新药时代骨髓增生异常综合征的治疗研究进展  

Research progress on treatment of myelodysplastic syndrome in the new drug era

作　　者：李娜[1] 张润泽 姚海英[2] LI Na;ZHANG Runze;YAO Haiying(Chengde Medical College,Chengde,Hebei,067000;Department of Hematology,Baoding City First Central Hospital of Hebei Province,Baoding,Hebei,071000)

机构地区：[1]承德医学院,河北承德067000 [2]河北省保定市第一中心医院血液科,河北保定071000

出　　处：《实用临床医药杂志》2021年第7期128-132,共5页Journal of Clinical Medicine in Practice

摘　　要：骨髓增生异常综合征(MDS)是克隆性造血干细胞疾病,具有向急性髓细胞白血病转化的高风险。既往MDS的治疗以去甲基化药物、造血干细胞移植为主。近年来,随着基因测序的发展,一些针对基因突变的靶向药物在MDS治疗中具有很好的前景。Bcl-2抑制剂venetoclax、免疫检测点阻断剂、Toll样受体抗体tomaralimab、端粒酶抑制剂imetelstat以及转化生长因子-β抑制剂luspatercept、galunisertib的上市,使得MDS的治疗获得突破性的进展。异柠檬酸脱氢酶抑制剂ivosidenib(AG-120)和enasidenib(AG-221)也初现成效。本研究对MDS患者个体化治疗方案进行综述。Myelodysplastic syndrome(MDS)is a clonal hematopoietic stem cell disease,which has a high risk of transforming into acute myeloid leukemia.In the past,MDS was mainly treated with demethylation drugs and hematopoietic stem cell transplantation.In recent years,with the development of gene sequencing,some targeted drugs for gene mutation have good prospects in the treatment of MDS.The marketing of venetoclax knew as Bcl-2 inhibitor,immune checkpoint blocker,tomaralimab knew as Toll like receptor antibody,imetelstat knew as telomerase inhibitor,and luspatercept and galunisertib knew as transforming growth factor-βinhibitors make a breakthrough in the treatment of MDS.The ivosidenib(AG-120)and enasidenib(AG-221)knew as inhibitors of isocitrate dehydrogenase are also effective.This study reviewed the individualized treatment of MDS patients.

关 键 词：骨髓增生异常综合征 靶向治疗 临床试验 Toll样受体抗体 端粒酶抑制剂 异柠檬酸脱氢酶 

分 类 号：R551.3[医药卫生—血液循环系统疾病] R457[医药卫生—内科学]