Title Hypomethylating agents plus venetoclax versus intensive chemotherapy in acute myeloid leukemia with chromosome 5 and 7 abnormalities
Authors Boussi, Leora ; Bewersdorf, Jan Philipp ; Liu, Yiwen ; Shallis, Rory M ; Aguirre, Luis E ; Bystrom, Rebecca P ; Žučenka, Andrius ; Garciaz, Sylvain ; DeAngelo, Daniel J ; Stone, Richard M ; Luskin, Marlise R ; Garcia, Jacqueline S ; Winer, Eric S ; Chen, Evan C ; Wadleigh, Martha ; Berton, Guillaume ; Ling, Kelly ; Zeidan, Amer M ; Stein, Eytan M ; Shimony, Shai ; Goldberg, Aaron D ; Stahl, Maximilian
DOI 10.3324/haematol.2025.288891
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Is Part of Haematologica.. Pavia : Ferrata Storti Foundation. 2026, vol. 111, no. 7, p. 2342-2352.. ISSN 0390-6078. eISSN 1592-8721
Abstract [eng] Abnormalities in chromosomes 5 and 7 are frequently identified in acute myeloid leukemia (AML), particularly enriched in therapy-and myelodysplasia-related disease, and confer an adverse prognosis. Given the high risk of relapse, allogeneic stem cell transplant (allo-SCT) is typically recommended for patients achieving complete remission (CR) following induction chemotherapy. We currently lack prospective data to decide whether intensive chemotherapy (IC) versus hypomethylating agent+venetoclax (HMA+ven) is the superior frontline treatment approach for these patients. Hence, we performed a retrospective study in a large cohort of patients with AML and deletion 7 (-7) and/or deletion 5 or 5q (-5/del5q) comparing outcomes between IC-versus HMA+ven-treated patients. Remission rates after IC and HMA+ven were found to be comparable (43% vs 52%, p=0.2). When adjusting for patient and disease characteristics in multivariable analysis (MVA), treatment with IC vs HMA+ven did not significantly impact overall survival (OS) (HR 1.02, p=0.9202), while age at diagnosis (HR 1.02, p=0.0324), prior myeloid disease (HR 1.42, p=0.0266), monosomal karyotype (HR 1.48, p=0.029), complex karyotype (HR 1.61, p=0.0156), and KRAS mutations (HR 2.21, p=0.0063) were associated with inferior survival. There was also no difference in OS in patients age 60-75 years by treatment strategy (7.8 vs 6.4 months, p=0.56), motivating future randomized trials of IC versus HMA+ven in this older population to inform optimal therapy. Importantly, OS was significantly improved in patients undergoing allo-SCT irrespective of frontline therapy, and allo-SCT consolidation was the most important predictor of long-term survival in MVA (HR 0.36, p.
Published Pavia : Ferrata Storti Foundation
Type Journal article
Language English
Publication date 2026
CC license CC license description