维奈克拉联合阿扎胞苷治疗骨髓增生异常综合征合并意义未明的单克隆球蛋白血症1例并文献复习  

Venetoclax combined with azacitidine in treatment of myelodysplastic syndromes complicated with monoclonal globulinemia of unknown significance:report of 1 case and review of literature

作　　者：翁光样 彭浩宇 游伟文[1] 刘焕勋[1] 蔡云[1] 杜新[1] Weng Guangyang;Peng Haoyu;You Weiwen;Liu Huanxun;Cai Yun;Du Xin(Department of Hematology,the Second People's Hospital of Shenzhen,Shenzhen 518000,China)

机构地区：[1]深圳市第二人民医院血液科,深圳518000

出　　处：《白血病．淋巴瘤》2022年第11期680-683,共4页Journal of Leukemia & Lymphoma

摘　　要：目的:探讨维奈克拉联合阿扎胞苷治疗骨髓增生异常综合征(MDS)合并意义未明的单克隆球蛋白血症(MGUS)患者的效果。方法:回顾性分析深圳市第二人民医院2020年12月收治的1例MDS合并MGUS患者的临床资料,并进行文献复习。结果:患者完善骨髓细胞学、细胞遗传学、二代测序检测后,诊断为MDS合并MGUS,伴ASXL1、RUNX1、EZH2、STAG2突变及t(11;14)。给予2个疗程阿扎胞苷、1个疗程阿扎胞苷联合HAG方案治疗后患者无治疗反应,改用维奈克拉联合阿扎胞苷治疗1个疗程后,疗效评估为完全缓解。RUNX1、STAG2突变转阴,M蛋白下降50%以上。随访至2021年9月仍为完全缓解状态。结论:维奈克拉联合阿扎胞苷治疗MDS合并MGUS伴t(11;14)患者效果显著,且耐受性好。Objective:To investigate the therapeutic effect of venetoclax combined with azacitidine in treatment of myelodysplastic syndromes (MDS) complicated with monoclonal globulinemia of unknown significance (MGUS).Methods:The clinical data of a patient with MDS complicated with MGUS in the Second People's Hospital of Shenzhen in December 2020 were retrospectively analyzed, and the literatures were reviewed.Results:According to results of bone marrow smear, cytogenetics, and next-generation sequencing, the patient was diagnosed as MDS and MGUS complicated with ASXL1, RUNX1, EZH2, STAG2 mutations as well as t(11;14). No response was observed after 2 courses of azacitidine and 1 course of azacitidine plus HAG. Later the patient achieved complete remission and negative RUNX1 and STAG2 mutations after a course of venetoclax combined with azacitidine. Meanwhile, M protein exhibited a decrease more than 50%. To date, the patient was still in complete remission.Conclusions:The regimen of venetoclax combined with azacitidine shows a significant efficacy and good tolerance to patient with co-occurrence of MDS and MGUS with t(11;14).

关 键 词：骨髓增生异常综合征 意义未明的单克隆球蛋白血症 维奈克拉 阿扎胞苷t(11 14) 

分 类 号：R551.3[医药卫生—血液循环系统疾病]