Mathai Susan, Williams Lynne
Department of Cardiology, Royal Papworth Hospital NHS Foundation Trust, Cambridge, UK.
Department of Cardiology, Royal Papworth Hospital NHS Foundation Trust, Papworth Road, Cambridge Biomedical Campus, Cambridge CB2 0AY, UK.
Ther Adv Chronic Dis. 2022 Nov 15;13:20406223221136074. doi: 10.1177/20406223221136074. eCollection 2022.
Hypertrophic cardiomyopathy (HCM) is a common genetic disorder characterised by unexplained left ventricular hypertrophy. Left ventricular outflow tract obstruction is an integral component of the disease, often resulting in significant symptoms, but also carrying a risk of progression to heart failure and death. Advancements in our understanding of the pathophysiology of HCM have led to the development of new therapies targeting the molecular basis of the disease at the level of the cardiac sarcomere, the basic contractile apparatus of the myocardium. Myosin modulators are a novel class of small molecules which target cardiac myosins directly to modulate cardiac contractility. The myosin inhibitors present the first advancement in pharmacological management of obstructive HCM in almost 35 years, with a growing body of evidence for the safety, tolerability and efficacy of mavacamten, and to a lesser extent aficamten. The aim of this review is to summarise the current management of patients with obstructive HCM and review the most recent available data from clinical trials pertaining to myosin inhibition.
肥厚型心肌病（HCM）是一种常见的遗传性疾病，其特征为原因不明的左心室肥厚。左心室流出道梗阻是该疾病的一个重要组成部分，常导致明显症状，同时也存在进展为心力衰竭和死亡的风险。我们对HCM病理生理学认识的进展已促使针对心肌基本收缩装置——心肌肌节水平的疾病分子基础开发新的治疗方法。肌球蛋白调节剂是一类新型小分子，可直接作用于心肌肌球蛋白以调节心脏收缩力。肌球蛋白抑制剂是近35年来梗阻性HCM药物治疗方面的首个进展，越来越多的证据表明mavacamten以及程度稍轻的aficamten具有安全性、耐受性和有效性。本综述的目的是总结梗阻性HCM患者的当前治疗情况，并回顾与肌球蛋白抑制相关的临床试验的最新可用数据。
