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
More than half of children with relapsed leukemia achieved complete remission with a venetoclax-chemotherapy combination, with most clearing detectable cancer cells.
The VENAML phase 1 expansion tested venetoclax combined with intensive cytarabine-based regimens in 44 children and young adults (ages 2-24) with relapsed/refractory AML across three US centers. Over 5.3 years of median follow-up, 57% achieved complete remission and the majority of responders cleared measurable residual disease, directly informing an ongoing randomized Phase 3 trial (NCT05183035).
What the study was
- Study design
- prospective_multicenter_phase1_expansion
- Category
- hematologic_malignancies
- Maturity
- Potentially Practice-Changing
- Journal
- Lancet Haematol
Why it surfaced
Landmark Phase 1 expansion from Lancet Haematol with >5-year follow-up; 57% CR/CRi and MRD negativity define go-forward dosing for NCT05183035 Phase 3; pediatric R/R AML is highest-unmet-need setting in hematologic oncology.
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