Abstract:Objective To explore the efficacy and safety of venetoclax combined with demethylation drugs in the treatment of elderly patients with AML unsuitable for intensive chemotherapy. Methods A retrospective analysis was performed on the clinical data of 42 elderly patients with acute myeloid leukemia (AML) who were treated with venetoclax combined with demethylation drugs (descitabinc, azacytidine) in the Affiliated Hospital of Nantong University from January 2020 to August 2022. The primary-end points in clinical observation were complete remission (CR), composite complete remission ［CR+CR with incomplete blood count recovery (CRi)］ and overall response rate (ORR), and the secondary end points were overall survival (OS) and drug safety. Results The median follow-up time of all patients was 5.0 months. The efficacy results were as follows: 9.5% CR, 33.3% CR+CRi and 66.7% ORR. Subgroup analysis showed that the ORR of newly diagnosed patients was 66.7%, with 11 months of the median survival time; the ORR of recurrent patients was 57.1%, with 12 months of the median survival time ; and the ORR of refractory patients was 85.7%, with 14 months of the median survival time. There was no significant difference among three subgroups (P＞0.05). The median survival time of remission patients was significantly higher than that of patients without remission ［14.0 (12.3-15.7) months vs 2.0 (0.1-6.1) months, P＜0.05］. The blood cells of 42 patients decreased to different degrees, including 35 cases of granulocytopenia (83.3%), 16 cases of anemia (38.1%) and 10 cases of thrombocytopenia (23.8%). In addition, the common side effects were mainly gastrointestinal reactions, which were controlled, and no tumor lysis syndrome occurred. Conclusion In the treatment of elderly AML patients, venetoclax combined with demethylation drugs is safe, effective and well tolerated, and provides a treatment option for elderly AML patients who are not suitable for intensive chemotherapy.