The Hypertrophic Cardiomyopathy Center, Department of Cardiovascular Medicine, Cleveland Clinic Foundation, Cleveland, Ohio.
The Hypertrophic Cardiomyopathy Center, Department of Cardiovascular Medicine, Cleveland Clinic Foundation, Cleveland, Ohio.
Am J Cardiol. 2024 Feb 1;212S:S33-S41. doi: 10.1016/j.amjcard.2023.10.074. Epub 2024 Jan 29.
Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic heart disease inherited in an autosomal dominant pattern with an estimated prevalence of 0.6% in the general population. Clinical manifestations of HCM vary considerably, with symptoms ranging from none or mild exercise intolerance to severe lifestyle-limiting symptoms, advanced heart failure, and sudden cardiac death. Current management options for HCM include lifestyle modifications, familial screening with genetic counseling, pharmacotherapy for symptom control, sudden cardiac death risk stratification with or without defibrillator implantation, septal reduction therapy, and, in some cases, heart transplantation. Only recently have strongly targeted medical therapies for HCM, such as myosin inhibitors, been studied in multicenter randomized controlled trials. In this report, we review the currently available medical treatments for HCM and the future directions of HCM pharmacotherapy, and we highlight important unmet needs in this population.
肥厚型心肌病(HCM)是一种常染色体显性遗传的异质性基因心脏病,在普通人群中的患病率估计为 0.6%。HCM 的临床表现差异很大,症状从无或轻度运动不耐受到严重限制生活方式的症状、晚期心力衰竭和心源性猝死不等。HCM 的当前治疗选择包括生活方式改变、家族筛查和遗传咨询、药物治疗以控制症状、进行心脏性猝死风险分层并在必要时植入除颤器、室间隔减容治疗,以及在某些情况下进行心脏移植。直到最近,才在多中心随机对照试验中研究了针对 HCM 的强效靶向药物治疗,如肌球蛋白抑制剂。在本报告中,我们回顾了目前用于 HCM 的治疗方法以及 HCM 药物治疗的未来方向,并强调了该人群中存在的重要未满足需求。