Efficacy and Safety of Azacitidine Combined With Lisaftoclax in the Treatment of Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndrome With Increased Blasts

ABSTRACT Objective To retrospectively analyze the early efficacy and safety of azacitidine plus lisaftoclax in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome with increased blasts (MDS‐IB), providing a reliable clinical reference. Methods A total of 17 patients admitted to the Department of Hematology between August 1 and December 31, 2025, were enrolled. The treatment was as follows: azacitidine 75 mg/m 2 (Days 1–7, subcutaneously) and lisaftoclax 200–600 mg (Days 1–10/14, orally), repeated in 28‐day cycles. Efficacy and safety were evaluated. Results Among nine AML patients, 55.6% achieved complete remission (CR) after one cycle, with 60.0% of responders being MRD‐negative. Among eight MDS‐IB patients, 62.5% achieved CR and 25.0% achieved hematologic improvement, with 40.0% of CR patients being MRD‐negative. The median time to first CR was 1 month for both AML and MDS‐IB patients. In the entire cohort, common Grade 3–4 adverse events included thrombocytopenia/leukopenia (58.8%), lymphopenia (47.1%), and febrile neutropenia (35.3%, AML: 22.2%, MDS‐IB: 50.0%); no treatment‐related deaths occurred. Median follow‐up was 2.1 months; median OS was 1.9 months, and RFS was unevaluable. Conclusion This regimen induces favorable responses with manageable toxicity, supporting its further investigation, while longer follow‐up and larger prospective studies are required to confirm the long‐term efficacy. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission