DFCI 05-001
Intravenous pegylated asparaginase versus intramuscular native Escherichia coli L-asparaginase in newly diagnosed childhood acute lymphoblastic leukaemia (Dana-Farber Cancer Institute ALL Consortium Protocol 05-001)
Phase 3N = 463NCT00400946
- Population
- Children aged 1 to 18 years with newly diagnosed acute lymphoblastic leukaemia who achieved complete remission after induction.
- Design
- Randomised, open-label phase 3 comparison within DFCI ALL Consortium Protocol 05-001 at 11 sites in the USA and Canada. Of 551 eligible patients enrolled between April 2005 and February 2010, 526 achieved complete remission after 32 days of induction and 463 were randomised 1:1, from week 7, to intravenous pegylated asparaginase or intramuscular native E coli asparaginase. Randomisation was stratified by final risk group. Primary endpoint: overall frequency of asparaginase-related toxicities (allergy, pancreatitis, thrombotic or bleeding complications). Analyses by intention to treat.
- Primary endpoint
- Asparaginase-related toxicity
- Arms
- Intravenous PEG-asparaginase (experimental, n = 232): PEG-asparaginase 2500 IU/m2 intravenously every 2 weeks, 15 doses, from week 7
- Intramuscular native E coli asparaginase (control, n = 231): Native E coli L-asparaginase 25 000 IU/m2 intramuscularly weekly, 30 doses, from week 7
- People
- Place AE, senior author Silverman LB, Dana-Farber Cancer Institute ALL Consortium
Δ Asparaginase-related toxicity+2 pointsΔ 60-month DFS+1 points
Show the numbers
| Endpoint | Analysis | Intravenous PEG-asparaginase | Intramuscular native E coli asparaginase | Effect |
|---|---|---|---|---|
| Asparaginase-related toxicityRandomised patients in complete remission (intention to treat)Overall frequency of asparaginase-related toxicities, defined as allergy, pancreatitis, and thrombotic or bleeding complications. | primaryPlace 2015 | 28% | 26% | P=0.6065 of 232 patients (intravenous PEG-asparaginase) versus 59 of 231 (intramuscular native E coli); the groups did not differ significantly. |
| DFSRandomised patients in complete remission (intention to treat) | primarymedian follow-up 6 yearsPlace 2015 | 90% at 5 y (86–94%) | 89% at 5 y (85–93%) | P=0.585-year disease-free survival, a predefined secondary endpoint. Median follow-up 6.0 years (IQR 5.0 to 7.1). |
| Endpoint | Analysis | Intravenous PEG-asparaginase | Intramuscular native E coli asparaginase | Effect |
|---|---|---|---|---|
| Asparaginase-related toxicityRandomised patients in complete remission (intention to treat)Overall frequency of asparaginase-related toxicities, defined as allergy, pancreatitis, and thrombotic or bleeding complications. | primaryPlace 2015 | 28% | 26% | P=0.6065 of 232 patients (intravenous PEG-asparaginase) versus 59 of 231 (intramuscular native E coli); the groups did not differ significantly. |
| DFSRandomised patients in complete remission (intention to treat) | primarymedian follow-up 6 yearsPlace 2015 | 90% at 5 y (86–94%) | 89% at 5 y (85–93%) | P=0.585-year disease-free survival, a predefined secondary endpoint. Median follow-up 6.0 years (IQR 5.0 to 7.1). |
Appears in
Sources
Place AE, Stevenson KE, Vrooman LM, Harris MH, Hunt SK, O'Brien JE, et al. Intravenous pegylated asparaginase versus intramuscular native Escherichia coli L-asparaginase in newly diagnosed childhood acute lymphoblastic leukaemia (DFCI 05-001): a randomised, open-label phase 3 trial. Lancet Oncol. 2015;16(16):1677-90.