.002 (95% CI 0.758–1.325)Milestone: Shorter radiotherapy: hypofractionation and SBRT (CHHiP, PACE-B) |
| ICON8 | Ovarian cancer | 2016 | Chemotherapy | Locally advanced, Metastatic | 3 | 1,566 | PFS: median 20.8 vs 17.7 monthsMilestone: Weekly paclitaxel: JGOG 3016 gain not seen in GOG-262 or ICON8 |
| JCOG0905 | Sarcoma and GIST | 2016 | Chemotherapy, Immunotherapy | Adjuvant | — | 287 | DFS: HR 1.05 (95% CI 0.55–1.98)Milestone: EURAMOS-1: response-adapted escalation did not improve EFS |
| JGOG 3016 | Ovarian cancer | 2016 | Chemotherapy | Locally advanced, Metastatic | 3 | 637 | PFS: median 28 vs 17.2 months, HR 0.71 (95% CI 0.58–0.88)Milestone: Weekly paclitaxel: JGOG 3016 gain not seen in GOG-262 or ICON8 |
| jongen-lavrencic-2018-ngs-mrd | Acute myeloid leukaemia (including APL) | 2016 | Understanding | Any setting | observational | 482 | Relapse risk: 55.4% vs 31.9% at 48 mo, HR 2.14Milestone: Measurable residual disease in AML: prognostic value and monitoring |
| KEYNOTE-012 | Head and neck cancer | 2016 | Immunotherapy | Metastatic | 1 | — | ORR: see trialMilestone: Pembrolizumab after platinum: accelerated approval, then KEYNOTE-040 |
| KEYNOTE-021 cohort G | Lung cancer | 2016 | Immunotherapy, Chemotherapy | Metastatic | 2 | 123 | ORR: 55% vs 29%Milestone: Pembrolizumab plus chemotherapy first-line: KEYNOTE-021G, -189, -407 |
| KEYNOTE-024 | Lung cancer | 2016 | Immunotherapy | Metastatic | 3 | 305 | PFS: median 10.3 vs 6 months, HR 0.50 (95% CI 0.37–0.68)Milestone: KEYNOTE-024: first-line pembrolizumab for PD-L1 >= 50% |
| KEYNOTE-040⚑ needs review | Head and neck cancer | 2016 | Immunotherapy | Metastatic | 3 | 495 | OS: median 8.4 vs 6.9 months, HR 0.80 (95% CI 0.65–0.98)Milestone: Pembrolizumab after platinum: accelerated approval, then KEYNOTE-040 |
| KEYNOTE-189 | Lung cancer | 2016 | Immunotherapy, Chemotherapy | Metastatic | 3 | 616 | OS: 69.2% vs 49.4% at 12 mo, HR 0.49 (95% CI 0.38–0.64)Milestone: Pembrolizumab plus chemotherapy first-line: KEYNOTE-021G, -189, -407 |
| KEYNOTE-407 | Lung cancer | 2016 | Immunotherapy, Chemotherapy | Metastatic | 3 | 559 | OS: median 15.9 vs 11.3 months, HR 0.64 (95% CI 0.49–0.85)Milestone: Pembrolizumab plus chemotherapy first-line: KEYNOTE-021G, -189, -407 |
| MINDACT | Breast cancer | 2016 | Understanding, Chemotherapy | Adjuvant | 3 | 6,693 | DMFS: see trialMilestone: Genomic assays guide who can skip chemotherapy |
| MOUNTAINEER | Colorectal cancer | 2016 | Targeted, Antibody–drug conjugate | Metastatic, Relapsed / refractory | 2 | 117 | ORR: see trialMilestone: HER2-targeted therapy for HER2-positive metastatic CRC |
| MSLT-II | Melanoma | 2016 | Surgery | Localised, Locally advanced | 3 | 1,939 | MSS: 86% vs 86% at 36 moMilestone: Completion node dissection vs observation: DeCOG-SLT and MSLT-II |
| munshi-2017-mrd-meta-analysis | Multiple myeloma | 2016 | Understanding | Any setting | meta-analysis | 1,273 | PFS: HR 0.41 (95% CI 0.36–0.48)Milestone: Minimal residual disease: IMWG criteria to an ODAC endpoint vote |
| MyPathway | Colorectal cancer | 2016 | Targeted, Antibody–drug conjugate | Metastatic, Relapsed / refractory | 2 | 57 | ORR: see trialMilestone: HER2-targeted therapy for HER2-positive metastatic CRC |
| NOVA/ENGOT-OV16 | Ovarian cancer | 2016 | Targeted | Maintenance, Relapsed / refractory | 3 | 553 | PFS: median 21 vs 5.5 months, HR 0.27 (95% CI 0.17–0.41)Milestone: Niraparib and rucaparib maintenance, relapsed disease: NOVA, ARIEL3 |
| NRG-HN002 | Head and neck cancer | 2016 | Radiation, Surgery, Chemotherapy | Locally advanced, Adjuvant | 2 | 306 | PFS: see trialMilestone: Reduced-dose radiotherapy tested for good-risk HPV-positive oropharynx |
| PACE-B | Prostate cancer | 2016 | Radiation | Localised | 3 | 874 | RTOG grade 2 or worse genitourinary toxicity: 3% vs 2% at 24 moMilestone: Shorter radiotherapy: hypofractionation and SBRT (CHHiP, PACE-B) |
| PROFIT | Prostate cancer | 2016 | Radiation | Localised | — | — | BCF-free survival: 85% vs 85% at 60 mo, HR 0.96Milestone: Shorter radiotherapy: hypofractionation and SBRT (CHHiP, PACE-B) |
| ProtecT | Prostate cancer | 2016 | Surgery, Radiation | Localised | — | 1,643 | Prostate cancer mortality: 2.2% vs 3.1%Milestone: ProtecT: monitoring, surgery or radiotherapy for PSA-detected cancer |
| PROTECT | Kidney cancer (renal cell carcinoma) | 2016 | Targeted | Adjuvant | 3 | 1,538 | DFS: HR 0.86 (95% CI 0.70–1.06)Milestone: Adjuvant VEGF-TKIs: ASSURE, S-TRAC, PROTECT and SORCE |
| RATHL | Hodgkin lymphoma | 2016 | Chemotherapy, Understanding | Locally advanced, Metastatic | 3 | 1,214 | PFS: HR 1.13 (95% CI 0.81–1.57)Milestone: RATHL: dropping bleomycin after a negative interim PET |
| RESORCE | Hepatocellular carcinoma | 2016 | Targeted | Metastatic | 3 | 573 | OS: median 10.6 vs 7.8 months, HR 0.63 (95% CI 0.50–0.79)Milestone: RESORCE: regorafenib after sorafenib progression |
| RxPONDER | Breast cancer | 2016 | Understanding, Chemotherapy | Adjuvant | — | 5,083 | iDFS: 91.9% vs 91.3% at 60 mo, HR 1.02 (95% CI 0.82–1.26)Milestone: Genomic assays guide who can skip chemotherapy |
| S-TRAC | Kidney cancer (renal cell carcinoma) | 2016 | Targeted | Adjuvant | 3 | 615 | DFS: median 6.8 vs 5.6 years, HR 0.76 (95% CI 0.59–0.98)Milestone: Adjuvant VEGF-TKIs: ASSURE, S-TRAC, PROTECT and SORCE |
| short-2020-mrd-meta | Acute myeloid leukaemia (including APL) | 2016 | Understanding | Any setting | meta-analysis | 11,151 | OS: 68% vs 34% at 60 mo, HR 0.36Milestone: Measurable residual disease in AML: prognostic value and monitoring |
| SORCE | Kidney cancer (renal cell carcinoma) | 2016 | Targeted | Adjuvant | 3 | 1,711 | DFS: HR 1.01 (95% CI 0.83–1.23)Milestone: Adjuvant VEGF-TKIs: ASSURE, S-TRAC, PROTECT and SORCE |
| Study 309 | Sarcoma and GIST | 2016 | Chemotherapy | Relapsed / refractory | 3 | 452 | OS: median 13.5 vs 11.5 months, HR 0.77 (95% CI 0.62–0.95)Milestone: Eribulin vs dacarbazine in liposarcoma and leiomyosarcoma |
| TAILORx | Breast cancer | 2016 | Understanding, Chemotherapy | Adjuvant | — | 10,273 | iDFS: 83.3% vs 84.3% at 108 mo, HR 1.08 (95% CI 0.94–1.24)Milestone: Genomic assays guide who can skip chemotherapy |
| 301 | Acute myeloid leukaemia (including APL) | 2017 | Chemotherapy | Any setting | 3 | 309 | OS: median 9.56 vs 5.95 months, HR 0.69 (95% CI 0.52–0.90)Milestone: CPX-351 versus 7+3 in older adults with high-risk or secondary AML |
| AGILE | Acute myeloid leukaemia (including APL) | 2017 | Targeted | Relapsed / refractory, Any setting | 3 | 146 | EFS: 37% vs 12% at 12 mo, HR 0.33 (95% CI 0.16–0.69)Milestone: IDH1 and IDH2 inhibitors in AML: enasidenib, ivosidenib, olutasidenib |
| ALCYONE | Multiple myeloma | 2017 | Immunotherapy | Any setting | 3 | 706 | PFS: 71.6% vs 50.2% at 18 mo, HR 0.50 (95% CI 0.38–0.65)Milestone: Daratumumab first line without transplant: ALCYONE, MAIA, CEPHEUS |
| ALEX | Lung cancer | 2017 | Targeted | Metastatic | 3 | 303 | PFS: 68.4% vs 48.7% at 12 mo, HR 0.47 (95% CI 0.34–0.65)Milestone: ALEX: alectinib vs crizotinib first-line |
| ANBL1221 | Neuroblastoma and Wilms tumour | 2017 | Chemotherapy, Immunotherapy, Targeted | Relapsed / refractory | 2 | 36 | ORR: see trialMilestone: Relapsed or refractory disease: randomised phase 2 chemo-immunotherapy |
| BEACON | Neuroblastoma and Wilms tumour | 2017 | Chemotherapy, Immunotherapy, Targeted | Relapsed / refractory | 2 | 160 | PFS: HR 0.89 (95% CI 0.63–1.27)Milestone: Relapsed or refractory disease: randomised phase 2 chemo-immunotherapy |
| BEACON Immuno | Neuroblastoma and Wilms tumour | 2017 | Chemotherapy, Immunotherapy, Targeted | Relapsed / refractory | 2 | 65 | ORR: 30.2% vs 18.2%Milestone: Relapsed or refractory disease: randomised phase 2 chemo-immunotherapy |
| CE.6 | Glioblastoma | 2017 | Radiation, Chemotherapy | Adjuvant | 3 | 562 | OS: median 9.3 vs 7.6 months, HR 0.67 (95% CI 0.56–0.80)Milestone: Short-course radiotherapy plus temozolomide in older people |
| CEPHEUS | Multiple myeloma | 2017 | Immunotherapy | Any setting | 3 | 395 | PFS: HR 0.57 (95% CI 0.41–0.79)Milestone: Daratumumab first line without transplant: ALCYONE, MAIA, CEPHEUS |
| CheckMate 040 | Hepatocellular carcinoma | 2017 | Immunotherapy | Metastatic | 1/2 | 262 | ORR: see trialMilestone: Nivolumab after sorafenib: CheckMate 040 and accelerated approval |
| CheckMate 238⚑ needs review | Melanoma | 2017 | Immunotherapy | Adjuvant | 3 | 906 | RFS: 70.5% vs 60.8% at 12 mo, HR 0.65Milestone: Adjuvant nivolumab versus ipilimumab: CheckMate 238 (2017) |
| COMBI-AD | Melanoma | 2017 | Targeted | Adjuvant | 3 | 870 | RFS: 58% vs 39% at 36 mo, HR 0.47 (95% CI 0.39–0.58)Milestone: Adjuvant dabrafenib plus trametinib: COMBI-AD (2017) |
| DASFREE | Chronic myeloid leukemia | 2017 | Targeted | Maintenance | 2 | 84 | TFR: see trialMilestone: Stopping TKIs: EURO-SKI, ENESTfreedom, ENESTop, DASFREE, label |
| DESTINY | Chronic myeloid leukemia | 2017 | Targeted | Maintenance | 2 | 174 | Molecular recurrence: HR 0.12Milestone: DESTINY: reducing the TKI dose before stopping |
| DETERMINATION | Multiple myeloma | 2017 | Cell therapy & transplant, Targeted | Any setting | 3 | — | PFS: median 67.5 vs 46.2 monthsMilestone: Transplant with RVD or RVD alone: IFM 2009 and DETERMINATION |
| ECHELON-1 | Hodgkin lymphoma | 2017 | Antibody–drug conjugate, Chemotherapy | Locally advanced, Metastatic | 3 | 1,334 | mPFS: 82.1% vs 77.2% at 24 mo, HR 0.77 (95% CI 0.60–0.98)Milestone: ECHELON-1: brentuximab vedotin plus AVD vs ABVD |
| ELIANA | Childhood acute lymphoblastic leukemia (ALL) | 2017 | Cell therapy & transplant | Relapsed / refractory, Any setting | 2 | — | Overall remission rate: see trialMilestone: FDA approves tisagenlecleucel for relapsed or refractory B-ALL |
| enasidenib-idh2-phase-1-2 | Acute myeloid leukaemia (including APL) | 2017 | Targeted | Relapsed / refractory, Any setting | 1/2 | — | ORR: see trialMilestone: IDH1 and IDH2 inhibitors in AML: enasidenib, ivosidenib, olutasidenib |
| ENESTfreedom | Chronic myeloid leukemia | 2017 | Targeted | Maintenance | 2 | 215 | TFR: see trialMilestone: Stopping TKIs: EURO-SKI, ENESTfreedom, ENESTop, DASFREE, label |
| ENESTop | Chronic myeloid leukemia | 2017 | Targeted | Maintenance | 2 | — | TFR: see trialMilestone: Stopping TKIs: EURO-SKI, ENESTfreedom, ENESTop, DASFREE, label |
| EORTC 26101 | Glioblastoma | 2017 | Targeted, Chemotherapy | Relapsed / refractory | 3 | 437 | OS: median 9.1 vs 8.6 months, HR 0.95 (95% CI 0.74–1.21)Milestone: Adding bevacizumab to lomustine does not extend survival (EORTC 26101) |
| ESPAC-4 | Pancreatic cancer | 2017 | Chemotherapy | Adjuvant | 3 | 732 | OS: median 28 vs 25.5 months, HR 0.82 (95% CI 0.68–0.98)Milestone: ESPAC-4: gemcitabine plus capecitabine as adjuvant therapy |
| EURO-SKI | Chronic myeloid leukemia | 2017 | Targeted | Maintenance | — | 758 | MRFS: see trialMilestone: Stopping TKIs: EURO-SKI, ENESTfreedom, ENESTop, DASFREE, label |
| FLAURA | Lung cancer | 2017 | Targeted | Metastatic | 3 | 556 | PFS: median 18.9 vs 10.2 months, HR 0.46 (95% CI 0.37–0.57)Milestone: FLAURA: osimertinib versus gefitinib or erlotinib, first line |
| GALLIUM | Non-Hodgkin lymphoma (DLBCL and follicular) | 2017 | Immunotherapy, Chemotherapy | Locally advanced | 3 | 1,202 | PFS: 80% vs 73.3% at 36 mo, HR 0.66 (95% CI 0.51–0.85)Milestone: GALLIUM: obinutuzumab vs rituximab plus chemotherapy in FL (2017) |
| Göteborg-2⚑ needs review | Prostate cancer | 2017 | Detection & prevention | Screening, Localised | — | — | Cumulative incidence of prostate cancer: 2.8% vs 4.5%Milestone: MRI before biopsy: PROMIS and PRECISION |
| HD18 | Hodgkin lymphoma | 2017 | Chemotherapy, Understanding | Locally advanced, Metastatic | 3 | 2,101 | PFS: 92.2% vs 90.8% at 60 moMilestone: HD18: PET-2-guided cycles of escalated BEACOPP |
| hong-kong-ebv-dna-npc-screening⚑ needs review | Head and neck cancer | 2017 | Detection & prevention | Screening | observational | 20,174 | Stage I or II at diagnosis: 71% vs 20%Milestone: Plasma EBV DNA tested as a screening test for nasopharyngeal cancer |
| HR-NBL1/SIOPEN | Neuroblastoma and Wilms tumour | 2017 | Immunotherapy | Maintenance | 3 | 406 | EFS: 60% vs 56% at 36 moMilestone: Dinutuximab beta in Europe: authorisation and the IL-2 question |
| IFM 2009 | Multiple myeloma | 2017 | Cell therapy & transplant, Targeted | Any setting | 3 | 700 | PFS: median 50 vs 36 months, HR 0.65Milestone: Transplant with RVD or RVD alone: IFM 2009 and DETERMINATION |
| ivosidenib-idh1-phase-1 | Acute myeloid leukaemia (including APL) | 2017 | Targeted | Relapsed / refractory, Any setting | 1 | 258 | CR+CRh: see trialMilestone: IDH1 and IDH2 inhibitors in AML: enasidenib, ivosidenib, olutasidenib |
| JULIET | Non-Hodgkin lymphoma (DLBCL and follicular) | 2017 | Cell therapy & transplant | Relapsed / refractory | 2 | 93 | ORR: see trialMilestone: ZUMA-1 and other CD19 CAR T trials in large B-cell lymphoma |
| KEYNOTE-045 | Bladder cancer | 2017 | Immunotherapy | Metastatic | 3 | 542 | OS: median 10.3 vs 7.4 months, HR 0.73 (95% CI 0.59–0.91)Milestone: KEYNOTE-045: pembrolizumab after platinum in advanced UC |
| KEYNOTE-052 | Bladder cancer | 2017 | Immunotherapy | Metastatic | 2 | 374 | ORR: see trialMilestone: KEYNOTE-045: pembrolizumab after platinum in advanced UC |
| KEYNOTE-158 | Pancreatic cancer | 2017 | Immunotherapy | Metastatic, Relapsed / refractory | 2 | 233 | ORR: see trialMilestone: MSI-H/dMMR tumours: pancreatic cases within tissue-agnostic evidence |
| LATITUDE | Prostate cancer | 2017 | Endocrine | Metastatic | 3 | 1,199 | OS: HR 0.62 (95% CI 0.51–0.76)Milestone: Abiraterone with ADT in hormone-sensitive disease (LATITUDE, STAMPEDE) |
| MAIA | Multiple myeloma | 2017 | Immunotherapy | Any setting | 3 | 737 | PFS: 70.6% vs 55.6% at 30 mo, HR 0.56 (95% CI 0.43–0.73)Milestone: Daratumumab first line without transplant: ALCYONE, MAIA, CEPHEUS |
| olutasidenib-idh1-phase-1-2 | Acute myeloid leukaemia (including APL) | 2017 | Targeted | Relapsed / refractory, Any setting | 1/2 | 153 | CR+CRh: see trialMilestone: IDH1 and IDH2 inhibitors in AML: enasidenib, ivosidenib, olutasidenib |
| PACIFIC | Lung cancer | 2017 | Immunotherapy | Locally advanced | 3 | 713 | PFS: median 16.8 vs 5.6 months, HR 0.52 (95% CI 0.42–0.65)Milestone: PACIFIC: durvalumab after chemoradiotherapy, stage III |
| PRECISION | Prostate cancer | 2017 | Detection & prevention | Screening, Localised | — | 500 | Detection of clinically significant prostate cancer: 38% vs 26%Milestone: MRI before biopsy: PROMIS and PRECISION |
| PROMIS | Prostate cancer | 2017 | Detection & prevention | Screening, Localised | — | 740 | Diagnostic sensitivity: 93% vs 48%Milestone: MRI before biopsy: PROMIS and PRECISION |
| RATIFY | Acute myeloid leukaemia (including APL) | 2017 | Targeted | Any setting | 3 | 717 | OS: median 74.7 vs 25.6 months, HR 0.78 (95% CI 0.63–0.96)Milestone: RATIFY: midostaurin added to chemotherapy in FLT3-mutated AML |
| ROAR | Thyroid cancer | 2017 | Targeted | Locally advanced, Metastatic | 2 | 36 | ORR: see trialMilestone: ROAR: dabrafenib plus trametinib for BRAF V600E anaplastic cancer |
| SARAH | Hepatocellular carcinoma | 2017 | Radiation, Targeted | Locally advanced | 3 | 467 | OS: median 8 vs 9.9 months, HR 1.15 (95% CI 0.94–1.41)Milestone: SARAH and SIRveNIB: no survival gain over sorafenib |
| SCHOLAR-1⚑ needs review | Non-Hodgkin lymphoma (DLBCL and follicular) | 2017 | Chemotherapy | Relapsed / refractory | observational | 636 | ORR: see trialMilestone: SCHOLAR-1: outcomes in refractory DLBCL (2017) |
| SIRveNIB | Hepatocellular carcinoma | 2017 | Radiation, Targeted | Locally advanced | 3 | 360 | OS: median 8.8 vs 10 months, HR 1.1 (95% CI 0.9–1.4)Milestone: SARAH and SIRveNIB: no survival gain over sorafenib |
| STAMPEDE | Prostate cancer | 2017 | Endocrine | Metastatic | 2/3 | 1,917 | OS: HR 0.63 (95% CI 0.52–0.76)Milestone: Abiraterone with ADT in hormone-sensitive disease (LATITUDE, STAMPEDE) |
| STHLM3-MRI | Prostate cancer | 2017 | Detection & prevention | Screening, Localised | — | 1,532 | Detection of clinically significant prostate cancer: 21% vs 18%Milestone: MRI before biopsy: PROMIS and PRECISION |
| TRANSCEND NHL 001 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2017 | Cell therapy & transplant | Relapsed / refractory | 1 | 269 | ORR: see trialMilestone: ZUMA-1 and other CD19 CAR T trials in large B-cell lymphoma |
| ZUMA-1 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2017 | Cell therapy & transplant | Relapsed / refractory | 1/2 | 111 | ORR: see trialMilestone: ZUMA-1 and other CD19 CAR T trials in large B-cell lymphoma |
| AALL0622 | Childhood acute lymphoblastic leukemia (ALL) | 2018 | Targeted, Chemotherapy | Any setting | 2/3 | 60 | EFS: see trialMilestone: Dasatinib with chemotherapy for Ph-positive ALL |
| ABC | Melanoma | 2018 | Immunotherapy | Metastatic | 2 | 79 | Intracranial response rate: 46% vs 20%Milestone: Brain metastases: ipilimumab-nivolumab trials (ABC, CheckMate 204) |
| ACHIEVE | Colorectal cancer | 2018 | Chemotherapy | Adjuvant | 3 | 1,313 | DFS: HR 0.95 (95% CI 0.76–1.20)Milestone: IDEA: 3 versus 6 months of adjuvant oxaliplatin chemotherapy |
| ADMIRAL | Acute myeloid leukaemia (including APL) | 2018 | Targeted | Relapsed / refractory | 3 | 371 | OS: median 9.3 vs 5.6 months, HR 0.64 (95% CI 0.49–0.83)Milestone: ADMIRAL: gilteritinib versus salvage chemotherapy, FLT3-mutated AML |
| ARAMIS | Prostate cancer | 2018 | Endocrine | Any setting | 3 | 1,509 | MFS: median 40.4 vs 18.4 months, HR 0.41 (95% CI 0.34–0.50)Milestone: AR inhibitors and metastasis-free survival in non-metastatic CRPC |
| AspECT | Gastric and oesophageal cancer | 2018 | Detection & prevention | Screening, Prevention | 3 | 2,557 | No results recordedMilestone: Barrett oesophagus: AspECT, BEST3 and BOSS randomised trials |
| BEST3 | Gastric and oesophageal cancer | 2018 | Detection & prevention | Screening, Prevention | — | 13,514 | No results recordedMilestone: Barrett oesophagus: AspECT, BEST3 and BOSS randomised trials |
| BOSS | Gastric and oesophageal cancer | 2018 | Detection & prevention | Screening, Prevention | — | 3,453 | OS: HR 0.95 (95% CI 0.82–1.10)Milestone: Barrett oesophagus: AspECT, BEST3 and BOSS randomised trials |
| CA180-372/AALL1122 | Childhood acute lymphoblastic leukemia (ALL) | 2018 | Targeted, Chemotherapy | Any setting | 2 | 106 | EFS: 65.5% vs 49.2% at 36 moMilestone: Dasatinib with chemotherapy for Ph-positive ALL |
| CARMENA | Kidney cancer (renal cell carcinoma) | 2018 | Surgery, Targeted | Metastatic | 3 | 450 | OS: median 18.4 vs 13.9 months, HR 0.89 (95% CI 0.71–1.10)Milestone: CARMENA and SURTIME question upfront cytoreductive nephrectomy |
| CASPIAN | Lung cancer | 2018 | Immunotherapy, Chemotherapy | Metastatic | 3 | 805 | OS: median 10.4 vs 10.5 months, HR 0.82 (95% CI 0.68–1.00)Milestone: Extensive-stage SCLC: chemotherapy plus atezolizumab or durvalumab |
| CELESTIAL | Hepatocellular carcinoma | 2018 | Targeted | Metastatic | 3 | 707 | OS: median 10.2 vs 8 months, HR 0.76 (95% CI 0.63–0.92)Milestone: CELESTIAL: cabozantinib after prior systemic therapy |
| CheckMate 204 | Melanoma | 2018 | Immunotherapy | Metastatic | 2 | 94 | Intracranial clinical benefit rate: see trialMilestone: Brain metastases: ipilimumab-nivolumab trials (ABC, CheckMate 204) |
| CheckMate 214 | Kidney cancer (renal cell carcinoma) | 2018 | Immunotherapy | Metastatic | 3 | 1,096 | OS: 75% vs 60% at 18 mo, HR 0.63Milestone: CheckMate 214: nivolumab plus ipilimumab versus sunitinib (2018) |
| CHIPOR | Ovarian cancer | 2018 | Surgery, Chemotherapy | Perioperative | 3 | 415 | OS: median 54.3 vs 45.8 months, HR 0.73 (95% CI 0.56–0.96)Milestone: HIPEC added to interval cytoreductive surgery: OVHIPEC-1 |
| dasatinib-vs-imatinib-china | Childhood acute lymphoblastic leukemia (ALL) | 2018 | Targeted, Chemotherapy | Any setting | 3 | 189 | EFS: 71% vs 48.9% at 48 moMilestone: Dasatinib with chemotherapy for Ph-positive ALL |
| De-ESCALaTE HPV | Head and neck cancer | 2018 | Targeted, Radiation, Chemotherapy | Locally advanced | 3 | 334 | OS: 89.4% vs 97.5% at 24 mo, HR 5.0 (95% CI 1.7–14.7)Milestone: Cetuximab worse than cisplatin with radiotherapy, HPV-positive cancer |
| entrectinib-ntrk-integrated | Thyroid cancer | 2018 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: NTRK fusions: larotrectinib and entrectinib across tumour types |
| EORTC 1325/KEYNOTE-054 | Melanoma | 2018 | Immunotherapy | Adjuvant | 3 | 1,019 | RFS: 75.4% vs 61% at 12 mo, HR 0.57Milestone: Adjuvant pembrolizumab: EORTC 1325/KEYNOTE-054 (2018) |
| EORTC 55994 | Cervical cancer | 2018 | Chemotherapy, Surgery, Radiation | Locally advanced | 3 | 626 | OS: 72% vs 76% at 60 moMilestone: Neoadjuvant chemotherapy then surgery did not replace chemoradiation |
| fader-uterine-serous-trastuzumab | Endometrial cancer | 2018 | Targeted, Chemotherapy | Metastatic | 2 | 61 | PFS: median 12.6 vs 8 months, HR 0.44Milestone: Trastuzumab with chemotherapy in HER2-positive uterine serous cancer |
| FRESCO | Colorectal cancer | 2018 | Targeted | Metastatic, Relapsed / refractory | 3 | 416 | OS: median 9.3 vs 6.6 months, HR 0.65 (95% CI 0.51–0.83)Milestone: Fruquintinib for refractory metastatic CRC (FRESCO and FRESCO-2) |
| FRESCO-2 | Colorectal cancer | 2018 | Targeted | Metastatic, Relapsed / refractory | 3 | 691 | OS: median 7.4 vs 4.8 months, HR 0.66 (95% CI 0.55–0.80)Milestone: Fruquintinib for refractory metastatic CRC (FRESCO and FRESCO-2) |
| HOME | Cervical cancer | 2018 | Detection & prevention | Screening | — | 19,851 | Screening uptake within 6 months of randomization: 26.3% vs 17.4%Milestone: HPV self-sampling: meta-analysis, mailed kits and FDA actions |
| HORG | Colorectal cancer | 2018 | Chemotherapy | Adjuvant | 3 | 1,115 | DFS: 77.2% vs 77.9% at 36 mo, HR 1.05 (95% CI 0.61–1.55)Milestone: IDEA: 3 versus 6 months of adjuvant oxaliplatin chemotherapy |
| IDEA | Colorectal cancer | 2018 | Chemotherapy | Adjuvant | meta-analysis | 12,834 | DFS: HR 1.07 (95% CI 1.00–1.15)Milestone: IDEA: 3 versus 6 months of adjuvant oxaliplatin chemotherapy |
| IDEA France | Colorectal cancer | 2018 | Chemotherapy | Adjuvant | 3 | 2,010 | DFS: 72% vs 76% at 36 mo, HR 1.24 (95% CI 1.05–1.46)Milestone: IDEA: 3 versus 6 months of adjuvant oxaliplatin chemotherapy |
| IMpower133 | Lung cancer | 2018 | Immunotherapy, Chemotherapy | Metastatic | 3 | — | OS: median 12.3 vs 10.3 months, HR 0.70 (95% CI 0.54–0.91)Milestone: Extensive-stage SCLC: chemotherapy plus atezolizumab or durvalumab |
| KATHERINE | Breast cancer | 2018 | Antibody–drug conjugate, Targeted | Adjuvant, Neoadjuvant | 3 | 1,486 | iDFS: 88.3% vs 77% at 36 mo, HR 0.50 (95% CI 0.39–0.64)Milestone: KATHERINE: T-DM1 for residual disease after neoadjuvant therapy |
| kerns-2018-cumulative-burden | Testicular germ cell tumors | 2018 | Supportive care, Chemotherapy | Any setting | observational | 1,214 | Cumulative burden of morbidity category: see trialMilestone: Cumulative burden of morbidity after cisplatin-based chemotherapy |
| KEYNOTE-158 | Cervical cancer | 2018 | Immunotherapy | Metastatic | 2 | 98 | ORR: see trialMilestone: KEYNOTE-158: pembrolizumab approved for PD-L1-positive disease (2018) |
| KEYNOTE-224 | Hepatocellular carcinoma | 2018 | Immunotherapy | Metastatic | 2 | 104 | ORR: see trialMilestone: Pembrolizumab: KEYNOTE-224, -240 and -394 and the FDA label |
| KEYNOTE-240 | Hepatocellular carcinoma | 2018 | Immunotherapy | Metastatic | 3 | 413 | OS: median 13.9 vs 10.6 months, HR 0.781 (95% CI 0.611–0.998)Milestone: Pembrolizumab: KEYNOTE-224, -240 and -394 and the FDA label |
| KEYNOTE-394 | Hepatocellular carcinoma | 2018 | Immunotherapy | Metastatic | 3 | 453 | OS: median 14.6 vs 13 months, HR 0.79 (95% CI 0.63–0.99)Milestone: Pembrolizumab: KEYNOTE-224, -240 and -394 and the FDA label |
| KOV-HIPEC-01 | Ovarian cancer | 2018 | Surgery, Chemotherapy | Perioperative | — | 184 | PFS: median 19.8 vs 18.8 monthsMilestone: HIPEC added to interval cytoreductive surgery: OVHIPEC-1 |
| LACC | Cervical cancer | 2018 | Surgery | Localised | — | 631 | DFS: 91.2% vs 97.1% at 36 mo, HR 3.74 (95% CI 1.63–8.58)Milestone: LACC: lower survival with minimally invasive radical hysterectomy |
| larotrectinib-trk-fusion-pooled | Thyroid cancer | 2018 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: NTRK fusions: larotrectinib and entrectinib across tumour types |
| NACTcervix | Cervical cancer | 2018 | Chemotherapy, Surgery, Radiation | Locally advanced | 3 | 635 | DFS: 69.3% vs 76.7% at 60 mo, HR 1.38 (95% CI 1.02–1.87)Milestone: Neoadjuvant chemotherapy then surgery did not replace chemoradiation |
| OpACIN⚑ needs review | Melanoma | 2018 | Immunotherapy, Surgery | Neoadjuvant | — | 20 | Pathological response rate: see trialMilestone: Neoadjuvant checkpoint blockade: OpACIN, OpACIN-neo and PRADO |
| OpACIN-neo | Melanoma | 2018 | Immunotherapy, Surgery | Neoadjuvant | 2 | 89 | Grade 3-4 immune-related adverse events: 20% vs 40%Milestone: Neoadjuvant checkpoint blockade: OpACIN, OpACIN-neo and PRADO |
| OVHIPEC-1 | Ovarian cancer | 2018 | Surgery, Chemotherapy | Perioperative | 3 | 245 | RFS: median 14.2 vs 10.7 months, HR 0.66 (95% CI 0.50–0.87)Milestone: HIPEC added to interval cytoreductive surgery: OVHIPEC-1 |
| PRADO | Melanoma | 2018 | Immunotherapy, Surgery | Neoadjuvant | 2 | 99 | Pathological response rate: see trialMilestone: Neoadjuvant checkpoint blockade: OpACIN, OpACIN-neo and PRADO |
| PRODIGE 24 | Pancreatic cancer | 2018 | Chemotherapy | Adjuvant | 3 | 493 | DFS: median 21.6 vs 12.8 months, HR 0.58 (95% CI 0.46–0.73)Milestone: PRODIGE 24: adjuvant modified FOLFIRINOX versus gemcitabine |
| PROSPER | Prostate cancer | 2018 | Endocrine | Any setting | 3 | 1,401 | MFS: median 36.6 vs 14.7 months, HR 0.29 (95% CI 0.24–0.35)Milestone: AR inhibitors and metastasis-free survival in non-metastatic CRPC |
| reflect-hcc | Hepatocellular carcinoma | 2018 | Targeted | Metastatic | 3 | 954 | OS: median 13.6 vs 12.3 months, HR 0.92 (95% CI 0.79–1.06)Milestone: REFLECT: lenvatinib non-inferior to sorafenib first line |
| RELEVANCE | Non-Hodgkin lymphoma (DLBCL and follicular) | 2018 | Immunotherapy, Chemotherapy | Locally advanced | 3 | 1,030 | CR rate: 48% vs 53%Milestone: RELEVANCE: rituximab-lenalidomide vs chemoimmunotherapy (2018) |
| RTOG 1016 | Head and neck cancer | 2018 | Targeted, Radiation, Chemotherapy | Locally advanced | 3 | 849 | OS: 77.9% vs 84.6% at 60 mo, HR 1.45Milestone: Cetuximab worse than cisplatin with radiotherapy, HPV-positive cancer |
| SCOT | Colorectal cancer | 2018 | Chemotherapy | Adjuvant | 3 | 6,088 | DFS: 76.7% vs 77.1% at 36 mo, HR 1.006 (95% CI 0.909–1.114)Milestone: IDEA: 3 versus 6 months of adjuvant oxaliplatin chemotherapy |
| SOLO-1 | Ovarian cancer | 2018 | Targeted | Maintenance | 3 | 391 | PFS: 60% vs 27% at 36 mo, HR 0.30 (95% CI 0.23–0.41)Milestone: SOLO-1: first-line olaparib maintenance for BRCA-mutated disease |
| SPARTAN | Prostate cancer | 2018 | Endocrine | Any setting | 3 | 1,207 | MFS: median 40.5 vs 16.2 months, HR 0.28 (95% CI 0.23–0.35)Milestone: AR inhibitors and metastasis-free survival in non-metastatic CRPC |
| SURTIME | Kidney cancer (renal cell carcinoma) | 2018 | Surgery, Targeted | Metastatic | 3 | 99 | 28-week progression-free rate: 43% vs 42%Milestone: CARMENA and SURTIME question upfront cytoreductive nephrectomy |
| TOSCA | Colorectal cancer | 2018 | Chemotherapy | Adjuvant | 3 | 3,759 | RFS: HR 1.14 (95% CI 0.99–1.32)Milestone: IDEA: 3 versus 6 months of adjuvant oxaliplatin chemotherapy |
| venetoclax-hma-phase-1b | Acute myeloid leukaemia (including APL) | 2018 | Targeted | Any setting | 1 | 145 | CR+CRi: see trialMilestone: Venetoclax with azacitidine for unfit AML (VIALE-A) |
| VIALE-A | Acute myeloid leukaemia (including APL) | 2018 | Targeted | Any setting | 3 | 431 | OS: median 14.7 vs 9.6 months, HR 0.66 (95% CI 0.52–0.85)Milestone: Venetoclax with azacitidine for unfit AML (VIALE-A) |
| VIALE-C | Acute myeloid leukaemia (including APL) | 2018 | Targeted | Any setting | 3 | 211 | OS: median 7.2 vs 4.1 months, HR 0.75 (95% CI 0.52–1.07)Milestone: Venetoclax with azacitidine for unfit AML (VIALE-A) |
| ANBL0532 | Neuroblastoma and Wilms tumour | 2019 | Cell therapy & transplant, Chemotherapy | Any setting | 3 | 355 | EFS: 61.6% vs 48.4% at 36 moMilestone: Tandem vs single autologous transplant in high-risk neuroblastoma |
| ANNOUNCE | Sarcoma and GIST | 2019 | Immunotherapy, Chemotherapy | Metastatic | 3 | 509 | OS: median 20.4 vs 19.7 months, HR 1.05 (95% CI 0.84–1.30)Milestone: Olaratumab: phase 2 survival benefit not confirmed in ANNOUNCE |
| ARCHES | Prostate cancer | 2019 | Endocrine | Metastatic | 3 | 1,150 | rPFS: HR 0.39 (95% CI 0.3–0.5)Milestone: AR inhibitors with ADT, hormone-sensitive (TITAN, ARCHES, ENZAMET) |
| ASCEND | Chronic lymphocytic leukaemia | 2019 | Targeted | Metastatic, Relapsed / refractory | 3 | 310 | PFS: 88% vs 68% at 12 mo, HR 0.31 (95% CI 0.20–0.49)Milestone: Acalabrutinib: ELEVATE-TN, ASCEND, ELEVATE-RR |
| BEACON CRC | Colorectal cancer | 2019 | Targeted | Metastatic, Relapsed / refractory | 3 | 665 | OS: median 9 vs 5.4 months, HR 0.52 (95% CI 0.39–0.70)Milestone: BEACON: encorafenib plus cetuximab for BRAF V600E mCRC |
| BLC2001 | Bladder cancer | 2019 | Targeted | Metastatic | 2 | — | ORR: see trialMilestone: Erdafitinib for FGFR-altered urothelial cancer: BLC2001 to THOR |
| CALGB 50303 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2019 | Chemotherapy, Immunotherapy | Locally advanced | 3 | 524 | PFS: 78.9% vs 75.5% at 24 mo, HR 0.93 (95% CI 0.68–1.27)Milestone: DA-EPOCH-R not better than R-CHOP: CALGB/Alliance 50303 (2019) |
| CAPItello-291 | Breast cancer | 2019 | Targeted, Endocrine | Metastatic | 3 | 708 | PFS: median 7.2 vs 3.6 months, HR 0.60 (95% CI 0.51–0.71)Milestone: PI3K/AKT pathway inhibitors after endocrine therapy |
| CASSIOPEIA | Multiple myeloma | 2019 | Immunotherapy, Cell therapy & transplant | Any setting | 3 | 1,085 | sCR rate: 29% vs 20%Milestone: Quadruplet induction before transplant: CASSIOPEIA, GRIFFIN, PERSEUS |
| CeTeG | Glioblastoma | 2019 | Chemotherapy | Maintenance | 3 | 141 | OS: median 48.1 vs 31.4 months, HR 0.60 (95% CI 0.35–1.03)Milestone: Lomustine plus temozolomide in MGMT-methylated glioblastoma (CeTeG) |
| CLL14 | Chronic lymphocytic leukaemia | 2019 | Targeted, Immunotherapy | Metastatic | 3 | 432 | PFS: 88.2% vs 64.1% at 24 mo, HR 0.35 (95% CI 0.23–0.53)Milestone: CLL14: fixed-duration venetoclax-obinutuzumab for comorbid patients |
| DESKTOP III | Ovarian cancer | 2019 | Surgery, Chemotherapy | Relapsed / refractory | — | 407 | OS: median 53.7 vs 46 months, HR 0.75 (95% CI 0.59–0.96)Milestone: Secondary cytoreduction at first relapse: GOG-213, SOC-1, DESKTOP III |
| E1609 | Melanoma | 2019 | Immunotherapy | Adjuvant | 3 | 1,670 | OS: HR 0.78Milestone: E1609: adjuvant ipilimumab versus high-dose interferon (2019) |
| ELEVATE-RR | Chronic lymphocytic leukaemia | 2019 | Targeted | Metastatic, Relapsed / refractory | 3 | 533 | PFS: median 38.4 vs 38.4 months, HR 1.00 (95% CI 0.79–1.27)Milestone: Acalabrutinib: ELEVATE-TN, ASCEND, ELEVATE-RR |
| ELEVATE-TN | Chronic lymphocytic leukaemia | 2019 | Targeted | Metastatic, Relapsed / refractory | 3 | 535 | PFS: 93% vs 47% at 24 mo, HR 0.10 (95% CI 0.06–0.17)Milestone: Acalabrutinib: ELEVATE-TN, ASCEND, ELEVATE-RR |
| ENZAMET | Prostate cancer | 2019 | Endocrine | Metastatic | 3 | 1,125 | OS: 67% vs 57% at 60 mo, HR 0.70 (95% CI 0.58–0.84)Milestone: AR inhibitors with ADT, hormone-sensitive (TITAN, ARCHES, ENZAMET) |
| EV-201 | Bladder cancer | 2019 | Antibody–drug conjugate | Metastatic | 2 | — | ORR: see trialMilestone: Enfortumab vedotin after platinum and PD-1/PD-L1: EV-201, EV-301 |
| EV-301 | Bladder cancer | 2019 | Antibody–drug conjugate | Metastatic | 3 | 608 | OS: median 12.88 vs 8.97 months, HR 0.70 (95% CI 0.56–0.89)Milestone: Enfortumab vedotin after platinum and PD-1/PD-L1: EV-201, EV-301 |
| FLOT4-AIO | Gastric and oesophageal cancer | 2019 | Chemotherapy, Surgery | Perioperative | 2/3 | 716 | OS: median 50 vs 35 months, HR 0.77 (95% CI 0.63–0.94)Milestone: FLOT4: perioperative FLOT versus ECF/ECX in gastric and GEJ cancer |
| GMMG-HD7 | Multiple myeloma | 2019 | Immunotherapy | Relapsed / refractory, Any setting | 3 | 660 | MRD: 50% vs 36%Milestone: Isatuximab: ICARIA-MM, IKEMA, HD7 and IMROZ |
| GO29365 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2019 | Antibody–drug conjugate, Chemotherapy, Immunotherapy | Relapsed / refractory | 1/2 | 80 | PFS: median 9.5 vs 3.7 months, HR 0.36 (95% CI 0.21–0.63)Milestone: Polatuzumab vedotin with bendamustine-rituximab: FDA 2019 |
| GOG-0213 (surgical objective) | Ovarian cancer | 2019 | Surgery, Chemotherapy | Relapsed / refractory | 3 | 485 | OS: median 50.6 vs 64.7 months, HR 1.29 (95% CI 0.97–1.72)Milestone: Secondary cytoreduction at first relapse: GOG-213, SOC-1, DESKTOP III |
| GOG-0252 | Ovarian cancer | 2019 | Chemotherapy | Locally advanced | 3 | 1,560 | PFS: HR 0.925 (95% CI 0.802–1.070)Milestone: GOG-252: IP regimens plus bevacizumab, no significant PFS gain |
| GOG-258 | Endometrial cancer | 2019 | Chemotherapy, Radiation | Adjuvant | 3 | 813 | RFS: 59% vs 58% at 60 mo, HR 0.9Milestone: GOG-258: adding radiotherapy to chemotherapy did not extend RFS |
| GRIFFIN | Multiple myeloma | 2019 | Immunotherapy, Cell therapy & transplant | Any setting | 2 | 207 | sCR rate: 42.4% vs 32%Milestone: Quadruplet induction before transplant: CASSIOPEIA, GRIFFIN, PERSEUS |
| HD16 | Hodgkin lymphoma | 2019 | Radiation, Understanding | Localised | 3 | 1,150 | PFS: 86.1% vs 93.4% at 60 mo, HR 1.78 (95% CI 1.02–3.12)Milestone: GHSG HD16 and HD17: PET-guided radiotherapy in early stages |
| HD17 | Hodgkin lymphoma | 2019 | Radiation, Understanding | Localised | 3 | 1,100 | PFS: 95.1% vs 97.3% at 60 mo, HR 0.523 (95% CI 0.226–1.211)Milestone: GHSG HD16 and HD17: PET-guided radiotherapy in early stages |
| ICARIA-MM | Multiple myeloma | 2019 | Immunotherapy | Relapsed / refractory, Any setting | 3 | 307 | PFS: median 11.5 vs 6.5 months, HR 0.596 (95% CI 0.44–0.81)Milestone: Isatuximab: ICARIA-MM, IKEMA, HD7 and IMROZ |
| IKEMA | Multiple myeloma | 2019 | Immunotherapy | Relapsed / refractory, Any setting | 3 | 302 | PFS: HR 0.53Milestone: Isatuximab: ICARIA-MM, IKEMA, HD7 and IMROZ |
| IMROZ | Multiple myeloma | 2019 | Immunotherapy | Relapsed / refractory, Any setting | 3 | 446 | PFS: 63.2% vs 45.2% at 60 mo, HR 0.6Milestone: Isatuximab: ICARIA-MM, IKEMA, HD7 and IMROZ |
| INAVO120 | Breast cancer | 2019 | Targeted, Endocrine | Metastatic | 3 | 325 | PFS: median 15 vs 7.3 months, HR 0.43 (95% CI 0.32–0.59)Milestone: PI3K/AKT pathway inhibitors after endocrine therapy |
| KEYNOTE-048⚑ needs review | Head and neck cancer | 2019 | Immunotherapy, Chemotherapy | Metastatic | 3 | 882 | OS: median 13 vs 10.7 months, HR 0.77 (95% CI 0.63–0.93)Milestone: KEYNOTE-048: pembrolizumab first line, recurrent or metastatic |
| KEYNOTE-146 | Endometrial cancer | 2019 | Targeted, Immunotherapy | Metastatic, Relapsed / refractory | 1/2 | 108 | ORR: see trialMilestone: Lenvatinib plus pembrolizumab after platinum: KEYNOTE-146 and -775 |
| KEYNOTE-426 | Kidney cancer (renal cell carcinoma) | 2019 | Immunotherapy, Targeted | Metastatic | 3 | 861 | OS: 89.9% vs 78.3% at 12 mo, HR 0.53 (95% CI 0.38–0.74)Milestone: KEYNOTE-426: pembrolizumab plus axitinib versus sunitinib (2019) |
| KEYNOTE-775 | Endometrial cancer | 2019 | Targeted, Immunotherapy | Metastatic, Relapsed / refractory | 3 | 827 | PFS: median 6.6 vs 3.8 months, HR 0.60 (95% CI 0.50–0.72)Milestone: Lenvatinib plus pembrolizumab after platinum: KEYNOTE-146 and -775 |
| LION | Ovarian cancer | 2019 | Surgery | Locally advanced | — | 647 | OS: median 69.2 vs 65.5 months, HR 1.06 (95% CI 0.83–1.34)Milestone: LION: lymphadenectomy not linked to longer survival |
| olaratumab-phase-1b-2 | Sarcoma and GIST | 2019 | Immunotherapy, Chemotherapy | Metastatic | 1/2 | 148 | PFS: median 6.6 vs 4.1 months, HR 0.67 (95% CI 0.44–1.02)Milestone: Olaratumab: phase 2 survival benefit not confirmed in ANNOUNCE |
| ORATOR | Head and neck cancer | 2019 | Surgery, Radiation | Localised | 2 | 68 | Grade 2-5 adverse events: 97% vs 91%Milestone: ORATOR: transoral robotic surgery vs radiotherapy, oropharynx |
| PAOLA-1 | Ovarian cancer | 2019 | Targeted | Maintenance | 3 | 806 | PFS: median 22.1 vs 16.6 months, HR 0.59 (95% CI 0.49–0.72)Milestone: PAOLA-1: olaparib plus bevacizumab maintenance, HRD-positive disease |
| PERSEUS | Multiple myeloma | 2019 | Immunotherapy, Cell therapy & transplant | Any setting | 3 | 709 | PFS: 84.3% vs 67.7% at 48 mo, HR 0.42 (95% CI 0.30–0.59)Milestone: Quadruplet induction before transplant: CASSIOPEIA, GRIFFIN, PERSEUS |
| POLO | Pancreatic cancer | 2019 | Targeted | Maintenance, Metastatic | 3 | 154 | PFS: median 7.4 vs 3.8 months, HR 0.53 (95% CI 0.35–0.82)Milestone: POLO: maintenance olaparib for germline BRCA-mutated disease |
| PRIMA/ENGOT-OV26/GOG-3012 | Ovarian cancer | 2019 | Targeted | Maintenance | 3 | 733 | PFS: median 21.9 vs 10.4 months, HR 0.43 (95% CI 0.31–0.59)Milestone: PRIMA: first-line niraparib maintenance beyond BRCA |
| REACH | Hepatocellular carcinoma | 2019 | Targeted | Metastatic | 3 | 565 | OS: median 9.2 vs 7.6 months, HR 0.87 (95% CI 0.72–1.05)Milestone: REACH-2: ramucirumab in AFP-selected patients after sorafenib |
| REACH-2 | Hepatocellular carcinoma | 2019 | Targeted | Metastatic | 3 | 292 | OS: median 8.5 vs 7.3 months, HR 0.710 (95% CI 0.531–0.949)Milestone: REACH-2: ramucirumab in AFP-selected patients after sorafenib |
| SOC-1 | Ovarian cancer | 2019 | Surgery, Chemotherapy | Relapsed / refractory | 3 | 357 | PFS: median 17.4 vs 11.9 months, HR 0.58 (95% CI 0.45–0.74)Milestone: Secondary cytoreduction at first relapse: GOG-213, SOC-1, DESKTOP III |
| SOLAR-1 | Breast cancer | 2019 | Targeted, Endocrine | Metastatic | 3 | 572 | PFS: median 11 vs 5.7 months, HR 0.65 (95% CI 0.50–0.85)Milestone: PI3K/AKT pathway inhibitors after endocrine therapy |
| THOR | Bladder cancer | 2019 | Targeted | Metastatic | 3 | — | OS: median 12.1 vs 7.8 months, HR 0.64 (95% CI 0.47–0.88)Milestone: Erdafitinib for FGFR-altered urothelial cancer: BLC2001 to THOR |
| TITAN | Prostate cancer | 2019 | Endocrine | Metastatic | 3 | 1,052 | rPFS: 68.2% vs 47.5% at 24 mo, HR 0.48 (95% CI 0.39–0.60)Milestone: AR inhibitors with ADT, hormone-sensitive (TITAN, ARCHES, ENZAMET) |
| VIKTORIA-1⚑ needs review | Breast cancer | 2019 | Targeted, Endocrine | Metastatic | 3 | 392 | PFS: median 9.3 vs 2 months, HR 0.24 (95% CI 0.17–0.35)Milestone: PI3K/AKT pathway inhibitors after endocrine therapy |
| AALL1621 | Childhood acute lymphoblastic leukemia (ALL) | 2020 | Antibody–drug conjugate | Relapsed / refractory | 2 | 48 | Overall remission rate: see trialMilestone: Inotuzumab ozogamicin for relapsed paediatric CD22+ ALL |
| ADAURA | Lung cancer | 2020 | Targeted | Adjuvant | 3 | 682 | DFS: 90% vs 44% at 24 mo, HR 0.17Milestone: ADAURA: adjuvant osimertinib after resection |
| ARROW | Lung cancer | 2020 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: RET fusion-positive NSCLC: selpercatinib and pralsetinib |
| ASCENT | Breast cancer | 2020 | Antibody–drug conjugate | Metastatic | 3 | 529 | PFS: median 5.6 vs 1.7 months, HR 0.41 (95% CI 0.32–0.52)Milestone: Sacituzumab govitecan (ASCENT): first TROP-2 ADC in metastatic TNBC |
| ATLANTIS | Lung cancer | 2020 | Chemotherapy, Immunotherapy | Relapsed / refractory, Maintenance | 3 | 613 | OS: median 8.6 vs 7.6 months, HR 0.97 (95% CI 0.82–1.15)Milestone: Lurbinectedin in SCLC: 2020 approval, IMforte, 2026 withdrawal |
| BELLINI | Multiple myeloma | 2020 | Targeted | Relapsed / refractory | 3 | 291 | PFS: median 22.4 vs 11.5 months, HR 0.63 (95% CI 0.44–0.90)Milestone: Venetoclax in myeloma: BELLINI signals harm, CANOVA misses PFS |
| CANOVA | Multiple myeloma | 2020 | Targeted | Relapsed / refractory | 3 | 263 | PFS: median 9.9 vs 5.8 months, HR 0.823 (95% CI 0.596–1.136)Milestone: Venetoclax in myeloma: BELLINI signals harm, CANOVA misses PFS |
| CheckMate 143 | Glioblastoma | 2020 | Immunotherapy | Relapsed / refractory, Adjuvant | 3 | 369 | OS: median 9.8 vs 10 months, HR 1.04 (95% CI 0.83–1.30)Milestone: Nivolumab phase 3 trials in glioblastoma do not meet their endpoints |
| CheckMate 498 | Glioblastoma | 2020 | Immunotherapy | Relapsed / refractory, Adjuvant | 3 | 560 | OS: median 13.4 vs 14.9 months, HR 1.31 (95% CI 1.09–1.58)Milestone: Nivolumab phase 3 trials in glioblastoma do not meet their endpoints |
| CheckMate 548 | Glioblastoma | 2020 | Immunotherapy | Relapsed / refractory, Adjuvant | 3 | 716 | PFS: median 10.6 vs 10.3 months, HR 1.1 (95% CI 0.9–1.3)Milestone: Nivolumab phase 3 trials in glioblastoma do not meet their endpoints |
| CLL12 | Chronic lymphocytic leukaemia | 2020 | Chemotherapy, Immunotherapy, Targeted | Localised | 3 | 363 | EFS: HR 0.25 (95% CI 0.14–0.43)Milestone: Early treatment of asymptomatic CLL: FCR (CLL7), ibrutinib (CLL12) |
| CLL7 | Chronic lymphocytic leukaemia | 2020 | Chemotherapy, Immunotherapy, Targeted | Localised | 3 | 800 | EFS: HR 0.22 (95% CI 0.15–0.33)Milestone: Early treatment of asymptomatic CLL: FCR (CLL7), ibrutinib (CLL12) |
| CROWN | Lung cancer | 2020 | Targeted | Metastatic | 3 | 296 | PFS: 78% vs 39% at 12 mo, HR 0.28 (95% CI 0.19–0.41)Milestone: CROWN: lorlatinib first-line, long follow-up |
| cs-003-nadofaragene | Bladder cancer | 2020 | Immunotherapy | Localised | 3 | 157 | Complete response in carcinoma in situ: see trialMilestone: Nadofaragene firadenovec for BCG-unresponsive NMIBC |
| DESTINY-Gastric01 | Gastric and oesophageal cancer | 2020 | Antibody–drug conjugate | Metastatic | 2 | 187 | OS: median 12.5 vs 8.4 months, HR 0.59 (95% CI 0.39–0.88)Milestone: Trastuzumab deruxtecan in HER2-positive gastric cancer |
| DESTINY-Gastric04 | Gastric and oesophageal cancer | 2020 | Antibody–drug conjugate | Metastatic | 3 | 494 | OS: median 14.7 vs 11.4 months, HR 0.70 (95% CI 0.55–0.90)Milestone: Trastuzumab deruxtecan in HER2-positive gastric cancer |
| GARNET | Endometrial cancer | 2020 | Immunotherapy | Relapsed / refractory | 1 | 104 | ORR: see trialMilestone: GARNET: dostarlimab in recurrent dMMR endometrial cancer |
| garrel-sentinel-node-vs-neck-dissection | Head and neck cancer | 2020 | Surgery | Localised | 3 | 307 | Neck node recurrence-free survival: 90.7% vs 89.6% at 24 moMilestone: Sentinel-node strategy vs neck dissection in early oral cancer |
| GEOMETRY mono-1 | Lung cancer | 2020 | Targeted | Metastatic | 2 | 364 | ORR: see trialMilestone: MET exon 14 skipping: capmatinib and tepotinib |
| IMbrave150 | Hepatocellular carcinoma | 2020 | Immunotherapy, Targeted | Metastatic | 3 | 501 | OS: 67.2% vs 54.6% at 12 mo, HR 0.58 (95% CI 0.42–0.79)Milestone: IMbrave150: atezolizumab plus bevacizumab versus sorafenib |
| IMforte | Lung cancer | 2020 | Chemotherapy, Immunotherapy | Relapsed / refractory, Maintenance | 3 | 483 | PFS: HR 0.54 (95% CI 0.43–0.67)Milestone: Lurbinectedin in SCLC: 2020 approval, IMforte, 2026 withdrawal |
| INVICTUS | Sarcoma and GIST | 2020 | Targeted | Relapsed / refractory, Metastatic | 3 | 129 | PFS: median 6.3 vs 1 months, HR 0.15 (95% CI 0.09–0.25)Milestone: Avapritinib (PDGFRA exon 18) and ripretinib (fourth line) |
| ITCC-059 | Childhood acute lymphoblastic leukemia (ALL) | 2020 | Antibody–drug conjugate | Relapsed / refractory | 1 | 25 | Overall remission rate: see trialMilestone: Inotuzumab ozogamicin for relapsed paediatric CD22+ ALL |
| JAVELIN Bladder 100 | Bladder cancer | 2020 | Immunotherapy | Maintenance | 3 | 700 | OS: median 21.4 vs 14.3 months, HR 0.69 (95% CI 0.56–0.86)Milestone: JAVELIN Bladder 100: avelumab maintenance after chemotherapy |
| KEYNOTE-057⚑ needs review | Bladder cancer | 2020 | Immunotherapy | Localised | 2 | 101 | Complete response in carcinoma in situ: see trialMilestone: KEYNOTE-057: pembrolizumab for BCG-unresponsive CIS |
| KEYNOTE-177 | Colorectal cancer | 2020 | Immunotherapy | Metastatic | 3 | 307 | PFS: median 16.5 vs 8.2 months, HR 0.60 (95% CI 0.45–0.80)Milestone: KEYNOTE-177: pembrolizumab first-line for MSI-H/dMMR metastatic CRC |
| KEYNOTE-522 | Breast cancer | 2020 | Immunotherapy, Chemotherapy | Neoadjuvant, Adjuvant, Perioperative | 3 | 1,174 | pCR: 64.8% vs 51.2%Milestone: KEYNOTE-522: pembrolizumab added to chemotherapy in early TNBC |
| LIBRETTO-001 | Thyroid cancer | 2020 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: Selpercatinib in RET-altered thyroid cancer (LIBRETTO-001) |
| LIBRETTO-001 | Lung cancer | 2020 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: RET fusion-positive NSCLC: selpercatinib and pralsetinib |
| LIBRETTO-431 | Lung cancer | 2020 | Targeted | Metastatic | 3 | 261 | PFS: median 24.8 vs 11.2 months, HR 0.46 (95% CI 0.31–0.70)Milestone: RET fusion-positive NSCLC: selpercatinib and pralsetinib |
| lurbinectedin-sclc-basket | Lung cancer | 2020 | Chemotherapy, Immunotherapy | Relapsed / refractory, Maintenance | 2 | 105 | ORR: see trialMilestone: Lurbinectedin in SCLC: 2020 approval, IMforte, 2026 withdrawal |
| monarchE | Breast cancer | 2020 | Targeted, Endocrine | Adjuvant | 3 | 5,637 | OS: 86.8% vs 85% at 84 mo, HR 0.842 (95% CI 0.722–0.981)Milestone: Adjuvant CDK4/6 inhibition: monarchE and NATALEE |
| NATALEE | Breast cancer | 2020 | Targeted, Endocrine | Adjuvant | 3 | 5,101 | iDFS: 90.4% vs 87.1% at 36 mo, HR 0.75 (95% CI 0.62–0.91)Milestone: Adjuvant CDK4/6 inhibition: monarchE and NATALEE |
| NAVIGATOR | Sarcoma and GIST | 2020 | Targeted | Relapsed / refractory, Metastatic | 1 | 82 | ORR: see trialMilestone: Avapritinib (PDGFRA exon 18) and ripretinib (fourth line) |
| NELSON⚑ needs review | Lung cancer | 2020 | Detection & prevention | Screening | — | — | Adherence to CT screening: see trialMilestone: NELSON: volume-CT screening and lung-cancer mortality |
| PREOPANC | Pancreatic cancer | 2020 | Chemotherapy, Radiation, Surgery | Neoadjuvant | 3 | 246 | OS: median 16 vs 14.3 months, HR 0.78 (95% CI 0.58–1.05)Milestone: PREOPANC: preoperative chemoradiotherapy versus immediate surgery |
| PROfound | Prostate cancer | 2020 | Targeted | Metastatic | 3 | 387 | rPFS: median 7.4 vs 3.6 months, HR 0.34 (95% CI 0.25–0.47)Milestone: PARP inhibitors for HRR-altered mCRPC (PROfound, TRITON) |
| proPSMA | Prostate cancer | 2020 | Understanding | Localised | — | 302 | Diagnostic accuracy: 92% vs 65%Milestone: PSMA PET-CT before curative-intent treatment (proPSMA) |
| QUAZAR AML-001 | Acute myeloid leukaemia (including APL) | 2020 | Chemotherapy | Maintenance | 3 | 472 | OS: median 24.7 vs 14.8 monthsMilestone: QUAZAR: oral azacitidine maintenance after first remission |
| RAPIDO | Colorectal cancer | 2020 | Chemotherapy, Radiation, Surgery | Neoadjuvant, Locally advanced | 3 | 920 | Disease-related treatment failure: 23.7% vs 30.4% at 36 mo, HR 0.75 (95% CI 0.60–0.95)Milestone: Total neoadjuvant therapy: RAPIDO and PRODIGE 23 |
| STRASS | Sarcoma and GIST | 2020 | Radiation, Surgery | Neoadjuvant | 3 | 266 | Abdominal recurrence-free survival: median 4.5 vs 5 years, HR 1.01 (95% CI 0.71–1.44)Milestone: STRASS: preoperative radiotherapy in retroperitoneal sarcoma |
| Study 201 | Neuroblastoma and Wilms tumour | 2020 | Immunotherapy | Relapsed / refractory | 2 | 74 | ORR: see trialMilestone: Naxitamab: accelerated approval for relapsed or refractory disease |
| TRITON2 | Prostate cancer | 2020 | Targeted | Metastatic | 2 | 115 | ORR: see trialMilestone: PARP inhibitors for HRR-altered mCRPC (PROfound, TRITON) |
| TRITON3 | Prostate cancer | 2020 | Targeted | Metastatic | 3 | 405 | rPFS: median 11.2 vs 6.4 months, HR 0.50 (95% CI 0.36–0.69)Milestone: PARP inhibitors for HRR-altered mCRPC (PROfound, TRITON) |
| UNICANCER-PRODIGE 23 | Colorectal cancer | 2020 | Chemotherapy, Radiation, Surgery | Neoadjuvant, Locally advanced | 3 | 461 | DFS: 76% vs 69% at 36 mo, HR 0.69 (95% CI 0.49–0.97)Milestone: Total neoadjuvant therapy: RAPIDO and PRODIGE 23 |
| VISION | Lung cancer | 2020 | Targeted | Metastatic | 2 | 152 | ORR: see trialMilestone: MET exon 14 skipping: capmatinib and tepotinib |
| AALL0932 | Childhood acute lymphoblastic leukemia (ALL) | 2021 | Chemotherapy | Maintenance | 3 | — | DFS: 95.1% vs 94.1% at 60 moMilestone: Fewer vincristine and dexamethasone pulses in standard risk |
| AALL1331 | Childhood acute lymphoblastic leukemia (ALL) | 2021 | Immunotherapy, Chemotherapy, Cell therapy & transplant | Relapsed / refractory | 3 | 208 | DFS: 54.4% vs 39% at 24 mo, HR 0.70 (95% CI 0.47–1.03)Milestone: Blinatumomab versus chemotherapy in first relapse of B-ALL |
| amgen-20120215 | Childhood acute lymphoblastic leukemia (ALL) | 2021 | Immunotherapy, Chemotherapy, Cell therapy & transplant | Relapsed / refractory | 3 | 108 | EFS: HR 0.33 (95% CI 0.18–0.61)Milestone: Blinatumomab versus chemotherapy in first relapse of B-ALL |
| ASCEMBL | Chronic myeloid leukemia | 2021 | Targeted | Relapsed / refractory | 3 | 233 | MMR: 25.5% vs 13.2%Milestone: Asciminib approved after ASCEMBL versus bosutinib |
| ATTRACTION-4 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 2/3 | 724 | PFS: median 10.45 vs 8.34 months, HR 0.68Milestone: First-line PD-1 plus chemotherapy in HER2-negative gastric cancer |
| Beamion LUNG-1 | Lung cancer | 2021 | Antibody–drug conjugate, Targeted | Metastatic | 1 | — | ORR: see trialMilestone: HER2-mutant NSCLC: trastuzumab deruxtecan and oral TKIs |
| BELINDA | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Cell therapy & transplant | Relapsed / refractory | 3 | 322 | EFS: median 3 vs 3 months, HR 1.07 (95% CI 0.82–1.40)Milestone: BELINDA: tisagenlecleucel not superior in second line (2021) |
| CAPTAIN-1st | Head and neck cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 263 | PFS: median 9.7 vs 6.9 months, HR 0.54 (95% CI 0.39–0.76)Milestone: PD-1 antibodies added to gemcitabine-cisplatin in first-line NPC |
| CARTITUDE-1 | Multiple myeloma | 2021 | Cell therapy & transplant | Relapsed / refractory | 1/2 | 113 | ORR: see trialMilestone: BCMA CAR T cells: ide-cel and cilta-cel, from KarMMa to CARTITUDE-4 |
| CARTITUDE-4 | Multiple myeloma | 2021 | Cell therapy & transplant | Relapsed / refractory | 3 | 419 | PFS: 75.9% vs 48.6% at 12 mo, HR 0.26 (95% CI 0.18–0.38)Milestone: BCMA CAR T cells: ide-cel and cilta-cel, from KarMMa to CARTITUDE-4 |
| CDRB436G2201 (high-grade glioma cohort) | Glioblastoma | 2021 | Targeted | Relapsed / refractory | 2 | 41 | ORR: see trialMilestone: Dabrafenib plus trametinib for BRAF V600E-mutant glioma |
| CDRB436G2201 (low-grade glioma cohort) | Glioblastoma | 2021 | Targeted | Relapsed / refractory | 2 | 110 | PFS: median 20.1 vs 7.4 months, HR 0.31 (95% CI 0.17–0.55)Milestone: Dabrafenib plus trametinib for BRAF V600E-mutant glioma |
| CheckMate 274⚑ needs review | Bladder cancer | 2021 | Immunotherapy | Adjuvant | 3 | 709 | DFS: median 20.8 vs 10.8 months, HR 0.7Milestone: CheckMate 274: adjuvant nivolumab after radical surgery |
| CheckMate 459 | Hepatocellular carcinoma | 2021 | Immunotherapy | Metastatic | 3 | 743 | OS: median 16.4 vs 14.7 months, HR 0.85 (95% CI 0.72–1.02)Milestone: CheckMate 459 and withdrawal of nivolumab's HCC indication |
| CheckMate 577 | Gastric and oesophageal cancer | 2021 | Immunotherapy | Adjuvant | 3 | 794 | DFS: median 22.4 vs 11 months, HR 0.69Milestone: CheckMate 577: adjuvant nivolumab after chemoradiotherapy and surgery |
| CheckMate 648 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 970 | OS: median 15.4 vs 9.1 months, HR 0.54Milestone: First-line PD-1 therapy: KEYNOTE-590 and CheckMate 648 |
| CheckMate 649 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 1,581 | OS: median 14.4 vs 11.1 months, HR 0.71Milestone: First-line PD-1 plus chemotherapy in HER2-negative gastric cancer |
| CheckMate 76K | Melanoma | 2021 | Immunotherapy | Adjuvant | 3 | 790 | RFS: 89% vs 79.4% at 12 mo, HR 0.42 (95% CI 0.30–0.59)Milestone: Adjuvant therapy in stage IIB/IIC: KEYNOTE-716 and CheckMate 76K |
| CheckMate 9ER | Kidney cancer (renal cell carcinoma) | 2021 | Immunotherapy, Targeted | Metastatic | 3 | 651 | PFS: median 16.6 vs 8.3 months, HR 0.51 (95% CI 0.41–0.64)Milestone: CheckMate 9ER: nivolumab plus cabozantinib versus sunitinib (2021) |
| CHRYSALIS | Lung cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 1 | 81 | ORR: see trialMilestone: EGFR exon 20 insertions: amivantamab, CHRYSALIS to PAPILLON |
| CLEAR | Kidney cancer (renal cell carcinoma) | 2021 | Immunotherapy, Targeted | Metastatic | 3 | 1,069 | PFS: median 23.9 vs 9.2 months, HR 0.39 (95% CI 0.32–0.49)Milestone: CLEAR: lenvatinib plus pembrolizumab versus sunitinib (2021) |
| CodeBreaK 100 | Lung cancer | 2021 | Targeted | Metastatic | 1/2 | 126 | ORR: see trialMilestone: KRAS G12C inhibitors: sotorasib and adagrasib |
| CodeBreaK 200 | Lung cancer | 2021 | Targeted | Metastatic | 3 | 345 | PFS: median 5.6 vs 4.5 months, HR 0.66 (95% CI 0.51–0.86)Milestone: KRAS G12C inhibitors: sotorasib and adagrasib |
| copanlisib-phase-2-indolent | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Targeted | Relapsed / refractory | 2 | 142 | ORR: see trialMilestone: PI3K inhibitors in FL: accelerated approvals withdrawn (2021-2024) |
| COSMIC-311 | Thyroid cancer | 2021 | Targeted | Metastatic, Relapsed / refractory | 3 | 187 | ORR: 15% vs 0%Milestone: COSMIC-311: cabozantinib after prior VEGFR therapy in DTC |
| DELTA | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Targeted | Relapsed / refractory | 2 | 125 | ORR: see trialMilestone: PI3K inhibitors in FL: accelerated approvals withdrawn (2021-2024) |
| DESTINY-Lung01 | Lung cancer | 2021 | Antibody–drug conjugate, Targeted | Metastatic | 2 | 91 | ORR: see trialMilestone: HER2-mutant NSCLC: trastuzumab deruxtecan and oral TKIs |
| DUO | Chronic lymphocytic leukaemia | 2021 | Targeted | Relapsed / refractory | 3 | 319 | PFS: median 13.3 vs 9.9 months, HR 0.52Milestone: PI3K inhibitors: CLL approvals, lymphoma indications withdrawn |
| durvalumab-phase-1-2-urothelial | Bladder cancer | 2021 | Immunotherapy | Metastatic | 1/2 | — | ORR: see trialMilestone: Atezolizumab and durvalumab urothelial indications withdrawn |
| DYNAMO | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Targeted | Relapsed / refractory | 2 | 129 | ORR: see trialMilestone: PI3K inhibitors in FL: accelerated approvals withdrawn (2021-2024) |
| ELARA | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Cell therapy & transplant | Relapsed / refractory | 2 | 98 | CR rate: see trialMilestone: ZUMA-5: axicabtagene ciloleucel in relapsed follicular lymphoma (2021) |
| ESCUDDO | Cervical cancer | 2021 | Detection & prevention | Prevention | — | 20,330 | No results recordedMilestone: Fewer HPV vaccine doses: India, KEN SHE and ESCUDDO (2021-2025) |
| fossa-2022-psychosocial-mortality | Testicular germ cell tumors | 2021 | Supportive care, Chemotherapy, Radiation | Any setting | observational | 775 | Cumulative overall mortality: see trialMilestone: Non-testicular-cancer mortality in long-term survivors (cisplatin era) |
| hellesnes-2021-non-tc-mortality | Testicular germ cell tumors | 2021 | Supportive care, Chemotherapy, Radiation | Any setting | observational | 5,707 | Non-testicular-cancer mortality: HR 1.47 (95% CI 0.91–2.39)Milestone: Non-testicular-cancer mortality in long-term survivors (cisplatin era) |
| iarc-india-hpv-dose-cohort | Cervical cancer | 2021 | Detection & prevention | Prevention | — | — | No results recordedMilestone: Fewer HPV vaccine doses: India, KEN SHE and ESCUDDO (2021-2025) |
| idelalisib-rituximab-cll | Chronic lymphocytic leukaemia | 2021 | Targeted | Relapsed / refractory | 3 | 220 | PFS: HR 0.15Milestone: PI3K inhibitors: CLL approvals, lymphoma indications withdrawn |
| igcccg-update-2021-nsgct | Testicular germ cell tumors | 2021 | Understanding | Metastatic | observational | 9,728 | OS: 89% vs 96% at 60 moMilestone: IGCCCG Update: modern survival estimates and refined prognosis |
| igcccg-update-2021-seminoma | Testicular germ cell tumors | 2021 | Understanding | Metastatic | observational | 2,451 | OS: 88% vs 95% at 60 moMilestone: IGCCCG Update: modern survival estimates and refined prognosis |
| IMagyn050 | Ovarian cancer | 2021 | Immunotherapy | Locally advanced, Metastatic, Maintenance | 3 | 1,301 | PFS: median 19.5 vs 18.4 months, HR 0.92 (95% CI 0.79–1.07)Milestone: First-line PD-L1 blockade: IMagyn050 and JAVELIN Ovarian 100 |
| IMCgp100-202 | Melanoma | 2021 | Immunotherapy | Metastatic | 3 | 378 | OS: 73% vs 59% at 12 mo, HR 0.51 (95% CI 0.37–0.71)Milestone: Tebentafusp: survival benefit in metastatic uveal melanoma (2021) |
| IMpassion130⚑ needs review | Breast cancer | 2021 | Immunotherapy | Metastatic | 3 | 902 | PFS: median 7.2 vs 5.5 months, HR 0.80 (95% CI 0.69–0.92)Milestone: Atezolizumab in PD-L1+ metastatic TNBC: approval, then withdrawal |
| IMpassion131 | Breast cancer | 2021 | Immunotherapy | Metastatic | 3 | 651 | PFS: median 6 vs 5.7 months, HR 0.82 (95% CI 0.60–1.12)Milestone: Atezolizumab in PD-L1+ metastatic TNBC: approval, then withdrawal |
| IMpower010⚑ needs review | Lung cancer | 2021 | Immunotherapy | Adjuvant | 3 | 1,005 | DFS: HR 0.66 (95% CI 0.50–0.88)Milestone: Adjuvant PD-(L)1 after resection: IMpower010 and PEARLS |
| IMvigor210 | Bladder cancer | 2021 | Immunotherapy | Metastatic | 2 | — | ORR: see trialMilestone: Atezolizumab and durvalumab urothelial indications withdrawn |
| IMvigor211 | Bladder cancer | 2021 | Immunotherapy | Metastatic | 3 | 931 | OS: median 11.1 vs 10.6 months, HR 0.87 (95% CI 0.63–1.21)Milestone: Atezolizumab and durvalumab urothelial indications withdrawn |
| IMvoke010 | Head and neck cancer | 2021 | Immunotherapy, Targeted, Chemotherapy, Radiation | Locally advanced, Adjuvant | 3 | 406 | EFS: median 59.5 vs 52.7 months, HR 0.94 (95% CI 0.70–1.26)Milestone: Four add-ons to definitive head and neck treatment show no benefit |
| innovaTV 204 | Cervical cancer | 2021 | Antibody–drug conjugate | Metastatic | 2 | 102 | ORR: see trialMilestone: Tisotumab vedotin: accelerated approval 2021, then innovaTV 301 |
| innovaTV 301 | Cervical cancer | 2021 | Antibody–drug conjugate | Metastatic | 3 | 502 | OS: median 11.5 vs 9.5 months, HR 0.70 (95% CI 0.54–0.89)Milestone: Tisotumab vedotin: accelerated approval 2021, then innovaTV 301 |
| JAVELIN Head and Neck 100 | Head and neck cancer | 2021 | Immunotherapy, Targeted, Chemotherapy, Radiation | Locally advanced, Adjuvant | 3 | 697 | PFS: HR 1.21 (95% CI 0.93–1.57)Milestone: Four add-ons to definitive head and neck treatment show no benefit |
| JAVELIN Ovarian 100 | Ovarian cancer | 2021 | Immunotherapy | Locally advanced, Metastatic, Maintenance | 3 | 998 | PFS: median 16.8 months, HR 1.43 (95% CI 1.05–1.95)Milestone: First-line PD-L1 blockade: IMagyn050 and JAVELIN Ovarian 100 |
| JUPITER-02⚑ needs review | Head and neck cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 289 | PFS: median 11.7 vs 8 months, HR 0.52 (95% CI 0.36–0.74)Milestone: PD-1 antibodies added to gemcitabine-cisplatin in first-line NPC |
| KarMMa | Multiple myeloma | 2021 | Cell therapy & transplant | Relapsed / refractory | 2 | 140 | ORR: see trialMilestone: BCMA CAR T cells: ide-cel and cilta-cel, from KarMMa to CARTITUDE-4 |
| KarMMa-3 | Multiple myeloma | 2021 | Cell therapy & transplant | Relapsed / refractory | 3 | 386 | PFS: median 13.3 vs 4.4 months, HR 0.49 (95% CI 0.38–0.65)Milestone: BCMA CAR T cells: ide-cel and cilta-cel, from KarMMa to CARTITUDE-4 |
| KEN SHE | Cervical cancer | 2021 | Detection & prevention | Prevention | — | 2,275 | No results recordedMilestone: Fewer HPV vaccine doses: India, KEN SHE and ESCUDDO (2021-2025) |
| KEYNOTE-062 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 763 | OS: median 10.6 vs 11.1 months, HR 0.91Milestone: First-line PD-1 plus chemotherapy in HER2-negative gastric cancer |
| KEYNOTE-355 | Breast cancer | 2021 | Immunotherapy | Metastatic | 3 | 847 | PFS: median 9.7 vs 5.6 months, HR 0.65 (95% CI 0.49–0.86)Milestone: Atezolizumab in PD-L1+ metastatic TNBC: approval, then withdrawal |
| KEYNOTE-412 | Head and neck cancer | 2021 | Immunotherapy, Targeted, Chemotherapy, Radiation | Locally advanced, Adjuvant | 3 | 804 | EFS: HR 0.83 (95% CI 0.68–1.03)Milestone: Four add-ons to definitive head and neck treatment show no benefit |
| KEYNOTE-564 | Kidney cancer (renal cell carcinoma) | 2021 | Immunotherapy | Adjuvant | 3 | 994 | DFS: 77.3% vs 68.1% at 24 mo, HR 0.68 (95% CI 0.53–0.87)Milestone: KEYNOTE-564: adjuvant pembrolizumab after nephrectomy (2021) |
| KEYNOTE-590 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 749 | OS: median 13.9 vs 8.8 months, HR 0.57 (95% CI 0.43–0.75)Milestone: First-line PD-1 therapy: KEYNOTE-590 and CheckMate 648 |
| KEYNOTE-716 | Melanoma | 2021 | Immunotherapy | Adjuvant | 3 | 976 | RFS: HR 0.65 (95% CI 0.46–0.92)Milestone: Adjuvant therapy in stage IIB/IIC: KEYNOTE-716 and CheckMate 76K |
| KEYNOTE-811 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Targeted, Chemotherapy | Metastatic | 3 | 698 | ORR: 74.4% vs 51.9%Milestone: KEYNOTE-811: pembrolizumab plus trastuzumab and chemotherapy |
| KEYNOTE-826 | Cervical cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 617 | PFS: median 10.4 vs 8.2 months, HR 0.62 (95% CI 0.50–0.77)Milestone: KEYNOTE-826: pembrolizumab added to first-line chemotherapy |
| KEYNOTE-859 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 1,579 | OS: median 12.9 vs 11.5 months, HR 0.78 (95% CI 0.70–0.87)Milestone: First-line PD-1 plus chemotherapy in HER2-negative gastric cancer |
| KRYSTAL-1 | Lung cancer | 2021 | Targeted | Metastatic | 1/2 | 116 | ORR: see trialMilestone: KRAS G12C inhibitors: sotorasib and adagrasib |
| KRYSTAL-12 | Lung cancer | 2021 | Targeted | Metastatic | 3 | 453 | PFS: median 5.5 vs 3.8 months, HR 0.58 (95% CI 0.45–0.76)Milestone: KRAS G12C inhibitors: sotorasib and adagrasib |
| LITESPARK-004 | Kidney cancer (renal cell carcinoma) | 2021 | Targeted | Any setting | 2 | 61 | ORR: see trialMilestone: Belzutifan for VHL disease-associated tumours (LITESPARK-004) |
| LN-144 | Melanoma | 2021 | Cell therapy & transplant | Metastatic, Relapsed / refractory | 2 | 66 | ORR: see trialMilestone: Lifileucel: from phase 2 to FDA accelerated approval (2024) |
| OlympiA | Breast cancer | 2021 | Targeted | Adjuvant | 3 | 1,836 | iDFS: 85.9% vs 77.1% at 36 mo, HR 0.58Milestone: OlympiA: one year of adjuvant olaparib for germline BRCA carriers |
| PAPILLON | Lung cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 308 | PFS: median 11.4 vs 6.7 months, HR 0.40 (95% CI 0.30–0.53)Milestone: EGFR exon 20 insertions: amivantamab, CHRYSALIS to PAPILLON |
| PEARLS | Lung cancer | 2021 | Immunotherapy | Adjuvant | 3 | 1,177 | DFS: median 53.6 vs 42 months, HR 0.76 (95% CI 0.63–0.91)Milestone: Adjuvant PD-(L)1 after resection: IMpower010 and PEARLS |
| POLARIX | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Antibody–drug conjugate, Immunotherapy, Chemotherapy | Locally advanced | 3 | 879 | PFS: 76.7% vs 70.2% at 24 mo, HR 0.73 (95% CI 0.57–0.95)Milestone: POLARIX: polatuzumab vedotin with R-CHP vs R-CHOP (2021) |
| PRODIGE 7 | Colorectal cancer | 2021 | Surgery, Chemotherapy | Metastatic | 3 | 265 | OS: median 41.7 vs 41.2 months, HR 1Milestone: PRODIGE 7: HIPEC added to cytoreductive surgery (peritoneal CRC) |
| RATIONALE-305 | Gastric and oesophageal cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 997 | OS: median 17.2 vs 12.6 months, HR 0.74 (95% CI 0.59–0.94)Milestone: First-line PD-1 plus chemotherapy in HER2-negative gastric cancer |
| RATIONALE-309 | Head and neck cancer | 2021 | Immunotherapy, Chemotherapy | Metastatic | 3 | 263 | PFS: HR 0.52 (95% CI 0.38–0.73)Milestone: PD-1 antibodies added to gemcitabine-cisplatin in first-line NPC |
| ROAR | Glioblastoma | 2021 | Targeted | Relapsed / refractory | 2 | 58 | ORR: see trialMilestone: Dabrafenib plus trametinib for BRAF V600E-mutant glioma |
| SOHO-01 | Lung cancer | 2021 | Antibody–drug conjugate, Targeted | Metastatic | 1/2 | 209 | ORR: see trialMilestone: HER2-mutant NSCLC: trastuzumab deruxtecan and oral TKIs |
| TheraP | Prostate cancer | 2021 | Radiation | Metastatic | 2 | 200 | PSA response rate: 66% vs 37%Milestone: 177Lu-PSMA-617 for PSMA-positive mCRPC (VISION, TheraP) |
| TRANSFORM | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Cell therapy & transplant | Relapsed / refractory | 3 | 184 | EFS: median 10.1 vs 2.3 months, HR 0.35 (95% CI 0.23–0.53)Milestone: ZUMA-7 and TRANSFORM: CAR T cells vs salvage plus transplant |
| TrilynX | Head and neck cancer | 2021 | Immunotherapy, Targeted, Chemotherapy, Radiation | Locally advanced, Adjuvant | 3 | 730 | EFS: median 19.4 vs 33.1 months, HR 1.33 (95% CI 1.05–1.67)Milestone: Four add-ons to definitive head and neck treatment show no benefit |
| UNITY-NHL | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Targeted | Relapsed / refractory | 2 | 208 | ORR: see trialMilestone: PI3K inhibitors in FL: accelerated approvals withdrawn (2021-2024) |
| VISION | Prostate cancer | 2021 | Radiation | Metastatic | 3 | 831 | rPFS: median 8.7 vs 3.4 months, HR 0.4Milestone: 177Lu-PSMA-617 for PSMA-positive mCRPC (VISION, TheraP) |
| ZUMA-5 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Cell therapy & transplant | Relapsed / refractory | 2 | 153 | ORR: see trialMilestone: ZUMA-5: axicabtagene ciloleucel in relapsed follicular lymphoma (2021) |
| ZUMA-7 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2021 | Cell therapy & transplant | Relapsed / refractory | 3 | 359 | EFS: median 8.3 vs 2 months, HR 0.40 (95% CI 0.31–0.51)Milestone: ZUMA-7 and TRANSFORM: CAR T cells vs salvage plus transplant |
| AHOD1331 | Hodgkin lymphoma | 2022 | Antibody–drug conjugate, Chemotherapy | Locally advanced, Metastatic | 3 | 600 | EFS: 92.1% vs 82.5% at 36 mo, HR 0.41 (95% CI 0.25–0.67)Milestone: AHOD1331: brentuximab vedotin in pediatric high-risk HL |
| ALPINE | Chronic lymphocytic leukaemia | 2022 | Targeted | Metastatic, Relapsed / refractory | 3 | 652 | ORR: 78.3% vs 62.5%Milestone: Zanubrutinib: SEQUOIA and the ALPINE head-to-head with ibrutinib |
| APACT | Pancreatic cancer | 2022 | Chemotherapy | Adjuvant | 3 | 866 | DFS: median 19.4 vs 18.8 months, HR 0.880 (95% CI 0.729–1.063)Milestone: APACT: adjuvant nab-paclitaxel plus gemcitabine, DFS not met |
| ARASENS | Prostate cancer | 2022 | Endocrine, Chemotherapy | Metastatic | 3 | 1,306 | OS: HR 0.68 (95% CI 0.57–0.80)Milestone: Triplet therapy: ADT, docetaxel and an AR inhibitor (ARASENS, PEACE-1) |
| ARIEL4 | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 3 | 349 | PFS: median 7.4 vs 5.7 months, HR 0.64 (95% CI 0.49–0.84)Milestone: PARP inhibitor later-line ovarian indications withdrawn (2022) |
| CALGB 140503 | Lung cancer | 2022 | Surgery | Localised | 3 | 697 | DFS: 63.6% vs 64.1% at 60 mo, HR 1.01Milestone: Segmentectomy and sublobar resection vs lobectomy (2022-2023) |
| CheckMate 651 | Head and neck cancer | 2022 | Immunotherapy, Targeted | Metastatic | 3 | 947 | OS: median 13.9 vs 13.5 months, HR 0.95Milestone: First-line immunotherapy combinations: no overall survival gain |
| CheckMate 816 | Lung cancer | 2022 | Immunotherapy, Chemotherapy, Surgery | Neoadjuvant | 3 | 358 | EFS: median 31.6 vs 20.8 months, HR 0.63Milestone: CheckMate 816: neoadjuvant nivolumab plus chemotherapy |
| CheckMate 914 | Kidney cancer (renal cell carcinoma) | 2022 | Immunotherapy | Adjuvant, Perioperative | 3 | 816 | DFS: HR 0.92 (95% CI 0.71–1.19)Milestone: Adjuvant checkpoint trials: IMmotion010, CheckMate 914, PROSPER |
| CodeBreaK 100 | Pancreatic cancer | 2022 | Targeted | Metastatic, Relapsed / refractory | 1/2 | 38 | ORR: see trialMilestone: KRAS G12C inhibitors in the small G12C-mutated subset |
| CodeBreaK 300⚑ needs review | Colorectal cancer | 2022 | Targeted | Metastatic, Relapsed / refractory | 3 | 160 | PFS: median 5.6 vs 2.2 months, HR 0.49 (95% CI 0.3–0.8)Milestone: KRAS G12C inhibitors with anti-EGFR antibodies |
| D0810C00042 | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 2 | 298 | ORR: see trialMilestone: PARP inhibitor later-line ovarian indications withdrawn (2022) |
| DESTINY-Breast03 | Breast cancer | 2022 | Antibody–drug conjugate, Targeted | Metastatic | 3 | 524 | PFS: 75.8% vs 34.1% at 12 mo, HR 0.28 (95% CI 0.22–0.37)Milestone: DESTINY-Breast03: T-DXd versus T-DM1 in HER2-positive disease |
| DESTINY-Breast04 | Breast cancer | 2022 | Antibody–drug conjugate, Targeted, Understanding | Metastatic | 3 | 557 | PFS: median 10.1 vs 5.4 months, HR 0.51Milestone: T-DXd in HER2-low breast cancer (DESTINY-Breast04, -06) |
| DESTINY-Breast06 | Breast cancer | 2022 | Antibody–drug conjugate, Targeted, Understanding | Metastatic | 3 | 866 | PFS: median 13.2 vs 8.1 months, HR 0.62 (95% CI 0.52–0.75)Milestone: T-DXd in HER2-low breast cancer (DESTINY-Breast04, -06) |
| dostarlimab-dmmr-rectal-phase2 | Colorectal cancer | 2022 | Immunotherapy | Neoadjuvant, Locally advanced | 2 | — | Clinical complete response: see trialMilestone: Dostarlimab alone for mismatch repair-deficient rectal cancer |
| DREAMseq | Melanoma | 2022 | Immunotherapy, Targeted | Metastatic | 3 | 265 | OS: see trialMilestone: Sequencing in BRAF-mutant disease: DREAMseq, SECOMBIT and EBIN |
| DYNAMIC | Colorectal cancer | 2022 | Chemotherapy, Understanding | Adjuvant | — | 455 | RFS: 93.5% vs 92.4% at 24 moMilestone: DYNAMIC: ctDNA-guided adjuvant therapy in stage II colon cancer |
| DYNAMIC-III | Colorectal cancer | 2022 | Chemotherapy, Understanding | Adjuvant | 2/3 | 968 | RFS: 85.3% vs 88.1% at 36 moMilestone: DYNAMIC: ctDNA-guided adjuvant therapy in stage II colon cancer |
| EBIN | Melanoma | 2022 | Immunotherapy, Targeted | Metastatic | 2 | 271 | PFS: median 9 vs 9 monthsMilestone: Sequencing in BRAF-mutant disease: DREAMseq, SECOMBIT and EBIN |
| EMERALD⚑ needs review | Breast cancer | 2022 | Endocrine | Metastatic | 3 | 477 | PFS: HR 0.70 (95% CI 0.55–0.88)Milestone: Elacestrant: the first oral SERD (EMERALD) |
| EMPOWER-Cervical 1 | Cervical cancer | 2022 | Immunotherapy | Metastatic | 3 | 608 | OS: median 12 vs 8.5 months, HR 0.69 (95% CI 0.56–0.84)Milestone: EMPOWER-Cervical 1: cemiplimab versus chemotherapy after platinum |
| EPCORE FL-1 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2022 | Immunotherapy | Relapsed / refractory | 3 | 488 | PFS: 85.5% vs 40.2% at 16 mo, HR 0.21 (95% CI 0.14–0.31)Milestone: Mosunetuzumab and epcoritamab in relapsed follicular lymphoma |
| EPCORE NHL-1 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2022 | Immunotherapy | Relapsed / refractory | 1/2 | — | ORR: see trialMilestone: Mosunetuzumab and epcoritamab in relapsed follicular lymphoma |
| EPCORE NHL-1 | Non-Hodgkin lymphoma (DLBCL and follicular) | 2022 | Immunotherapy | Relapsed / refractory | 1/2 | 157 | ORR: see trialMilestone: Glofitamab and epcoritamab in relapsed DLBCL (2022-2023) |
| ESTIMABL2 | Thyroid cancer | 2022 | Surgery, Radiation | Adjuvant | 3 | 776 | Composite event-free rate: 95.6% vs 95.9%Milestone: ESTIMABL2 and IoN: low-risk thyroid cancer without radioiodine |
| GAIA | Chronic lymphocytic leukaemia | 2022 | Targeted, Immunotherapy | Metastatic | 3 | 926 | PFS: 90.5% vs 75.5% at 36 mo, HR 0.32Milestone: Fixed-duration combinations: GLOW and CLL13 (GAIA) |
| glofitamab-phase-1-2-dlbcl | Non-Hodgkin lymphoma (DLBCL and follicular) | 2022 | Immunotherapy | Relapsed / refractory | 1/2 | 155 | CR rate: see trialMilestone: Glofitamab and epcoritamab in relapsed DLBCL (2022-2023) |
| GLOW | Chronic lymphocytic leukaemia | 2022 | Targeted, Immunotherapy | Metastatic | 3 | 211 | PFS: HR 0.216 (95% CI 0.131–0.357)Milestone: Fixed-duration combinations: GLOW and CLL13 (GAIA) |
| GOG 281/LOGS | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 2/3 | 260 | PFS: median 13 vs 7.2 months, HR 0.48 (95% CI 0.36–0.64)Milestone: Low-grade serous disease: MEK inhibition (GOG 281, RAMP 201) |
| HIMALAYA | Hepatocellular carcinoma | 2022 | Immunotherapy | Metastatic | 3 | 1,171 | OS: median 16.43 vs 13.77 months, HR 0.78Milestone: HIMALAYA: tremelimumab plus durvalumab (STRIDE) versus sorafenib |
| IMmotion010 | Kidney cancer (renal cell carcinoma) | 2022 | Immunotherapy | Adjuvant, Perioperative | 3 | 778 | DFS: median 57.2 vs 49.5 months, HR 0.93 (95% CI 0.75–1.15)Milestone: Adjuvant checkpoint trials: IMmotion010, CheckMate 914, PROSPER |
| INDUCE-3 | Head and neck cancer | 2022 | Immunotherapy, Targeted | Metastatic | 2/3 | 315 | OS: HR 1.51 (95% CI 0.99–2.29)Milestone: First-line immunotherapy combinations: no overall survival gain |
| IoN | Thyroid cancer | 2022 | Surgery, Radiation | Adjuvant | 3 | 504 | RFS: 97.9% vs 96.3% at 60 moMilestone: ESTIMABL2 and IoN: low-risk thyroid cancer without radioiodine |
| JAVELIN Gastric 100 | Gastric and oesophageal cancer | 2022 | Immunotherapy | Metastatic | 3 | 499 | OS: median 10.4 vs 10.9 months, HR 0.91 (95% CI 0.74–1.11)Milestone: Later-line pembrolizumab in gastric cancer: approved, then withdrawn |
| JCOG0802/WJOG4607L | Lung cancer | 2022 | Surgery | Localised | 3 | 1,106 | OS: 94.3% vs 91.1% at 60 mo, HR 0.663 (95% CI 0.474–0.927)Milestone: Segmentectomy and sublobar resection vs lobectomy (2022-2023) |
| KESTREL | Head and neck cancer | 2022 | Immunotherapy, Targeted | Metastatic | 3 | — | OS: median 10.9 vs 10.9 months, HR 0.96 (95% CI 0.69–1.32)Milestone: First-line immunotherapy combinations: no overall survival gain |
| KEYNOTE-059 | Gastric and oesophageal cancer | 2022 | Immunotherapy | Metastatic | 2 | 259 | ORR: see trialMilestone: Later-line pembrolizumab in gastric cancer: approved, then withdrawn |
| KEYNOTE-061 | Gastric and oesophageal cancer | 2022 | Immunotherapy | Metastatic | 3 | 592 | OS: median 9.1 vs 8.3 months, HR 0.82 (95% CI 0.66–1.03)Milestone: Later-line pembrolizumab in gastric cancer: approved, then withdrawn |
| KRYSTAL-1 | Colorectal cancer | 2022 | Targeted | Metastatic, Relapsed / refractory | 1/2 | — | ORR: see trialMilestone: KRAS G12C inhibitors with anti-EGFR antibodies |
| KRYSTAL-1 | Pancreatic cancer | 2022 | Targeted | Metastatic, Relapsed / refractory | 1/2 | 63 | ORR: see trialMilestone: KRAS G12C inhibitors in the small G12C-mutated subset |
| LEAP-010 | Head and neck cancer | 2022 | Immunotherapy, Targeted | Metastatic | 3 | 511 | PFS: median 6.2 vs 2.8 months, HR 0.64 (95% CI 0.50–0.81)Milestone: First-line immunotherapy combinations: no overall survival gain |
| LINKER-MM1 | Multiple myeloma | 2022 | Immunotherapy | Relapsed / refractory | 1/2 | — | ORR: see trialMilestone: Bispecifics: teclistamab, talquetamab, elranatamab, linvoseltamab |
| MagnetisMM-3 | Multiple myeloma | 2022 | Immunotherapy | Relapsed / refractory | 2 | 123 | ORR: see trialMilestone: Bispecifics: teclistamab, talquetamab, elranatamab, linvoseltamab |
| MAGNITUDE | Prostate cancer | 2022 | Targeted, Endocrine | Metastatic | 3 | — | rPFS: median 16.6 vs 10.9 months, HR 0.53 (95% CI 0.36–0.79)Milestone: PARP inhibitor plus ARPI first line (PROpel, MAGNITUDE, TALAPRO-2) |
| MajesTEC-1 | Multiple myeloma | 2022 | Immunotherapy | Relapsed / refractory | 1/2 | 165 | ORR: see trialMilestone: Bispecifics: teclistamab, talquetamab, elranatamab, linvoseltamab |
| MILO/ENGOT-ov11 | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 3 | — | PFS: median 9.1 vs 10.6 months, HR 1.21 (95% CI 0.79–1.86)Milestone: Low-grade serous disease: MEK inhibition (GOG 281, RAMP 201) |
| MIRASOL | Ovarian cancer | 2022 | Antibody–drug conjugate | Relapsed / refractory | 3 | 453 | OS: median 16.46 vs 12.75 months, HR 0.67 (95% CI 0.50–0.89)Milestone: Mirvetuximab soravtansine for folate receptor-high disease |
| MonumenTAL-1 | Multiple myeloma | 2022 | Immunotherapy | Relapsed / refractory | 1 | 232 | ORR: see trialMilestone: Bispecifics: teclistamab, talquetamab, elranatamab, linvoseltamab |
| mosunetuzumab-go29781-fl | Non-Hodgkin lymphoma (DLBCL and follicular) | 2022 | Immunotherapy | Relapsed / refractory | 2 | 90 | CR rate: see trialMilestone: Mosunetuzumab and epcoritamab in relapsed follicular lymphoma |
| nki-til-vs-ipilimumab | Melanoma | 2022 | Cell therapy & transplant | Metastatic | 3 | 168 | PFS: median 7.2 vs 3.1 months, HR 0.50 (95% CI 0.35–0.72)Milestone: TIL therapy versus ipilimumab: randomised phase 3 trial (2022) |
| NordICC | Colorectal cancer | 2022 | Detection & prevention | Screening | — | 84,585 | Colorectal cancer incidence: 0.98% vs 1.2%Milestone: NordICC: invitation to screening colonoscopy |
| OPRA | Colorectal cancer | 2022 | Chemotherapy, Radiation | Neoadjuvant, Locally advanced | 2 | 324 | DFS: see trialMilestone: OPRA: total neoadjuvant therapy and selective watch-and-wait |
| PEACE-1 | Prostate cancer | 2022 | Endocrine, Chemotherapy | Metastatic | 3 | 1,173 | OS: HR 0.75Milestone: Triplet therapy: ADT, docetaxel and an AR inhibitor (ARASENS, PEACE-1) |
| PRIMETEST | Testicular germ cell tumors | 2022 | Surgery | Localised | 2 | 33 | Relapse rate: see trialMilestone: Primary RPLND for stage IIA/B seminoma: PRIMETEST |
| PROpel | Prostate cancer | 2022 | Targeted, Endocrine | Metastatic | 3 | — | rPFS: median 24.8 vs 16.6 months, HR 0.66 (95% CI 0.54–0.81)Milestone: PARP inhibitor plus ARPI first line (PROpel, MAGNITUDE, TALAPRO-2) |
| PROSPER EA8143⚑ needs review | Kidney cancer (renal cell carcinoma) | 2022 | Immunotherapy | Adjuvant, Perioperative | 3 | 819 | RFS: HR 0.94 (95% CI 0.74–1.21)Milestone: Adjuvant checkpoint trials: IMmotion010, CheckMate 914, PROSPER |
| QUADRA | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 2 | 463 | ORR: see trialMilestone: PARP inhibitor later-line ovarian indications withdrawn (2022) |
| QUILT-3.032 | Bladder cancer | 2022 | Immunotherapy | Localised | 2/3 | — | Complete response in carcinoma in situ: see trialMilestone: N-803 plus BCG in BCG-unresponsive NMIBC |
| RAMP 201 | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 2 | 115 | ORR: see trialMilestone: Low-grade serous disease: MEK inhibition (GOG 281, RAMP 201) |
| RELATIVITY-047 | Melanoma | 2022 | Immunotherapy | Metastatic | 2/3 | 714 | PFS: median 10.1 vs 4.6 months, HR 0.75 (95% CI 0.62–0.92)Milestone: Relatlimab plus nivolumab: RELATIVITY-047 and LAG-3 blockade (2022) |
| SECOMBIT | Melanoma | 2022 | Immunotherapy, Targeted | Metastatic | 2 | 209 | OS: see trialMilestone: Sequencing in BRAF-mutant disease: DREAMseq, SECOMBIT and EBIN |
| SEQUOIA | Chronic lymphocytic leukaemia | 2022 | Targeted | Metastatic, Relapsed / refractory | 3 | 590 | PFS: HR 0.42 (95% CI 0.28–0.63)Milestone: Zanubrutinib: SEQUOIA and the ALPINE head-to-head with ibrutinib |
| SOLO3 | Ovarian cancer | 2022 | Targeted | Relapsed / refractory | 3 | 266 | PFS: median 13.4 vs 9.2 months, HR 0.62 (95% CI 0.43–0.91)Milestone: PARP inhibitor later-line ovarian indications withdrawn (2022) |
| SORAYA | Ovarian cancer | 2022 | Antibody–drug conjugate | Relapsed / refractory | 2 | 106 | ORR: see trialMilestone: Mirvetuximab soravtansine for folate receptor-high disease |
| TALAPRO-2 | Prostate cancer | 2022 | Targeted, Endocrine | Metastatic | 3 | — | rPFS: HR 0.63 (95% CI 0.51–0.78)Milestone: PARP inhibitor plus ARPI first line (PROpel, MAGNITUDE, TALAPRO-2) |
| TRISST | Testicular germ cell tumors | 2022 | Understanding | Localised | 3 | 669 | Stage IIC or higher relapse incidence: 2.8% vs 0.3%Milestone: TRISST: fewer scans and MRI instead of CT in seminoma surveillance |
| AEGEAN | Lung cancer | 2023 | Immunotherapy, Chemotherapy, Surgery | Perioperative | 3 | 802 | EFS: 73.4% vs 64.5% at 12 mo, HR 0.68 (95% CI 0.53–0.88)Milestone: Perioperative chemo-immunotherapy: KEYNOTE-671, AEGEAN, CheckMate 77T |
| ARROW | Thyroid cancer | 2023 | Targeted | Metastatic | 1/2 | — | ORR: see trialMilestone: Pralsetinib in RET thyroid cancer; MTC indication withdrawn 2023 |
| AUGMENT-101 | Acute myeloid leukaemia (including APL) | 2023 | Targeted | Relapsed / refractory | 1/2 | — | CR+CRh: see trialMilestone: Menin inhibitors: revumenib and ziftomenib in KMT2A or NPM1 leukemia |
| BEATcc | Cervical cancer | 2023 | Immunotherapy, Targeted, Chemotherapy | Metastatic | 3 | 410 | PFS: median 13.7 vs 10.4 months, HR 0.62 (95% CI 0.49–0.78)Milestone: BEATcc and COMPASSION-16: atezolizumab, cadonilimab |
| blinatumomab-infants-kmt2a | Childhood acute lymphoblastic leukemia (ALL) | 2023 | Immunotherapy, Chemotherapy | Any setting | — | 30 | DFS: 81.6% vs 49.4% at 24 moMilestone: Blinatumomab added to chemotherapy in infant ALL |
| BRUIN | Chronic lymphocytic leukaemia | 2023 | Targeted | Relapsed / refractory, Metastatic | 1/2 | 317 | ORR: see trialMilestone: Pirtobrutinib: non-covalent BTK inhibitor after covalent BTK failure |
| BRUIN CLL-313 | Chronic lymphocytic leukaemia | 2023 | Targeted | Relapsed / refractory, Metastatic | 3 | 282 | PFS: 93.4% vs 70.7% at 24 mo, HR 0.199 (95% CI 0.107–0.367)Milestone: Pirtobrutinib: non-covalent BTK inhibitor after covalent BTK failure |
| BRUIN CLL-321⚑ needs review | Chronic lymphocytic leukaemia | 2023 | Targeted | Relapsed / refractory, Metastatic | 3 | 238 | PFS: HR 0.58 (95% CI 0.38–0.89)Milestone: Pirtobrutinib: non-covalent BTK inhibitor after covalent BTK failure |
| CALLA | Cervical cancer | 2023 | Chemotherapy, Immunotherapy, Radiation | Locally advanced | 3 | 770 | PFS: 76% vs 73.3% at 12 mo, HR 0.84 (95% CI 0.65–1.08)Milestone: OUTBACK and CALLA: adjuvant chemotherapy and durvalumab add-ons |
| CheckMate 77T | Lung cancer | 2023 | Immunotherapy, Chemotherapy, Surgery | Perioperative | 3 | — | EFS: 70.2% vs 50% at 18 mo, HR 0.58Milestone: Perioperative chemo-immunotherapy: KEYNOTE-671, AEGEAN, CheckMate 77T |
| COMPASSION-16 | Cervical cancer | 2023 | Immunotherapy, Targeted, Chemotherapy | Metastatic | 3 | 445 | PFS: median 12.7 vs 8.1 months, HR 0.62 (95% CI 0.49–0.80)Milestone: BEATcc and COMPASSION-16: atezolizumab, cadonilimab |
| DeLLphi-301 | Lung cancer | 2023 | Immunotherapy | Relapsed / refractory | 2 | 220 | ORR: see trialMilestone: Tarlatamab: DLL3 T-cell engager for previously treated SCLC |
| DeLLphi-304 | Lung cancer | 2023 | Immunotherapy | Relapsed / refractory | 3 | 509 | OS: median 13.6 vs 8.3 months, HR 0.60 (95% CI 0.47–0.77)Milestone: Tarlatamab: DLL3 T-cell engager for previously treated SCLC |
| DREAMM-2 | Multiple myeloma | 2023 | Antibody–drug conjugate | Relapsed / refractory | 2 | 196 | ORR: see trialMilestone: Belantamab mafodotin: withdrawn in 2023, approved again in 2025 |
| DREAMM-3 | Multiple myeloma | 2023 | Antibody–drug conjugate | Relapsed / refractory | 3 | 325 | PFS: median 11.2 vs 7 months, HR 1.03 (95% CI 0.72–1.47)Milestone: Belantamab mafodotin: withdrawn in 2023, approved again in 2025 |
| DREAMM-7 | Multiple myeloma | 2023 | Antibody–drug conjugate | Relapsed / refractory | 3 | 494 | PFS: median 36.6 vs 13.4 months, HR 0.41 (95% CI 0.31–0.53)Milestone: Belantamab mafodotin: withdrawn in 2023, approved again in 2025 |
| DREAMM-8 | Multiple myeloma | 2023 | Antibody–drug conjugate | Relapsed / refractory | 3 | 302 | PFS: 71% vs 51% at 12 mo, HR 0.52 (95% CI 0.37–0.73)Milestone: Belantamab mafodotin: withdrawn in 2023, approved again in 2025 |
| DUO-E | Endometrial cancer | 2023 | Immunotherapy, Chemotherapy, Targeted | Metastatic | 3 | 718 | PFS: median 10.2 vs 9.6 months, HR 0.71 (95% CI 0.57–0.89)Milestone: DUO-E: durvalumab with or without olaparib, first line |
| EMBARK | Prostate cancer | 2023 | Endocrine | Any setting | 3 | 1,068 | MFS: 87.3% vs 71.4% at 60 mo, HR 0.42 (95% CI 0.30–0.61)Milestone: Enzalutamide for high-risk biochemical recurrence (EMBARK) |
| EV-103 | Bladder cancer | 2023 | Antibody–drug conjugate, Immunotherapy | Metastatic | 1/2 | — | ORR: 64.5% vs 45.2%Milestone: EV-302: enfortumab vedotin plus pembrolizumab as first-line |
| EV-302 | Bladder cancer | 2023 | Antibody–drug conjugate, Immunotherapy | Metastatic | 3 | 886 | PFS: median 12.5 vs 6.3 months, HR 0.45 (95% CI 0.38–0.54)Milestone: EV-302: enfortumab vedotin plus pembrolizumab as first-line |
| FLAURA2 | Lung cancer | 2023 | Targeted, Chemotherapy, Immunotherapy | Metastatic | 3 | 557 | PFS: 57% vs 41% at 24 mo, HR 0.62 (95% CI 0.49–0.79)Milestone: First-line EGFR combinations: FLAURA2 and MARIPOSA |
| FOxTROT | Colorectal cancer | 2023 | Chemotherapy, Surgery | Neoadjuvant, Perioperative | 2/3 | 1,053 | Residual disease or recurrence within 2 years: 16.9% vs 21.5%Milestone: FOxTROT: neoadjuvant chemotherapy for locally advanced colon cancer |
| GLOW | Gastric and oesophageal cancer | 2023 | Targeted | Metastatic | 3 | 507 | PFS: median 8.21 vs 6.8 months, HR 0.687 (95% CI 0.544–0.866)Milestone: Zolbetuximab for CLDN18.2-positive disease: SPOTLIGHT and GLOW |
| INDIGO | Glioblastoma | 2023 | Targeted | Adjuvant | 3 | 331 | PFS: median 27.7 vs 11.1 months, HR 0.39 (95% CI 0.27–0.56)Milestone: Vorasidenib delays progression in IDH-mutant grade 2 glioma (INDIGO) |
| KEYNOTE-671 | Lung cancer | 2023 | Immunotherapy, Chemotherapy, Surgery | Perioperative | 3 | 797 | EFS: 62.4% vs 40.6% at 24 mo, HR 0.58 (95% CI 0.46–0.72)Milestone: Perioperative chemo-immunotherapy: KEYNOTE-671, AEGEAN, CheckMate 77T |
| KOMET-001 | Acute myeloid leukaemia (including APL) | 2023 | Targeted | Relapsed / refractory | 1/2 | — | CR+CRh: see trialMilestone: Menin inhibitors: revumenib and ziftomenib in KMT2A or NPM1 leukemia |
| LIBRETTO-531 | Thyroid cancer | 2023 | Targeted | Metastatic | 3 | 291 | PFS: 86.8% vs 65.7% at 12 mo, HR 0.28 (95% CI 0.16–0.48)Milestone: LIBRETTO-531: selpercatinib versus cabozantinib or vandetanib |
| LITESPARK-005⚑ needs review | Kidney cancer (renal cell carcinoma) | 2023 | Targeted | Metastatic | 3 | 746 | PFS: median 5.6 vs 5.6 monthsMilestone: LITESPARK-005: belzutifan versus everolimus after ICI and TKI (2023) |
| MARIPOSA | Lung cancer | 2023 | Targeted, Chemotherapy, Immunotherapy | Metastatic | 3 | 1,074 | PFS: median 23.7 vs 16.6 months, HR 0.70 (95% CI 0.58–0.85)Milestone: First-line EGFR combinations: FLAURA2 and MARIPOSA |
| NAPOLI 3 | Pancreatic cancer | 2023 | Chemotherapy | Metastatic | 3 | 770 | OS: median 11.1 vs 9.2 months, HR 0.83 (95% CI 0.70–0.99)Milestone: NAPOLI-3: NALIRIFOX versus nab-paclitaxel plus gemcitabine |
| NMTRC003B | Neuroblastoma and Wilms tumour | 2023 | Targeted | Maintenance | 2 | 141 | EFS: see trialMilestone: Eflornithine maintenance to reduce relapse: FDA approval (2023) |
| NRG-GY018 | Endometrial cancer | 2023 | Immunotherapy, Chemotherapy | Metastatic | 3 | 816 | PFS: 74% vs 38% at 12 mo, HR 0.30 (95% CI 0.19–0.48)Milestone: NRG-GY018: pembrolizumab added to chemotherapy, both MMR cohorts |
| NRG/RTOG 0912 | Thyroid cancer | 2023 | Targeted, Radiation, Chemotherapy | Locally advanced | 2 | 89 | OS: median 5.7 vs 7.3 months, HR 0.86 (95% CI 0.52–1.43)Milestone: NRG/RTOG 0912: pazopanib plus chemoradiotherapy in anaplastic cancer |
| OUTBACK | Cervical cancer | 2023 | Chemotherapy, Immunotherapy, Radiation | Locally advanced | 3 | 926 | OS: 72% vs 71% at 60 mo, HR 0.90 (95% CI 0.70–1.17)Milestone: OUTBACK and CALLA: adjuvant chemotherapy and durvalumab add-ons |
| PROSPECT | Colorectal cancer | 2023 | Chemotherapy, Radiation, Surgery | Neoadjuvant | 2/3 | 1,194 | DFS: 80.8% vs 78.6% at 60 mo, HR 0.92Milestone: PROSPECT: neoadjuvant FOLFOX with selective chemoradiotherapy |
| QuANTUM-First | Acute myeloid leukaemia (including APL) | 2023 | Targeted | Any setting | 3 | 539 | OS: median 31.9 vs 15.1 months, HR 0.78 (95% CI 0.62–0.98)Milestone: QuANTUM-First: quizartinib with chemotherapy in FLT3-ITD AML |
| RUBY | Endometrial cancer | 2023 | Immunotherapy, Chemotherapy | Metastatic | 3 | 494 | PFS: 61.4% vs 15.7% at 24 mo, HR 0.28 (95% CI 0.16–0.50)Milestone: RUBY: dostarlimab added to carboplatin-paclitaxel, first line |
| SPOTLIGHT | Gastric and oesophageal cancer | 2023 | Targeted | Metastatic | 3 | 565 | PFS: median 10.61 vs 8.67 months, HR 0.75 (95% CI 0.60–0.94)Milestone: Zolbetuximab for CLDN18.2-positive disease: SPOTLIGHT and GLOW |
| SWOG S1801 | Melanoma | 2023 | Immunotherapy, Surgery | Neoadjuvant, Perioperative | 2 | 313 | EFS: 72% vs 49% at 24 moMilestone: SWOG S1801: neoadjuvant-adjuvant versus adjuvant-only pembrolizumab |
| AALL1731 | Childhood acute lymphoblastic leukemia (ALL) | 2024 | Immunotherapy, Chemotherapy | Any setting | 3 | — | DFS: 96% vs 87.9% at 36 moMilestone: Blinatumomab added to chemotherapy in standard-risk B-ALL |
| ADRIATIC | Lung cancer | 2024 | Immunotherapy | Locally advanced, Maintenance | 3 | — | OS: median 55.9 vs 33.4 months, HR 0.73Milestone: ADRIATIC: durvalumab after chemoradiotherapy, limited-stage SCLC |
| ALINA | Lung cancer | 2024 | Targeted | Adjuvant | 3 | 257 | DFS: 93.8% vs 63% at 24 mo, HR 0.24 (95% CI 0.13–0.45)Milestone: ALINA: adjuvant alectinib in resected ALK-positive disease |
| ARANOTE | Prostate cancer | 2024 | Endocrine | Metastatic | 3 | 669 | rPFS: HR 0.54 (95% CI 0.41–0.71)Milestone: Darolutamide plus ADT without docetaxel (ARANOTE) |
| ASC4FIRST | Chronic myeloid leukemia | 2024 | Targeted | Any setting | 3 | 405 | MMR: 67.7% vs 49%Milestone: ASC4FIRST: asciminib in newly diagnosed chronic-phase CML |
| BREAKWATER | Colorectal cancer | 2024 | Targeted, Chemotherapy | Metastatic | 3 | — | PFS: median 12.8 vs 7.1 months, HR 0.53 (95% CI 0.41–0.68)Milestone: BREAKWATER: first-line encorafenib, cetuximab and chemotherapy |
| CAP | Prostate cancer | 2024 | Detection & prevention | Screening | — | 415,357 | Cumulative incidence of prostate cancer: 4.3% vs 3.6%Milestone: Screening follow-up: CAP (15-year) and ERSPC (23-year) |
| CheckMate 8HW | Colorectal cancer | 2024 | Immunotherapy | Metastatic | 3 | — | PFS: HR 0.62 (95% CI 0.48–0.81)Milestone: CheckMate 8HW: nivolumab plus ipilimumab in MSI-H/dMMR mCRC |
| eNRGy | Pancreatic cancer | 2024 | Targeted | Metastatic, Relapsed / refractory | 2 | 204 | ORR: see trialMilestone: Zenocutuzumab: pancreatic-cancer indication for NRG1 gene fusions |
| HD21 | Hodgkin lymphoma | 2024 | Antibody–drug conjugate, Chemotherapy | Locally advanced, Metastatic | 3 | 1,500 | PFS: 94.3% vs 90.9% at 48 mo, HR 0.66 (95% CI 0.45–0.97)Milestone: HD21: BrECADD versus escalated BEACOPP |
| INTERLACE | Cervical cancer | 2024 | Chemotherapy, Radiation | Locally advanced | 3 | 500 | PFS: 72% vs 64% at 60 mo, HR 0.65 (95% CI 0.46–0.91)Milestone: INTERLACE: weekly induction chemotherapy before chemoradiation |
| KEYNOTE-942 | Melanoma | 2024 | Immunotherapy | Adjuvant | 2 | 157 | RFS: 79% vs 62% at 18 mo, HR 0.561 (95% CI 0.309–1.017)Milestone: Individualised mRNA neoantigen therapy plus pembrolizumab: KEYNOTE-942 |
| KEYNOTE-A18 | Cervical cancer | 2024 | Immunotherapy, Radiation, Chemotherapy | Locally advanced | 3 | 1,060 | PFS: 68% vs 57% at 24 mo, HR 0.70 (95% CI 0.55–0.89)Milestone: KEYNOTE-A18: pembrolizumab added to chemoradiation (FDA Jan 2024) |
| KEYNOTE-B21 | Endometrial cancer | 2024 | Immunotherapy, Chemotherapy | Adjuvant | 3 | 1,095 | DFS: 75% vs 76% at 24 mo, HR 1.02 (95% CI 0.79–1.32)Milestone: KEYNOTE-B21: adjuvant pembrolizumab, no DFS gain overall |
| LAURA | Lung cancer | 2024 | Targeted, Radiation | Locally advanced | 3 | 216 | PFS: median 39.1 vs 5.6 months, HR 0.16 (95% CI 0.10–0.24)Milestone: LAURA: osimertinib after chemoradiotherapy, stage III |
| NADINA | Melanoma | 2024 | Immunotherapy, Surgery | Neoadjuvant | 3 | 423 | EFS: 83.7% vs 57.2% at 12 mo, HR 0.32Milestone: NADINA: neoadjuvant ipilimumab plus nivolumab in stage III (2024) |
| NIAGARA | Bladder cancer | 2024 | Immunotherapy, Chemotherapy | Perioperative | 3 | 1,063 | EFS: 67.8% vs 59.8% at 24 mo, HR 0.68 (95% CI 0.56–0.82)Milestone: NIAGARA: durvalumab added to neoadjuvant gemcitabine-cisplatin |
| NICHE-2 | Colorectal cancer | 2024 | Immunotherapy, Surgery | Neoadjuvant | 2 | 115 | Timely surgery: see trialMilestone: NICHE-2: neoadjuvant nivolumab plus ipilimumab for dMMR colon cancer |
| NORPACT-1 | Pancreatic cancer | 2024 | Chemotherapy, Surgery | Neoadjuvant | 2 | 140 | OS: median 25.1 vs 38.5 months, HR 1.52 (95% CI 1.00–2.33)Milestone: NORPACT-1: neoadjuvant FOLFIRINOX versus upfront surgery |
| onc201-integrated-analysis-h3-k27m-dmg | Glioblastoma | 2024 | Targeted | Relapsed / refractory | — | 50 | ORR: see trialMilestone: Dordaviprone gets accelerated approval for H3 K27M midline glioma |
| PSMAfore | Prostate cancer | 2024 | Radiation | Metastatic | 3 | 468 | rPFS: median 9.3 vs 5.55 months, HR 0.41 (95% CI 0.29–0.56)Milestone: 177Lu-PSMA-617 before taxane chemotherapy (PSMAfore) |
| S1826 | Hodgkin lymphoma | 2024 | Immunotherapy, Chemotherapy | Locally advanced, Metastatic | 3 | 994 | PFS: HR 0.48Milestone: S1826: nivolumab plus AVD versus BV plus AVD |
| SHAPE | Cervical cancer | 2024 | Surgery | Localised | — | 700 | Pelvic recurrence: 2.52% vs 2.17% at 36 moMilestone: SHAPE: simple hysterectomy for low-risk early cervical cancer |
| SU2C-SARC032 | Sarcoma and GIST | 2024 | Immunotherapy, Radiation, Surgery | Neoadjuvant, Adjuvant | 2 | 143 | DFS: 67% vs 52% at 24 mo, HR 0.61Milestone: SU2C-SARC032: pembrolizumab added to radiotherapy before surgery |
| TROPHY-U-01 | Bladder cancer | 2024 | Antibody–drug conjugate | Metastatic | 2 | — | ORR: see trialMilestone: Sacituzumab govitecan urothelial indication withdrawn |
| TROPiCS-04 | Bladder cancer | 2024 | Antibody–drug conjugate | Metastatic | 3 | 711 | OS: median 10.3 vs 9 months, HR 0.86 (95% CI 0.73–1.02)Milestone: Sacituzumab govitecan urothelial indication withdrawn |
| AMPLIFY | Chronic lymphocytic leukaemia | 2025 | Targeted | Metastatic | 3 | 867 | PFS: 76.5% vs 66.5% at 36 mo, HR 0.65 (95% CI 0.49–0.87)Milestone: AMPLIFY: fixed-duration acalabrutinib-venetoclax vs chemoimmunotherapy |
| AMPLITUDE | Prostate cancer | 2025 | Targeted, Endocrine | Metastatic | 3 | 696 | rPFS: HR 0.52 (95% CI 0.37–0.72)Milestone: Biomarker-selected mHSPC: AMPLITUDE (BRCA) and CAPItello-281 (PTEN) |
| ASCENT-03 | Breast cancer | 2025 | Antibody–drug conjugate, Immunotherapy | Metastatic | 3 | 558 | PFS: median 9.7 vs 6.9 months, HR 0.62 (95% CI 0.50–0.77)Milestone: ADCs reach first-line metastatic TNBC (ASCENT-03/-04, TROPION-02) |
| ASCENT-04 | Breast cancer | 2025 | Antibody–drug conjugate, Immunotherapy | Metastatic | 3 | 443 | PFS: median 11.2 vs 7.8 months, HR 0.65 (95% CI 0.51–0.84)Milestone: ADCs reach first-line metastatic TNBC (ASCENT-03/-04, TROPION-02) |
| CAPItello-281 | Prostate cancer | 2025 | Targeted, Endocrine | Metastatic | 3 | 1,012 | rPFS: median 33.2 vs 25.7 months, HR 0.81 (95% CI 0.66–0.98)Milestone: Biomarker-selected mHSPC: AMPLITUDE (BRCA) and CAPItello-281 (PTEN) |
| CheckMate 9DW | Hepatocellular carcinoma | 2025 | Immunotherapy | Metastatic | 3 | 668 | OS: median 23.7 vs 20.6 months, HR 0.79 (95% CI 0.65–0.96)Milestone: CheckMate 9DW: first-line nivolumab plus ipilimumab |
| DESTINY-Breast09 | Breast cancer | 2025 | Antibody–drug conjugate, Targeted | Metastatic | 3 | 1,157 | PFS: median 40.7 vs 26.9 months, HR 0.56 (95% CI 0.44–0.71)Milestone: DESTINY-Breast09: T-DXd plus pertuzumab in first-line therapy |
| EMERALD-1 | Hepatocellular carcinoma | 2025 | Immunotherapy, Targeted, Surgery | Locally advanced | 3 | 616 | PFS: median 15 vs 8.2 months, HR 0.77 (95% CI 0.61–0.98)Milestone: TACE plus immunotherapy: EMERALD-1 and LEAP-012 (PFS) |
| ESOPEC | Gastric and oesophageal cancer | 2025 | Chemotherapy, Radiation, Surgery | Neoadjuvant, Perioperative | 3 | 438 | OS: 57.4% vs 50.7% at 36 mo, HR 0.70 (95% CI 0.53–0.92)Milestone: ESOPEC: perioperative FLOT versus CROSS in adenocarcinoma |
| IGNYTE | Melanoma | 2025 | Immunotherapy | Metastatic, Relapsed / refractory | 2 | 140 | ORR: see trialMilestone: RP1 plus nivolumab after anti-PD-1 failure: IGNYTE and FDA approval |
| KEYNOTE-585⚑ needs review | Gastric and oesophageal cancer | 2025 | Immunotherapy, Chemotherapy, Surgery | Perioperative | 3 | 1,007 | OS: median 71.8 vs 55.7 months, HR 0.86 (95% CI 0.71–1.06)Milestone: MATTERHORN: perioperative durvalumab plus FLOT, and KEYNOTE-585 |
| KEYNOTE-689 | Head and neck cancer | 2025 | Immunotherapy, Surgery, Radiation | Perioperative | 3 | 714 | EFS: 59.8% vs 45.9% at 36 mo, HR 0.66 (95% CI 0.49–0.88)Milestone: KEYNOTE-689: perioperative pembrolizumab for resectable disease |
| KEYNOTE-905/EV-303 | Bladder cancer | 2025 | Antibody–drug conjugate, Immunotherapy | Perioperative | 3 | 344 | EFS: 74.7% vs 39.4% at 24 mo, HR 0.40 (95% CI 0.28–0.57)Milestone: KEYNOTE-905/EV-303: EV plus pembrolizumab around cystectomy |
| LEAP-012 | Hepatocellular carcinoma | 2025 | Immunotherapy, Targeted, Surgery | Locally advanced | 3 | 480 | PFS: median 14.6 vs 10 months, HR 0.66 (95% CI 0.51–0.84)Milestone: TACE plus immunotherapy: EMERALD-1 and LEAP-012 (PFS) |
| MajesTEC-3 | Multiple myeloma | 2025 | Immunotherapy | Relapsed / refractory | 3 | 587 | PFS: 83.4% vs 29.7% at 36 mo, HR 0.17 (95% CI 0.12–0.23)Milestone: Bispecifics move earlier: MajesTEC-3, MajesTEC-9, MonumenTAL-3 |
| MajesTEC-9 | Multiple myeloma | 2025 | Immunotherapy | Relapsed / refractory | 3 | 593 | PFS: 69.8% vs 26.9% at 18 mo, HR 0.29 (95% CI 0.23–0.38)Milestone: Bispecifics move earlier: MajesTEC-3, MajesTEC-9, MonumenTAL-3 |
| MATTERHORN | Gastric and oesophageal cancer | 2025 | Immunotherapy, Chemotherapy, Surgery | Perioperative | 3 | 948 | EFS: 67.4% vs 58.5% at 24 mo, HR 0.71 (95% CI 0.58–0.86)Milestone: MATTERHORN: perioperative durvalumab plus FLOT, and KEYNOTE-585 |
| MonumenTAL-3 | Multiple myeloma | 2025 | Immunotherapy | Relapsed / refractory | 3 | 864 | PFS: 81.3% vs 51.2% at 24 mo, HR 0.28 (95% CI 0.2–0.4)Milestone: Bispecifics move earlier: MajesTEC-3, MajesTEC-9, MonumenTAL-3 |
| Neo-AEGIS | Gastric and oesophageal cancer | 2025 | Chemotherapy, Radiation, Surgery | Neoadjuvant, Perioperative | 3 | 377 | OS: median 49.2 vs 48 months, HR 1.03 (95% CI 0.77–1.38)Milestone: ESOPEC: perioperative FLOT versus CROSS in adenocarcinoma |
| NIVOPOSTOP | Head and neck cancer | 2025 | Immunotherapy, Radiation, Chemotherapy | Adjuvant | 3 | 680 | DFS: HR 0.76 (95% CI 0.60–0.98)Milestone: NIVOPOSTOP: nivolumab added to postoperative cisplatin-radiotherapy |
| PANOVA-3 | Pancreatic cancer | 2025 | Targeted | Locally advanced | 3 | 571 | OS: median 16.2 vs 14.2 months, HR 0.82 (95% CI 0.68–0.99)Milestone: PANOVA-3: tumour treating fields plus chemotherapy, locally advanced |
| PORTEC-4a | Endometrial cancer | 2025 | Radiation, Understanding | Adjuvant | 3 | 569 | Vaginal recurrence: 4.5% vs 1.6% at 60 mo, HR 2.71 (95% CI 0.79–9.34)Milestone: PORTEC-4a: molecular profile guides adjuvant treatment |
| PREOPANC-2 | Pancreatic cancer | 2025 | Chemotherapy, Radiation, Surgery | Neoadjuvant | 3 | 375 | OS: median 21.9 vs 21.3 months, HR 0.88 (95% CI 0.69–1.13)Milestone: PREOPANC-2: neoadjuvant FOLFIRINOX versus chemoradiotherapy |
| ROSELLA/GOG-3073/ENGOT-ov72 | Ovarian cancer | 2025 | Targeted, Chemotherapy | Relapsed / refractory | 3 | 381 | PFS: median 6.54 vs 5.52 months, HR 0.70 (95% CI 0.54–0.91)Milestone: Relacorilant plus nab-paclitaxel, platinum-resistant: ROSELLA |
| SANO | Gastric and oesophageal cancer | 2025 | Surgery, Chemotherapy, Radiation | Neoadjuvant | 3 | 309 | OS: 74% vs 71% at 24 mo, HR 1.14 (95% CI 0.74–1.78)Milestone: SANO: active surveillance versus surgery after complete response |
| SunRISe-1 | Bladder cancer | 2025 | Chemotherapy | Localised | 2 | — | Complete response in carcinoma in situ: see trialMilestone: TAR-200 for BCG-unresponsive high-risk NMIBC |
| TROPION-Breast02 | Breast cancer | 2025 | Antibody–drug conjugate, Immunotherapy | Metastatic | 3 | 644 | PFS: median 10.8 vs 5.6 months, HR 0.57Milestone: ADCs reach first-line metastatic TNBC (ASCENT-03/-04, TROPION-02) |
| AIEOP-BFM ALL 2017 | Childhood acute lymphoblastic leukemia (ALL) | 2026 | Immunotherapy, Chemotherapy | Any setting | 3 | 709 | EFS: 83% vs 70.3% at 48 mo, HR 0.51 (95% CI 0.35–0.73)Milestone: Blinatumomab replaces chemotherapy cycles in high-risk B-ALL |
| ATOMIC | Colorectal cancer | 2026 | Immunotherapy, Chemotherapy | Adjuvant | 3 | 712 | DFS: 86.3% vs 76.2% at 36 mo, HR 0.50 (95% CI 0.35–0.73)Milestone: ATOMIC: atezolizumab plus adjuvant FOLFOX in dMMR stage III colon |
| BRUIN CLL-322 | Chronic lymphocytic leukaemia | 2026 | Targeted, Immunotherapy | Relapsed / refractory | 3 | 639 | PFS: 87% vs 72% at 24 mo, HR 0.547 (95% CI 0.400–0.748)Milestone: BRUIN CLL-322: pirtobrutinib added to venetoclax-rituximab |
| ENDURANCE | Multiple myeloma | 2026 | Targeted | Maintenance | 3 | 1,087 | OS: 69% vs 68.6% at 84 moMilestone: ENDURANCE: continuous or fixed-duration lenalidomide |
| ENGOT-ov65/KEYNOTE-B96 | Ovarian cancer | 2026 | Immunotherapy, Chemotherapy | Relapsed / refractory | 3 | 643 | PFS: median 8.3 vs 7.2 months, HR 0.72 (95% CI 0.58–0.89)Milestone: KEYNOTE-B96: pembrolizumab plus weekly paclitaxel, PD-L1 CPS 1+ |
| EZH-202 cohort 5 | Sarcoma and GIST | 2026 | Targeted | Metastatic, Locally advanced | 2 | 62 | ORR: see trialMilestone: Tazemetostat: epithelioid sarcoma accelerated approval withdrawn |
| HERIZON-GEA-01 | Gastric and oesophageal cancer | 2026 | Immunotherapy, Chemotherapy | Metastatic | 3 | — | PFS: median 12.4 vs 8.1 months, HR 0.63 (95% CI 0.51–0.78)Milestone: HERIZON-GEA-01: zanidatamab, with or without tislelizumab |
| IMbrave050 | Hepatocellular carcinoma | 2026 | Immunotherapy, Targeted | Adjuvant | 3 | 668 | RFS: HR 0.72Milestone: IMbrave050: early recurrence-free benefit not sustained on update |
| KEYNOTE-B15/EV-304 | Bladder cancer | 2026 | Antibody–drug conjugate, Immunotherapy | Perioperative | 3 | 808 | EFS: 79.4% vs 66.2% at 24 mo, HR 0.53 (95% CI 0.41–0.70)Milestone: KEYNOTE-B15/EV-304: EV-pembrolizumab versus cisplatin-gemcitabine |
| LITESPARK-011⚑ needs review | Kidney cancer (renal cell carcinoma) | 2026 | Targeted | Metastatic | 3 | 747 | PFS: median 14.6 vs 10.6 months, HR 0.74 (95% CI 0.61–0.89)Milestone: LITESPARK-011: belzutifan plus lenvatinib versus cabozantinib (2026) |
| LITESPARK-022 | Kidney cancer (renal cell carcinoma) | 2026 | Targeted, Immunotherapy | Adjuvant | 3 | — | DFS: 80.7% vs 73.7% at 24 mo, HR 0.72 (95% CI 0.59–0.87)Milestone: LITESPARK-022: adjuvant belzutifan added to pembrolizumab (2026) |
| PSMAddition | Prostate cancer | 2026 | Radiation | Metastatic | 3 | 1,144 | rPFS: HR 0.72 (95% CI 0.58–0.90)Milestone: 177Lu-PSMA-617 plus ARPI in PSMA-positive mHSPC (PSMAddition) |
| RASolute 302 | Pancreatic cancer | 2026 | Targeted | Metastatic, Relapsed / refractory | 3 | 500 | OS: median 13.2 vs 6.6 months, HR 0.4Milestone: Daraxonrasib: RAS(ON) inhibitor extends survival after prior therapy |