CPX-351 (Liposomal Cytarabine and Daunorubicin) versus venetoclax plus hypomethylating agent therapy in newly diagnosed acute myeloid leukemia: a retrospective comparison involving 600 Mayo Clinic patients

作者：Saubia Fathima, Lior Rokach, Nour Ghosoun, Mahsa Rezasoltani, Rimal Ilyas, Ali Alsugair, Kristen McCullough, Maymona Abdelmagid, Aref Al-Kali, Hassan B. Alkhateeb, Kebede H. Begna, Abhishek A. Mangaonkar, Aasiya Matin, Antoine N. Saliba, Mehrdad Hefazi Torghabeh, Mark R. Litzow, William Hogan, Meera Shah, Mrinal M. Patnaik, Animesh Pardanani, Talha Badar, Hemant S. Murthy, James Foran, Jeanne Palmer, Nathan Punwani, Lisa Sproat, Nandita Khera, Cecilia Arana Yi, Ayalew Tefferi, Naseema Gangat · 发表于：Blood Cancer Journal · 年份：2026 · DOI：10.1038/s41408-026-01495-x · 被引用次数：2 · 研究领域：Acute Myeloid Leukemia Research、Retinoids in leukemia and cellular processes、Acute Lymphoblastic Leukemia research

The comparative value of liposomal cytarabine/daunorubicin (CPX-351) versus venetoclax plus a hypomethylating agent (Ven-HMA) in the frontline treatment of older adults with primary (de novo) or secondary acute myeloid leukemia (AML) remains uncertain. In the current study, we retrospectively examined outcomes of 600 patients with newly diagnosed AML treated with CPX-351 ( N = 112) or Ven-HMA ( N = 488). AML subtypes included de novo ( N = 277, 46%), post-myelodysplastic syndrome (post-MDS, N = 114,19%), post-myeloproliferative neoplasm (post-MPN, N = 70, 12%), post-MDS/MPN ( N = 36, 6%), and t-AML ( N = 103, 17%). Patients receiving CPX-351 were younger (median 65 vs. 73 years; p < 0.01), predominantly female (50% vs. 38%; p = 0.02), more likely to have secondary AML (68% vs. 51%; p < 0.01), and less likely to harbor NPM1 MUT (5% vs. 12%; p = 0.02). Rates of complete response with or without count recovery (CR/CRi) were comparable between CPX-351 and Ven-HMA (55% vs. 60%; p = 0.30), including AML with myelodysplasia-related gene mutations or cytogenetic abnormalities (AML-MR 60% vs. 63%; p = 0.70). Ven-HMA use was associated with fewer infectious complications (62% vs. 83%; p < 0.01) and yielded higher CR/CRi rates in males (60% vs. 45%; p = 0.04), de novo AML (68% vs. 50%; p = 0.03), and in the presence of STAG2 MUT (86% vs. 44%; p = 0.02), or CEBPA MUT (88% vs. 50%; p = 0.03). Overall survival censored for transplant, was similar (median 10 vs. 13 months; p = 0.90), with V...