Venetoclax plus hypomethylating agents versus priming regimen as the first-line therapy for newly diagnosed acute myeloid leukemia with myelodysplasia-related changes: a propensity score-matched analysis

Acute myeloid leukemia with myelodysplasia-related changes (AML-MR), a high-risk subtype affecting older patients intolerant to intensive chemotherapy. Venetoclax-based regimens are approved for such patients, while studies suggest priming regimens combining aclarubicin, homoharringtonine or idarubicin and low-dose cytarabine with granulocyte colony-stimulating factor may replace intensive therapy, while direct comparisons are lacking in two groups. This retrospective study compared venetoclax plus hypomethylating agents (VEN+HMA) and priming regimens in 150 propensity score-matched AML-MR patients. VEN+HMA demonstrated superior overall remission (ORR: 78.7% vs. 60.0%, p = 0.013) and complete remission (CR: 48.0% vs. 21.3%, p < 0.001) rates. Notably, VEN+HMA significantly improved median overall survival (OS: not reached vs. 36.37 months, p = 0.026) and event-free survival (EFS: not reached vs. 7.37 months, p = 0.003). Multivariate analysis confirmed VEN+HMA as an independent predictor of better ORR (OR = 2.855, p = 0.014), OS (HR = 0.449, p = 0.036), and EFS (HR = 0.458, p = 0.003). These findings suggest VEN+HMA may be a preferable low-intensity induction therapy over priming regimens for AML-MR.