治疗肥厚型心肌病的新型心肌肌球蛋白抑制剂——玛伐凯泰  

A novel cardiac myosin inhibitor for the treatment of hypertrophic cardiomyopathy:mavacamten

作　　者：张煜竹 肖玉涵 成柯润 刘凯[1] Yuzhu ZHANG;Yuhan XIAO;Kerun CHENG;Kai LIU(Department of Cardiology,West China Hospital of Sichuan University,Chengdu 610041,China;West China Medicine School,Sichuan University,Chengdu 610041,China)

机构地区：[1]四川大学华西医院心内科,成都610041 [2]四川大学华西临床医学院,成都610041

出　　处：《临床药物治疗杂志》2024年第10期16-20,共5页Clinical Medication Journal

摘　　要：肥厚型心肌病(HCM)是临床常见的心肌病,其中约2/3为梗阻性肥厚型心肌病(oHCM)。玛伐凯泰是第一个靶向心脏特异性肌球蛋白抑制剂,2022年4月美国FDA批准用于治疗美国纽约心脏病学会心功能分级Ⅱ~Ⅲ级的症状性梗阻性肥厚型心肌病成人患者。玛伐凯泰可逆地抑制肌动蛋白与肌球蛋白的结合,减少横桥周期,降低心肌高收缩力。近年Ⅱ期和Ⅲ期临床试验已证实玛伐凯泰可降低左心室流出道压力,改善心功能分级和运动耐力。本文对玛伐凯泰的作用机制、临床价值及安全性分析进行综述。Hypertrophic cardiomyopathy(HCM)is a common type of cardiomyopathy,with approximately two-thirds of cases presenting as obstructive hypertrophic cardiomyopathy(oHCM).Mavacamten,the first heart-specific myosin inhibitor,was approved by the U.S.FDA in April 2022 for the treatment of adult patients with symptomatic oHCM and heart function classified as NYHA classⅡtoⅢ.Mavacamten reversibly inhibits actin-myosin binding,reduces the cross-bridge cycle,and decreases myocardial hypercontractility.Recent phaseⅡand phaseⅢclinical trials have demonstrated that Mavacamten reduces left ventricular outflow tract pressure and improves both cardiac function and exercise tolerance.The mechanism of action,clinical value and safety profile of Mavacamten are reviewed.

关 键 词：肥厚型心肌病 玛伐凯泰 肌球蛋白抑制剂 药物治疗 

分 类 号：R972.9[医药卫生—药品]