CPX‑351 vs Ven + HMA in newly diagnosed AML: A retrospective study

A retrospective comparison evaluating CPX-351 (liposomal cytarabine and daunorubicin; n = 112) vs venetoclax (Ven) + hypomethylating agent (HMA) therapy (n = 488) in adults with newly diagnosed (ND) acute myeloid leukemia (AML) was published in Blood Cancer Journal by Fathima et al. The primary objective was to compare response and survival outcomes between treatment strategies, particularly in AML with myelodysplasia-related gene mutations or cytogenetic abnormalities (AML-MR). 

Key data: Complete response with or without count recovery (CR/CRi) was comparable between CPX-351 and Ven + HMA (55% vs 60%; p = 0.30), including in the AML-MR subgroup (60% vs 63%; p = 0.68). Among responders (n = 188), measurable residual disease (MRD) negativity was higher with Ven + HMA (48% vs 75%; p = 0.01). Transplant-censored event-free survival (EFS) favored Ven + HMA (5 vs 9 months; p < 0.01). In AML-MR, median OS was 10 months with CPX-351 vs 14 months with Ven + HMA (p = 0.61), while median EFS favored Ven + HMA (5 vs 9 months; p = 0.01). Infectious complications were more frequent with CPX-351 (82% vs 62%; p < 0.01), while renal complications were more common with Ven + HMA (13% vs 6%; p = 0.04). 

Key learning: Ven + HMA may provide deeper responses and longer EFS with lower infectious toxicity in ND AML compared with CPX‑351, including in AML‑MR; however, specific molecular subgroups may derive differential benefit between therapies.