Authors
Amir T Fathi, Alexander E Perl, Geoffrey G Fell, Brian A Jonas, Brittany K Ragon, Alice S Mims, Uma Borate, Gabriel N Mannis, Karen Quillen, Maximilian Stahl, Brandon J Aubrey, Paul Koller, Andrew S Artz, Monzr M Al Malki, Anthony S Stein, Guido Marcucci, Mary Linton B Peters, Timothy A Graubert, Peter Westervelt, Philip C Amrein, Hanno R Hock, Andrew M Brunner, Gabriela Hobbs, Rupa Narayan, Michelle H Lee, Peter G Miller, Alyssa L Watson, Richard Hao, Abigail C Marola, Jennifer Lombardi-Story, Shilton Dhaver, Michael R Grunwald, Yi-Bin Chen, Matthew J Weinstock, Andrew H Matthews, Ian McGovern, Brent L Wood, Christopher S Hourigan, Donna S Neuberg, Areej El-Jawahri, Ibrahim Aldoss
Published in
The New England journal of medicine. Volume 395. Issue 9. Pages 845-858. Sep 03, 2026.
Abstract
Induction chemotherapy has long been a key component of curative therapy for fit patients with acute myeloid leukemia (AML), despite its frequently severe side effects and substantial health care utilization. For patients who are ineligible for induction chemotherapy, hypomethylating therapy plus venetoclax is the standard treatment owing to its efficacy and side-effect profile.
In this multicenter, phase 2 trial, we randomly assigned, in a 1:1 ratio, previously untreated adults with AML who were eligible for induction chemotherapy to receive either azacitidine plus venetoclax or induction chemotherapy. Patients with core binding factor fusions, mutations in the gene encoding FMS-like tyrosine kinase 3 (FLT3), or mutations in the gene encoding nucleophosmin-1 (NPM1; unless the patient was ≥60 years of age) were excluded. The primary end point was event-free survival.
A total of 172 patients underwent randomization, with 86 patients assigned to each group. The median age of the patients was 64 years. A total of 72% of the patients had adverse-risk disease according to the European LeukemiaNet 2022 classification. At a median follow-up of 21.9 months, the median event-free survival was 14.5 months (95% confidence interval [CI], 10.4 to 24.4) in the azacitidine-venetoclax group, as compared with 6.2 months (95% CI, 4.1 to 10.1) in the induction chemotherapy group, corresponding to a hazard ratio for event or death of 0.57 (95% CI, 0.39 to 0.84; P = 0.002 by the stratified log-rank test). Infection of grade 3 or higher occurred in 28% of the patients (95% CI, 19 to 39) receiving azacitidine-venetoclax and in 41% of those (95% CI, 30 to 52) receiving induction chemotherapy; hemorrhage of grade 3 or higher occurred in 2% (95% CI, 0.3 to 8) and 12% (95% CI, 6 to 20), respectively.
In this phase 2, randomized trial, azacitidine-venetoclax therapy led to significantly longer event-free survival than induction chemotherapy among induction-eligible patients with AML. (Funded by AbbVie and others; PARADIGM ClinicalTrials.gov number, NCT04801797.).
PMID:
42685316
Bibliographic data and abstract were imported from PubMed on 03 Sep 2026.
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