关键词: 阿扎胞苷, 维奈克拉, 老年急性髓系白血病, 首次治疗, 治疗反应, 生存预后, 安全性分析

Abstract: Objective： To evaluate the efficacy and safety of azacitidine (AZA) combined with venetoclax (VEN) in elderly patients with newly diagnosed acute myeloid leukemia (AML). Methods： A retrospective analysis was conducted on 41 elderly AML patients treated at our hospital between May 2020 and December 2024. All patients received AZA+VEN therapy. The primary endpoint was the composite complete remission rate (CR/CRi)， while secondary endpoints included overall response rate (ORR) and minimal residual disease (MRD) negativity. Univariate and multivariate analyses were performed to identify predictive factors. Results： After a median of 3 treatment cycles， the CR/CRi rate was 51.22% (21/41)， and the ORR was 63.41% (26/41). In the first cycle treatment， 43.90% achieved CR/CRi， and 61.90% patients attained MRD negativity within 1-2 cycles. Multivariate analysis identified age ≥75 years， baseline hemoglobin (Hb)≥80 g·L-1， ASXL1 mutation， and absence of TP53 mutation as independent predictors of response (P<0.05). With a median follow-up of 10.6 months， the 1-year overall survival (OS) rate was 55.39%. Baseline blast count ≥50%， TP53 mutation， and failure to achieve CR/CRi significantly reduced survival (P<0.05) and were independent adverse prognostic factors. All patients experienced grade Ⅲ-Ⅳ hematologic toxicity， with pulmonary infections being the predominant non-hematologic toxicity. One infection-related death occurred. Conclusion： AZA plus VEN demonstrates significant efficacy and manageable safety in elderly newly diagnosed AML patients. Age， Hb level， and ASXL1/TP53 mutations can predict treatment response， while blast burden， TP53 aberrations， and remission status are key prognostic indicators.

Key words: Azacitidine, Venetoclax, Elderly acute myeloid leukemia, Newly diagnosed, Treatment response, Survival prognosis, Safety analysis