Unit of Inherited and Rare Cardiovascular Diseases, Onassis Cardiac Surgery Center, Athens, Greece.
Cardiology Department, Athens General Hospital "G. Gennimatas", Athens, Greece.
Heart Fail Rev. 2022 Jul;27(4):1173-1191. doi: 10.1007/s10741-021-10139-0. Epub 2021 Jul 14.
Dilated cardiomyopathy (DCM) is an umbrella term entailing a wide variety of genetic and non-genetic etiologies, leading to left ventricular systolic dysfunction and dilatation, not explained by abnormal loading conditions or coronary artery disease. The clinical presentation can vary from asymptomatic to heart failure symptoms or sudden cardiac death (SCD) even in previously asymptomatic individuals. In the last 2 decades, there has been striking progress in the understanding of the complex genetic basis of DCM, with the discovery of additional genes and genotype-phenotype correlation studies. Rigorous clinical work-up of DCM patients, meticulous family screening, and the implementation of advanced imaging techniques pave the way for a more efficient and earlier diagnosis as well as more precise indications for implantable cardioverter defibrillator implantation and prevention of SCD. In the era of precision medicine, genotype-directed therapies have started to emerge. In this review, we focus on updates of the genetic background of DCM, characteristic phenotypes caused by recently described pathogenic variants, specific indications for prevention of SCD in those individuals and genotype-directed treatments under development. Finally, the latest developments in distinguishing athletic heart syndrome from subclinical DCM are described.
扩张型心肌病(DCM)是一个涵盖了广泛的遗传和非遗传病因的总称,导致左心室收缩功能障碍和扩张,不能用异常负荷条件或冠状动脉疾病来解释。临床表现从无症状到心力衰竭症状或心脏性猝死(SCD)不等,即使在以前无症状的个体中也是如此。在过去的 20 年中,人们对 DCM 的复杂遗传基础有了显著的认识进展,发现了其他基因和基因型-表型相关性研究。对 DCM 患者进行严格的临床检查、细致的家族筛查以及先进的成像技术的应用,为更高效、更早的诊断以及更精确的植入式心脏复律除颤器植入和预防 SCD 提供了可能。在精准医学时代,基于基因型的治疗方法已经开始出现。在这篇综述中,我们重点介绍了 DCM 的遗传背景的最新进展、最近描述的致病性变异引起的特征性表型、这些个体中预防 SCD 的特定适应证以及正在开发的基于基因型的治疗方法。最后,还描述了区分运动员心脏综合征和亚临床 DCM 的最新进展。