真实世界中维奈克拉联合方案一线治疗新诊断unfit-AML的疗效和影响因素  

Efficacy and factors influencing the first-line treatment of newly diagnosed unfit-AML with a combination regimen of venetoclax in the real world

作　　者：余国攀[1] 尹昭 蒋玲[1] 刘慧[1] 史鹏程[1] 金华 徐丹[1] 江雪杰[1] 张钰[1] Yu Guo-pan;Yin Zhao;Jiang Ling;Liu Hui;Shi Peng-cheng;Jin Hua;Xu Dan;Jiang Xue-jie;Zhang Yu(Department of Hematology,Southern Hospital,Southern Medical University,Guangzhou 510515,China)

机构地区：[1]南方医科大学南方医院血液科,广东广州510515

出　　处：《兰州大学学报（医学版）》2022年第12期34-40,46,共8页Journal of Lanzhou University（Medical Sciences）

基　　金：国家自然科学基金资助项目(82270178)。

摘　　要：目的探索真实世界中维奈克拉联合方案一线治疗新诊断不适合强化疗的急性髓系白血病(unfit-AML)的疗效和安全性,以及疗效的影响因素。方法回顾性分析南方医科大学南方医院2019-2022年维奈克拉联合方案一线治疗的73例unfit-AML患者的临床资料,分析疗效、安全性以及影响疗效的因素。结果维奈克拉联合方案以联合阿扎胞苷为主。完全缓解(CR)率为65.8%,53.6%获得微小残留病(MRD)阴性。CR和MRD阴转率均随着疗程增加逐渐增加。低危组、IDH1/2、NPM1、FLT3-ITD/TKD突变和维奈克拉足疗程使用的患者治疗反应较好。获得CR和MRD阴性患者的中位生存期(OS)显著优于未获得CR和MRD阳性患者的;策略性调整患者较维奈克拉联合方案持续治疗患者的复发率低、OS长。结论真实世界中维奈克拉联合方案一线治疗新诊断unfit-AML安全有效,疗效受遗传学特征、治疗策略等因素影响。Objective To explore the efficacy,safety and the factors influencing efficacy of first-line treatment of newly diagnosed unfit-acute myeloid leukemia(unfit-AML)in the real world with the venetoclax combination regimen.Methods Retrospective analysis was performed to examine the efficacy,safety and factors affecting the efficacy of 73 patients with unfit-AML treated in the first line with the combination regimen of venetoclax from 2019 to 2022 in Southern Hospital,Southern Medical University.Results The venetoclax combination regimen was dominated by the combination of Azacitidine.The complete response(CR)rate was 65.8%and 53.6%were negative for measurable residual disease(MRD).Both the CR and MRD negative rates increased gradually with increasing course of therapy.Patients in the low-risk group,with IDH1/2,NPM1,FLT3-ITD/TKD mutations,and with a full course of venetoclax had a better response to treatment.Median overall survival(OS)was significantly better in patients who achieved CR and MRD-negative than in those who did not,strategically adjusted patients had a lower relapse rate and longer OS than patients treated continuously with the venetoclax combination regimen.Conclusion The first-line treatment of newly diagnosed unfit-AML with the venetoclax combination regimen is safe and effective in the real world,and efficacy is influenced by genetic characteristics,treatment strategy,and other factors.

关 键 词：维奈克拉 急性髓系白血病 疗效 安全性 

分 类 号：R733.71[医药卫生—肿瘤]