KEYNOTE-057
Needs reviewPembrolizumab monotherapy for the treatment of high-risk non-muscle-invasive bladder cancer unresponsive to BCG (KEYNOTE-057): an open-label, single-arm, multicentre, phase 2 study
Phase 2N = 101NCT02625961
- Population
- Adults with histologically confirmed BCG-unresponsive carcinoma in situ of the bladder, with or without papillary tumours, ECOG performance status 0-2, ineligible for or declining radical cystectomy (cohort A; 101 enrolled).
- Design
- Open-label, single-arm, multicentre phase 2 study at 54 sites in 14 countries; this record covers cohort A (BCG-unresponsive carcinoma in situ with or without papillary tumours, ineligible for or declining radical cystectomy). Cohort B (BCG-unresponsive high-grade Ta or T1 papillary disease without carcinoma in situ) is reported separately. Complete response assessed by cystoscopy and urine cytology about 3 months after the first dose. The registry record (NCT02625961, title MK-3475-057/KEYNOTE-057) covers more than cohort A and lists phase 2, actual enrolment 296.
- Primary endpoint
- Complete response in carcinoma in situ
- Arms
- Pembrolizumab (experimental, n = 101): Pembrolizumab 200 mg intravenously every 3 weeks for up to 24 months or until persistence, recurrence or progression confirmed centrally, unacceptable toxic effects or withdrawal of consent
- People
- Balar AV
Show the numbers
| Endpoint | Analysis | Pembrolizumab | Control | Effect |
|---|---|---|---|---|
| Complete response in carcinoma in situEfficacy population: 96 patients with BCG-unresponsive carcinoma in situ (with or without papillary tumours) who met the FDA definition; not described as intention-to-treatClinical complete response: absence of high-risk non-muscle-invasive bladder cancer or progressive disease on cystoscopy and urine cytology, about 3 months after the first dose | primarymedian follow-up 36.4 monthsBalar 2021 | 41% (30.7–51.1%) | — | 39 of 96 patients. Five of the 101 enrolled patients did not meet the FDA definition of BCG-unresponsive disease and were excluded from the efficacy analysis. |
| Grade 3 or worse treatment-related adverse eventsSafety population: 101 patients who received at least one doseGrade 3 or 4 treatment-related adverse events | primaryBalar 2021 | 13% | — | Most common: hyponatraemia (3 patients) and arthralgia (2 patients). Serious treatment-related adverse events in 8 patients (8%); no deaths were considered treatment related. |
| Endpoint | Analysis | Pembrolizumab | Control | Effect |
|---|---|---|---|---|
| Complete response in carcinoma in situEfficacy population: 96 patients with BCG-unresponsive carcinoma in situ (with or without papillary tumours) who met the FDA definition; not described as intention-to-treatClinical complete response: absence of high-risk non-muscle-invasive bladder cancer or progressive disease on cystoscopy and urine cytology, about 3 months after the first dose | primarymedian follow-up 36.4 monthsBalar 2021 | 41% (30.7–51.1%) | — | 39 of 96 patients. Five of the 101 enrolled patients did not meet the FDA definition of BCG-unresponsive disease and were excluded from the efficacy analysis. |
| Grade 3 or worse treatment-related adverse eventsSafety population: 101 patients who received at least one doseGrade 3 or 4 treatment-related adverse events | primaryBalar 2021 | 13% | — | Most common: hyponatraemia (3 patients) and arthralgia (2 patients). Serious treatment-related adverse events in 8 patients (8%); no deaths were considered treatment related. |
Appears in
Sources
Balar AV, Kamat AM, Kulkarni GS, Uchio EM, Boormans JL, Roumiguié M, et al. Pembrolizumab monotherapy for the treatment of high-risk non-muscle-invasive bladder cancer unresponsive to BCG (KEYNOTE-057): an open-label, single-arm, multicentre, phase 2 study. Lancet Oncol. 2021;22(7):919-930.