Mavacamten治疗肥厚型心肌病的研究进展  

Mavacamten in Treatment of Hypertrophic Cardiomyopathy

作　　者：蓝庆肃 武锋超 马兰虎 韩冰 徐金[1] 彭瑜 张钲[2,3] LAN Qingsu;WU Fengchao;MA Lanhu;HAN Bing;XU Jin;PENG Yu;ZHANG Zheng(The First Clinical Medical College,Lanzhou University,Lanzhou 730000,Gansu,China;Heart Center,The First Hospital of Lanzhou University,Lanzhou 730000,Gansu,China;Key Laboratory of Cardiovascular Diseases of Gansu Province,Lanzhou 730000,Gansu,China)

机构地区：[1]兰州大学第一临床医学院,甘肃兰州730000 [2]兰州大学第一医院心脏中心,甘肃兰州730000 [3]甘肃省心血管疾病重点实验室,甘肃兰州730000

出　　处：《心血管病学进展》2021年第11期1028-1031,共4页Advances in Cardiovascular Diseases

基　　金：科技部国家重点研发计划项目(2018YFC1311505);甘肃省心血管疾病重点实验室建设项目(1206RTSA025);甘肃省心血管病临床医学研究中心(甘科技〔2018〕20号);2020兰州大学中央高校基本科研专项资金(lzujbky-2020-kb18)。

摘　　要：肥厚型心肌病是一种常见的遗传性心肌病,通常由编码肌节相关蛋白质的基因突变所引起,主要表现为劳力性呼吸困难、胸痛及晕厥等。肥厚型心肌病已报道半个多世纪,但在药物治疗上无较大的进展,直至肌球蛋白靶向抑制剂mavacamten的出现,为不同类型的肥厚型心肌病患者带来了曙光,是遗传性心肌病精准治疗的一大进步。现对国外有关mavacamten治疗肥厚型心肌病的研究进展进行综述。Hypertrophic cardiomyopathy(HCM),a common hereditary cardiomyopathy,is usually caused by a mutation in a gene that codes for sarcomere related proteins.The main manifestations were exertional dyspnea,pectoralgia and syncope.HCM has been reported for more than half a century,but has not made great progress in drug therapy.Until the appearance of the myosin targeted inhibitor mavacamten,it brings the dawn for patients with different types of HCM,which is a great progress in the precise treatment of hereditary cardiomyopathy.In this article,we will summarize the foreign research on mavacamten in the treatment of HCM.

关 键 词：Mavacamten 肥厚型心肌病 肌球蛋白 N末端脑钠肽前体 

分 类 号：R542.2[医药卫生—心血管疾病]