维奈克拉联合低剂量阿糖胞苷治疗初诊急性髓系白血病的疗效与安全性分析  

Efficacy and safety of venetoclax combined with low-dose cytarabine in newly diagnosed acute myeloid leukemia

作　　者：曹林萍 费小明[1] 雷芳[1] 余先球[1] 杨元林 汤郁[2] Linping Cao;Xiaoming Fei;Fang Lei;Xianqiu Yu;Yuanlin Yang;Yu Tang(Department of Hematology,Affiliated Hospital of Jiangsu University,Zhenjiang 212001,China;Department of Rheumatology,Affiliated Hospital of Jiangsu University,Zhenjiang 212001,China)

机构地区：[1]江苏大学附属医院血液科,江苏省镇江市212001 [2]江苏大学附属医院风湿科,江苏省镇江市212001

出　　处：《中国肿瘤临床》2025年第3期122-126,共5页Chinese Journal of Clinical Oncology

基　　金：镇江市社会重点研发计划-社会发展项目(编号:SH2024084)资助。

摘　　要：目的:观察维奈克拉(venetoclax,VEN)联合小剂量阿糖胞苷(low-dose cytarabine,LDAC)方案对不适合接受强化疗的新诊断急性髓系白血病(newly diagnosed acute myeloid leukemia,unfit AML)患者的治疗效果、安全性及对总生存影响因素的探讨。方法:本研究回顾性分析2019年12月至2024年1月在江苏大学附属医院接受VEN+LDAC方案治疗的33例unfit AML患者的临床资料,评估该方案的治疗疗效及生存情况。结果:共入组33例患者(中位年龄72岁),其中原发急性髓系白血病(acute myeloid leukemia,AML)29例,继发AML 4例,中位随访时间为781天,中位总生存期(overall survival,OS)为467(104~812)天,中位无进展生存期(progression-free survival,PFS)为395(104~637)天。诱导化疗结束后,总反应率(overall response rate,ORR)为69.7%,其中完全缓解(complete response,CR)和完全缓解伴血液学未完全恢复(CR with incomplete blood count recovery,CRi)总和达到36.4%,形态学无白血病状态(morphologic leukemia-free state,MLFS)达到3%,部分缓解(partial remission,PR)达到30.3%。中位治疗疗程为3.0(1.0~6.5)个疗程。治疗后不良反应主要是血液学不良反应,多为≥Ⅲ级血液学毒性。Kaplan-Meier曲线分析结果表明ECOG评分、TP53基因、疗程数、治疗反应均会影响OS率(P<0.05)。单因素联合多因素Cox回归分析结果表明,治疗疗程是影响患者OS的独立危险因素(P<0.05)。结论:维奈克拉联合小剂量阿糖胞苷治疗初诊AML患者安全有效,治疗后有反应且维持治疗的患者有较好的生存结果。Objective:To evaluate the therapeutic efficacy,safety,and factors influencing overall survival(OS)in patients with newly diagnosed acute myeloid leukemia(unfit AML)treated with venetoclax(VEN)plus low-dose cytarabine(LDAC)treatment regimen.Methods:In this study,we retrospectively analyzed clinical data from 33 patients with unfit AML treated with VEN plus LDAC at the Affiliated Hospital of Jiangsu University between December 2019 and January 2024.The efficacy and survival outcomes of this regimen were assessed.Results:Thirty-three patients(median age:72 years)were enrolled,including 29 with de novo AML and four with secondary AML.The median followup duration was 781 days,with a median OS and progression-free survival(PFS)of 467 days(range:104–812 days)and 395 days(range:104–637 days),respectively.After induction chemotherapy,the overall response rate(ORR)was 69.7%,with a composite rate of complete response(CR)and CR with incomplete blood count recovery(CRi)reaching 36.4%.Morphologic leukemia-free state(MLFS)and partial remission(PR)were observed in 3%and 30.3%of patients,respectively.The median number of treatment cycles was three(range:1–6.5).Treatment-related adverse events were primarily hematological,with high rates of grade 3–4 hematologic toxicities.Kaplan–Meier analysis revealed significant associations between survival and ECOG performance status,TP53 mutation status,treatment cycles,and response(P<0.05).Univariate and multivariate Cox regression analyses identified treatment cycles as an independent risk factor for OS(P<0.05).Conclusions:The VEN plus LDAC regimen demonstrated feasibility and efficacy in patients with newly diagnosed AML;initial response and continuous treatment were associated with favorable survival.

关 键 词：维奈克拉 低剂量阿糖胞苷 初诊急性髓系白血病 

分 类 号：R73[医药卫生—肿瘤]