Mavacamten在肥厚型心肌病中的治疗进展  

Advances in the Treatment of Mavacamten in Hypertrophic Cardiomyopathy

作　　者：李莎 严霜霜 熊峰 LI Sha;YAN Shuangshuang;XIONG Feng(Department of Rehabilitation Medicine,The Third People’s Hospital of Chengdu,The Affiliated Hospital of Southwest Jiaotong University,Clincal College of Southwest Jiaotong University,Chengdu 610031,Sichuan,China;Department of Cardiovascular Medicine,The Third People’s Hospital of Chengdu,The Affiliated Hospital of Southwest Jiaotong University,Clincal College of Southwest Jiaotong University,Chengdu 610031,Sichuan,China)

机构地区：[1]成都市第三人民医院康复医学科西南交通大学附属医院西南交通大学临床医学院,四川成都610031 [2]成都市第三人民医院心血管内科西南交通大学附属医院西南交通大学临床医学院,四川成都610031

出　　处：《心血管病学进展》2024年第10期907-911,917,共6页Advances in Cardiovascular Diseases

基　　金：国家自然科学基金(82270486);四川省自然科学基金面上项目(24NSFSC0262)。

摘　　要：肥厚型心肌病(HCM)是一种主要由编码心肌肌小节蛋白的致病性遗传基因突变引起的一种原发性心肌疾病。由于HCM发病机制主要是肌小节蛋白编码基因变异,因此常规药物很难从根本上解决心肌肥厚所导致的一系列临床症候群。新型的心肌肌球蛋白抑制剂Mavacamten(MYK-461)是一种针对HCM的靶向治疗药物,通过减少过度的肌球蛋白-肌动蛋白交叉桥接来减轻心肌过度收缩。Mavacamten是一种心脏特异性肌球蛋白腺苷三磷酸酶的变构抑制剂,有望为HCM的治疗带来改变。现对Mavacamten的作用机制及临床研究进展做一综述。Hypertrophic cardiomyopathy(HCM)is a primary myocardial disease caused by a pathogenic genetic mutation encoding sarcoprotein.Since HCM pathogenesis is dominated by mybar protein-coding gene variation,it is difficult for conventional drugs to solve a series of clinical syndromes caused by cardiac hypertrophic cardiomyopathy.The novel myocardial myosin inhibitor Mavacamten(MYK-461)is a targeted therapy for hypertrophic cardiomyopathy that alleviates myocardial hypercontraction by reducing excessive myosin-actin cross-bridging.It is a cardiac specific myosin adenosine triphosphatase allosteric inhibitor,which is expected to take changes in the treatment of hypertrophic cardiomyopathy.This article reviews the mechanism of action and clinical research progress of Mavacamten.

关 键 词：肥厚型心肌病 梗阻性肥厚型心肌病 心肌肌球蛋白抑制剂 Mavacamten 

分 类 号：R542.2[医药卫生—心血管疾病]