维奈克拉联合大剂量阿糖胞苷治疗复发/难治性急性髓系白血病的临床分析  

Clinical observation of venetoclax combined with high-dose cytarabine in relapsed/refractory AML

作　　者：雷小茹 戴进前[1] 李巧燕 史瑞 温静 吴雯 任婧婧[1] 李光[1] 宋艳萍[1] LEI Xiaoru;DAI Jinqian;LI Qiaoyan;SHI Rui;WEN Jing;WU Wen;REN Jingjing;LI Guang;SONG Yanping(Institute of Hematology of Xi'an,Xi'an Central Hospital,Shaanxi Xi'an 710004,China)

机构地区：[1]西安市中心医院西安市血液病研究所,陕西西安710004

出　　处：《现代肿瘤医学》2024年第16期3060-3065,共6页Journal of Modern Oncology

基　　金：陕西省西安市科技计划项目(编号:21YXYJ0017)。

摘　　要：目的:探讨维奈克拉(VEN)联合大剂量阿糖胞苷(HiDAC)方案治疗复发/难治性急性髓系白血病(R/R AML)的临床疗效和安全性。方法:回顾性分析2019年6月至2023年11月期间在我院采用VEN+HiDAC再诱导治疗的31例成人R/R AML患者的临床资料,评估其疗效、不良反应及生存情况,并探讨影响疗效和生存的因素。结果:31例患者的总反应率(ORR)为77.4%(24例),其中综合完全缓解(CRc,CR/CRi/MLFS)20例,部分缓解(PR)4例。复发、难治状态对ORR及CRc无明确影响(P>0.05),两组总生存期(OS)和无事件生存期(EFS)差异无统计学意义(P>0.05)。患者总体OS为10.8(3.5~36)个月,EFS为8.5(0.5~32)个月。生存分析结果显示,初次治疗有反应的患者具有更优的OS及EFS(P<0.01)。桥接异基因造血干细胞移植(allo-HSCT)(13例)较继续接受化疗的患者(18例),OS及EFS更佳(P<0.01)。患者不良反应主要是骨髓抑制,所有患者均出现≥Ⅲ级血液学毒性,感染、胃肠道反应较为常见,但患者均可耐受,未发生治疗相关死亡事件。结论:VEN+HiDAC方案是R/R AML患者有效的挽救性治疗方案,耐受性较好,桥接allo-HSCT可改善患者的远期生存。Objective:To investigate the efficacy and safety of venetoclax(VEN)combined with high-dose cytarabine(HiDAC)regimen for relapsed or refractory acute myeloid leukemia(R/R AML).Methods:The clinical data of 31 adult R/R AML patients who received the combination of VEN with HiDAC in our institute from June 2019 to November 2023 were retrospectively analyzed.To evaluate the treatment response,adverse events and survival,and explore the factors influencing the efficacy and survival.Results:The overall response rate(ORR)of 31 patients was 77.4%(24 cases),including 20 cases of complete response(CR)and CR with incomplete count recovery(CRi)and morphological leukemic free status(MLFS).4 cases of partial response(PR).Relapse and refractory status had no significant effect on ORR and CRc(P>0.05),with no statistically significant difference in overall survival(OS)and event-free survival(EFS)between the two groups(P>0.05).The median OS of all the patients was 10.8(3.5~36)months and median EFS was 8.5(0.5~32)months.The survival analysis showed that the patients who responded to the first treatment had a better OS and EFS(P<0.01).The patients(13 cases)who treated with bridge allogeneic hematopoietic stem cell transplantation(allo-HSCT)had superior OS and EFS than the patients(18 cases)treated with continue chemotherapy(both P<0.01).Adverse effects were mainly bone marrow suppression.All patients had experienced≥Ⅲgrade hematologic toxicity.Infection and gastrointestinal discomfort was common,but these could be all tolerated by patients.There were no treatment related deaths.Conclusion:VEN combined with HiDAC regimen is an effective salvage therapy for patients with R/R AML and is well tolerated by patients.Bridging allo-HSCT can improve long-term survival of patients.

关 键 词：维奈克拉 阿糖胞苷 复发/难治性急性髓系白血病 

分 类 号：R733.7[医药卫生—肿瘤]