阿扎胞苷治疗骨髓增生异常综合征合并额外性梳样蛋白1基因突变疗效及安全性分析  

Efficacy and safety of azacitidine in treatment of MDS with ASXL1 gene mutation

作　　者：李振波 陆诚 LI Zhenbo;LU Cheng(Department of Hematology,Baoji High-Tech Hospital,Baoji 721000,China)

机构地区：[1]宝鸡市高新医院血液科,陕西宝鸡721000 [2]宝鸡市中医医院肿瘤血液科,陕西宝鸡721001

出　　处：《陕西医学杂志》2021年第9期1135-1138,1142,共5页Shaanxi Medical Journal

基　　金：陕西省重点研发计划项目(2018SF-054)。

摘　　要：目的:探究阿扎胞苷(AZA)治疗骨髓增生异常综合征(MDS)合并额外性梳样蛋白1(ASXL1)基因突变的疗效及安全性。方法:选取MDS合并ASXL1基因突变患者40例,依据采用的治疗方案不同分为观察组(17例)和对照组(23例)。观察组单用AZA治疗。对照组使用减量CAG方案[阿糖胞苷(Ara-C)+阿克拉霉素(ACR)+粒细胞集落刺激因子(G-CSF)]联合地西他滨治疗。比较两组疗效、不良反应、预后危险度及生存情况。结果:治疗1、3、6个疗程后,观察组总有效率(ORR)均略低于对照组,但组间比较差异无统计学意义(均P>0.05)。观察组Ⅲ-Ⅳ级血红蛋白减少、血小板减少、中性粒细胞绝对值减少发生率低于对照组(均P<0.05)。两组预后危险度构成比较差异无统计学意义(P>0.05)。观察组总生存时间(OS)较对照组缩短(P<0.05)。结论:AZA治疗MDS合并ASXL1基因突变有一定疗效及安全性,但在改善患者预后方面不及减量CAG联合地西他滨方案。Objective:To explore the efficacy and safety of azacitidine(AZA)in the treatment of myelodysplastic syndrome(MDS)with additional sex combs-like 1(ASXL1)gene mutation.Methods:Forty MDS patients with ASXL1 gene mutation were selected and divided into observation group(17 cases)and control group(23 cases)according to the different treatment schemes adopted.The observation group was treated with AZA alone.The control group was treated with decrease CAG regimen combined with decitabine.The efficacy,adverse reactions,prognostic risk and survival were compared between the two groups.Results:After 1,3,and 6 courses of treatment,the total response rate(ORR)of the observation group was slightly lower than that of the control group,but the difference between the two groups was not statistically significant(all P>0.05).The incidence of gradeⅢ-Ⅳhemoglobin reduction,thrombocytopenia,and absolute reduction of neutrophils in the observation group was lower than that in the control group(all P<0.05).There was no significant difference in the composition of the prognostic risk between the two groups(P>0.05).The overall survival time(OS)of the observation group was shorter than that of the control group(P<0.05).Conclusion:AZA has certain efficacy and safety in the treatment of MDS patients with ASXL1 gene mutation.However,it is inferior to decrease CAG regimen combined with decitabine in improving the prognosis.

关 键 词：骨髓增生异常综合征 基因突变 阿扎胞苷 额外性梳样蛋白1 疗效 安全性 

分 类 号：R551.3[医药卫生—血液循环系统疾病]