Abstract
BACKGROUND: Azacitidine (AZA) plus venetoclax (VEN) has emerged as a widely accepted treatment option for acute myeloid leukemia (AML), particularly in older patients or those unfit for intensive chemotherapy. However, real-world data on AZA + VEN efficacy and safety in Southeast Asia remain limited. OBJECTIVES: To evaluate the real-world effectiveness and safety of AZA + VEN in newly diagnosed (ND) and relapsed/refractory (R/R) AML patients in Thailand. DESIGN: A retrospective observational multicenter study. METHODS: This is a multicenter retrospective study included ND and R/R AML patients treated between 2021 and 2024 at three tertiary hospitals in Thailand. All patients received AZA at 75 mg/m(2) for 7 days per cycle. VEN dosing and duration were individualized based on physician judgment, drug availability, and patient affordability. Data collection included clinical characteristics, cytogenetics, treatment details, response rates, survival outcomes, and toxicities. RESULTS: A total of 81 patients were analyzed, included 54 ND and 27 R/R AML cases, with a median age of 65 years. Based on European LeukemiaNet 2022 classification, 51.9% had intermediate risk, and 33.3% had adverse risk. The composite complete remission was 56.8% (ND: 64.8%, R/R: 40.7%). VEN was administered at a median dose of 100 mg for 28 days, combined with potent CYP3A4 inhibitor of antifungal prophylaxis (posaconazole 51.0%, voriconazole 30.4%, itraconazole 17.7%). The median overall survival was 9.2 months and relapse-free survival was 8.1 months. Grades 3-4 neutropenia and febrile neutropenia occurred in 93.8% and 60.5% of patients, respectively. CONCLUSION: This real-world practice highlights the feasibility and effectiveness of AZA-VEN in combination with antifungal prophylaxis for elderly or unfit AML patients in resource-limited countries. However, infectious complications remain a concern with this low-intensity regimen.