Citations 1
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
Gangat, Naseema, Fathima, Saubia , Rokach, Lior , Ghosoun, Nour, Rezasoltani, Mahsa , Ilyas, Rimal, Alsugair, Ali, McCullough, Kristen , Abdelmagid, Maymona, Al‐Kali, Aref , Alkhateeb, H., Begna, Kebede H. , Mangaonkar, Abhishek, Matin, Aasiya, Hefazi, Mehrdad T., LITZOW, Mark R., Shah, Maleeha, Patnaik, Mrinal M. , Pardanani, Animesh, Foran, James M. , Palmer, Jeanne , Punwani, Nathan , Sproat, Lisa, Khera, Nandita , Tefferi, Ayalew, McCullough, Kristen B , Fathima, Saubia , Ghosoun, Nour, Ilyas, Rimal, Alkhateeb, Hassan B. , Mangaonkar, Abhishek A. , Matin, Aasiya , Saliba, Antoine N. , Hefazi, Mehrdad , Litzow, Mark R. , Hogan, William J , Shah, Mithun V , Pardanani, Animesh , Badar, Talha , Murthy, Hemant S. , Sproat, Lisa, Yi, Cecilia Arana , Tefferi, Ayalew , Gangat, Naseema
No container title available | December 22, 2025 | Volume: No volume available | Issue: No issue available | Pages: No page available
Abstract 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 (46%), post-myelodysplastic syndrome (MDS) (19%), post-myeloproliferative neoplasm (MPN) (12%), post-MDS/MPN (6%), therapy-related (17%) and AML with myelodysplasia-related genetic abnormalities (AML-MR; 46%). 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 in 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 Ven-HMA being superior in post-MDS AML (median 12 vs. 7 months; p = 0.02) and CPX-351 in the presence of SF3B1 MUT (not reached vs. 14 months; p < 0.01). Our findings suggest that Ven-HMA is as effective and less toxic than CPX-351 in newly diagnosed AML, including AML-MR, despite selection of younger, fitter patients for CPX-351.