COG AAML0531
Adding the antibody-drug conjugate gemtuzumab ozogamicin to chemotherapy lowered the chance of relapse in children with acute myeloid leukaemia. Years after the drug had been withdrawn from the US market, this trial helped bring it back for children.
Overview
Randomised phase 3 (Gamis et al., JCO 2014), 1,022 evaluable patients. Gemtuzumab ozogamicin (GO, 3 mg/m2) given once in induction course 1 and once in intensification course 2 improved 3-year event-free survival (53.1% versus 46.9%; HR 0.83; 95% CI 0.70 to 0.99) through a reduction in relapse risk, with a non-significant trend in overall survival and slightly higher post-remission treatment-related mortality. Benefit was later shown to track with CD33 expression and CD33 splicing genotype, an early example of a pharmacogenomic biomarker in paediatric oncology. GO had been withdrawn in the US in 2010 after the adult SWOG S0106 trial; AAML0531 together with the adult ALFA-0701 trial supported the 2017 FDA re-approval, which included children. Successor COG trials (AAML1031, AAML1831) build on this backbone with FLT3 inhibitors and CPX-351.
- 53.1 vs 46.9 out of 100 free of a major event at 3 years with Chemotherapy + gemtuzumab ozogamicin compared with Chemotherapy; 6.2 more per 100.
- Roughly one extra person helped for every 16 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
- Put another way, the treated group had about 17 percent lower chance of the event at any given time (hazard ratio 0.83, likely range 0.7 to 0.99).
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- These results apply to the people the trial enrolled: Newly diagnosed AML, age 0-29: standard five-course chemotherapy with or without two doses of gemtuzumab ozogamicin. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
1,070 participants enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| 3-year event-free survivalprimary | Chemotherapy + gemtuzumab ozogamicin | — | 53.1% | 0.83 (0.7–0.99) | — | link |
| Chemotherapy | — | 46.9% |
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