摘要
目的:评估阿扎胞苷(Azacitidine,AZA)联合维奈克拉(Venetoclax,VEN)治疗老年初治急性髓系白血病(acute myeloid leukemia,AML)的疗效与安全性。方法:回顾性分析2020年5月至2024年12月我院收治的41例老年初治AML患者的临床数据。所有患者接受AZA+VEN治疗,主要终点为完全缓解/完全缓解伴不完全血细胞恢复率(CR/CRi),次要终点包括总缓解率(ORR)及微小残留病灶(MRD)阴转率。采用单因素/多因素模型分析影响因素。结果:中位治疗3个周期,CR/CRi率51.22%(21/41),ORR达63.41%(26/41);首周期治疗CR/CRi率为43.90%,61.90%的患者在1~2个周期内实现MRD转阴。多因素分显示,年龄≥75岁、血红蛋白(Hb)≥80 g·L^(-1)、ASXL1基因突变和不存在TP53基因突变是患者获得治疗缓解的独立预测因素(P<0.05)。中位随访10.6个月,1年总生存期(OS)为55.39%。初诊原始细胞≥50%、TP53突变及未达CR/CRi与OS缩短显著相关(P<0.05)。安全性方面,所有患者均出现Ⅲ~Ⅳ级血液学毒性,非血液学毒性以肺部感染最常见,1例患者因重症感染死亡。结论:AZA联合VEN治疗老年初治AML疗效显著且安全可控。年龄、Hb、ASXL1/TP53突变可预测疗效,原始细胞负荷、TP53异常及缓解状态是重要预后指标。
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 gradeⅢ-Ⅳhematologic 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.
作者
刘缦缦
裴晓杭
LIU Manman;PEI Xiaohang(Hebi People′s Hospital,Henan Hebi 458030,China;Henan Provincial People′s Hospital,Henan Zhengzhou 450003,China)
出处
《中国药物评价》
2025年第4期300-305,共6页
Chinese Journal of Drug Evaluation
