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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 Years after the drug had been withdrawn from the US market, this trial helped bring it back for children.
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 GO had been withdrawn in the US in 2010 after the adult SWOG S0106 trial; AAML0531 together with the adult ALFA-0701 trial supported the 2017 FDA re-approval, which included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.
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 enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| 3-year event-free survivalprimary | Chemotherapy + gemtuzumab ozogamicin | - | 53.1% | 0.83 (0.7 to 0.99) | - | link |
| Chemotherapy | - | 46.9% |
The full inclusion and exclusion criteria are on the registry record. Read 2026-10-07. Recruiting overall does not mean a particular site is open; ask the study team.
ClinicalTrials.gov data courtesy of the US National Library of Medicine.
Shares Cytarabine + anthracycline ('7+3'), Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Children's Oncology Group (COG) and the tags paediatric, haematologic.
Shares Children's Oncology Group (COG), Acute myeloid leukaemia and the tags paediatric, haematologic.
Shares Children's Oncology Group (COG) and the tags paediatric, haematologic.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first and the tag paediatric.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Children's Oncology Group (COG) and the tag paediatric.
Shares Children's Oncology Group (COG) and the tag paediatric.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Children's Oncology Group (COG) and the tag paediatric.
Shares Acute myeloid leukaemia and the tag haematologic.
Every source this page cites, grouped by who published it. Grouping is read from the web address, not assigned by hand.
A source that has moved or gone is reported by the weekly link check. /audit/