Venetoclax联合去甲基化药物治疗复发/难治性急性髓系白血病的疗效及预后分析  

Efficacy and prognosis analysis of venetoclax in combination with demethylation drugs for relapsed/refractory acute myeloid leukemia

作　　者：盘婉盈 张映璇 涂三芳[1] 黄睿[1] 郭梦琪 何颖芝[1] 杜静文 宋朝阳[1] 李玉华[1] 黄宇贤[1] PAN Wanying;ZHANG Yingxuan;TU Sanfang;HUANG Rui;GUO Mengqi;HE Yingzhi;DU Jingwen;SONG Chao yang;LI Yuhua;HUANG Yuxian(Department of Hematology,Zhujiang Hospital Affiliated to Southern Medical University,Guangzhou,510282,China;Southern Medical University)

机构地区：[1]南方医科大学珠江医院血液科,广州510282 [2]南方医科大学

出　　处：《临床血液学杂志》2021年第9期650-654,共5页Journal of Clinical Hematology

基　　金：广东省科技创新战略专项资金(“攀登计划”专项资金)(No:pdjh2020b0122)。

摘　　要：目的:评估venetoclax联合去甲基化药物治疗复发/难治性急性髓系白血病(AML)患者的疗效、不良反应及预后情况。方法:回顾性分析2018年7月—2020年12月接受venetoclax联合去甲基化药物的22例复发/难治性AML患者的临床资料。结果:中位随访时间9.86(95%CI 7.58~12.14)个月,19例患者获得最佳客观应答,其中7例达完全缓解,6例达骨髓完全缓解伴不完全血液学恢复,6例达部分缓解,3例患者对venetoclax联合治疗无反应。发生Ⅲ~Ⅳ级血小板计数下降、白细胞减少的患者分别为5例和10例;3例患者因Ⅲ~Ⅳ级血液学毒性停药。1例患者因过度疲乏停药。2例患者发生实验室定义的肿瘤溶解综合征,无一例临床肿瘤溶解综合征。12例患者死亡,中位总生存期为8.71(95%CI 4.65~12.77)个月,1年的预期累积总生存率为(36.6%±11.8%);7例患者复发,中位无事件生存期为5.23(95%CI 2.07~8.39)个月,1年的预期累积无事件生存率为(29.7%±11.0%)。结论:Venetoclax耐受性良好,具有较好的安全性。Venetoclax联合去甲基化药物的靶向治疗可使复发/难治性AML患者获得较高的短期缓解率,从而为造血干细胞移植提供有利的时间窗。Objective: To evaluate the efficacy, adverse events and prognosis of venetoclax in combination with demethylation drugs in treating patients with relapsed/refractory acute myeloid leukemia(AML). Methods: Clinical data of 22 patients with relapsed/refractory AML treated with venetoclax combined with demethylation drugs from July 2018 to December 2020 were retrospectively analyzed. Results: The median follow-up time was 9.86(95%CI 7.58-12.14) months. Nineteen patients achieved the best objective response, of whom 7 cases achieved complete remission, 6 cases achieved marrow complete remission with incomplete hematological recovery, and 6 cases achieved partial remission. Three patients did not respond to venetoclax combination treatment. There were 5 patients and 10 patients with grade Ⅲ-Ⅳ platelet count decline and leukopenia, respectively. Three patients were discontinued due to grade Ⅲ-Ⅳ hematological toxicity. One patient discontinued the drug due to excessive fatigue. Two patients developed laboratory-defined tumor lysis syndrome, and none of them had clinical tumor lysis syndrome. With 12 patients died, the median overall survival was 8.71(95%CI 4.65-12.77) months, the 1-year expected cumulative overall survival rate was(36.6%±11.8%). Seven patients relapsed, with a median event-free survival of 5.23(95%CI 2.07-8.39) months, and the 1-year expected cumulative event-free survival rate was(29.7%±11.0%). Conclusion: Venetoclax is well tolerated and of satisfactory safety. Targeted therapy with venetoclax in combination with demethylated drugs in patients with relapsed/refractory AML results in a higher short-term remission rate, thus providing a bridge for hematopoietic stem cell transplantation.

关 键 词：venetoclax 复发性 难治性 急性髓系白血病 疗效 预后 

分 类 号：R733.71[医药卫生—肿瘤]