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

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: (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.