ArticleThe Lancet. Haematology2026
Venetoclax in combination with cytarabine with or without idarubicin or azacitidine in children, adolescents, and young adults with relapsed or refractory acute myeloid leukaemia (VENAML): a multicentre, phase 1 expansion study.
Article in The Lancet. Haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05183035 (A Randomized Phase 3 Trial of Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Children With Relapsed AML), which is not on this map. Not yet cited in PubMed.
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A Randomized Phase 3 Trial of Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Children With Relapsed AML
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Abstract
backgroundThe prognosis for paediatric patients with relapsed or refractory acute myeloid leukaemia remains poor. Although encouraging, published paediatric data on venetoclax combined with intensive chemotherapy are scarce. We aimed to refine efficacy and toxicity estimates from the previously published dose-escalation phase of this study.
methodsThis multicentre, phase 1 study included two phases, an initial dose-escalation phase to identify the primary endpoint of recommended phase 2 dose (RP2D; previously reported), followed by an expansion cohort phase, the results of which are reported here. Paediatric patients (aged 2-24 years) with relapsed or refractory acute myeloid leukaemia from three US research hospitals were included. Patients had at least 5% blasts in the bone marrow by morphology or at least 1% blasts by flow cytometry. Treatment varied across cohorts. Patients received oral venetoclax 360 mg/m
findingsFrom July 1, 2017, to July 22, 2022, 61 patients were enrolled and 44 patients were subsequently included and treated at the RP2D (21 [48%] female, 23 [52%] male; 22 [50%] in cohort A, 15 [34%] in cohort B, and seven [16%] in cohort C). The median follow-up was 5·3 years (IQR 4·2-6·3). After 1 cycle, 25 (57% [95% CI 41-72]) patients had a complete response with or without haematological recovery; 19 were negative for measurable residual disease. Common grade 3 or 4 adverse events included febrile neutropenia (23 [52%]), gastrointestinal disorders (14[32%]]) including colitis (four [9%]), and infections (11 [25%]). There were two grade 5 adverse events due to sepsis and multiorgan failure.
interpretationVenetoclax with high-dose cytarabine is active with acceptable safety in paediatric patients with relapsed or refractory AML. These findings support ongoing research of venetoclax with high-dose cytarabine in this population, including the randomised paediatric phase 3 trial (NCT05183035).
fundingUS National Institutes of Health (NIH), American Lebanese Syrian Associated Charities (ALSAC), AbbVie, Gateway for Cancer Research.
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