Efficacy of venetoclax combined with homoharringtonine and cytarabine for younger adults with newly diagnosed AML

Abstract

Venetoclax (VEN)--based induction therapy has demonstrated considerable promise in treating acute myeloid leukemia (AML); however, the optimal VEN-based combination therapy remains to be established. This study evaluated the efficacy and safety of the venetoclax-homoharringtonine-cytarabine (VHA) regimen in patients with newly diagnosed (ND) AML. A retrospective analysis was conducted on 55 patients treated with the VHA regimen. The overall response rate (ORR) was 92.7% (51/55, 95% CI 82%-98%), and the composite complete remission (CRc) rate was 87.3% (48/55, 95% CI 76%-95%). Among the 48 patients who achieved CRc, 91.7% (44/48, 95% CI 67%-90%) achieved complete remission (CR), and 85.4% (41/48, 95% CI 72%-94%) reached measurable residual disease (MRD)-negative CR. In the adverse-risk group, ORR and CRc were 95% (19/20, 95% CI 75%-100%) and 75% (15/20, 95% CI 51%-91%), respectively. The most common grade 3–4 adverse events were febrile neutropenia (32.7%), pneumonia (16.3%), and sepsis (9.1%). Median overall survival (OS) was 26 months, while event-free survival (EFS) was not reached. One-year OS was 83%, and one-year EFS was 82%. These preliminary data suggest that the VHA regimen achieves a very high rate of CR and low toxicity, particularly for adverse-risk AML patients.