Michels M, Olivotto I, Asselbergs F W, van der Velden J
Thoraxcenter, Department of Cardiology, Erasmus MC, Rotterdam, The Netherlands.
Careggi University Hospital, Florence, Italy.
Neth Heart J. 2017 Mar;25(3):186-199. doi: 10.1007/s12471-016-0943-2.
Hypertrophic cardiomyopathy (HCM) is the most common genetic heart disease, characterised by complex pathophysiology and extensive genetic and clinical heterogeneity. In most patients, HCM is caused by mutations in cardiac sarcomere protein genes and inherited as an autosomal dominant trait. The clinical phenotype ranges from severe presentations at a young age to lack of left ventricular hypertrophy in genotype-positive individuals. No preventative treatment is available as the sequence and causality of the pathomechanisms that initiate and exacerbate HCM are unknown. Sudden cardiac death and end-stage heart failure are devastating expressions of this disease. Contemporary management including surgical myectomy and implantable cardiac defibrillators has shown significant impact on long-term prognosis. However, timely recognition of specific scenarios - including transition to the end-stage phase - may be challenging due to limited awareness of the progression patterns of HCM. This in turn may lead to missed therapeutic opportunities. To illustrate these difficulties, we describe two HCM patients who progressed from the typical hyperdynamic stage of asymmetric septal thickening to end-stage heart failure with severely reduced ejection fraction. We highlight the different stages of this complex inherited cardiomyopathy based on the clinical staging proposed by Olivotto and colleagues. In this way, we aim to provide a practical guide for clinicians and hope to increase awareness for this common form of cardiac disease.
肥厚型心肌病(HCM)是最常见的遗传性心脏病,其病理生理复杂,具有广泛的遗传和临床异质性。在大多数患者中,HCM由心脏肌节蛋白基因突变引起,并作为常染色体显性性状遗传。临床表型范围从年轻时的严重表现到基因型阳性个体中无左心室肥厚。由于引发和加重HCM的病理机制的顺序和因果关系尚不清楚,因此没有预防性治疗方法。心源性猝死和终末期心力衰竭是这种疾病的毁灭性表现。包括外科心肌切除术和植入式心脏除颤器在内的当代治疗方法已显示出对长期预后有显著影响。然而,由于对HCM进展模式的认识有限,及时识别特定情况(包括向终末期阶段的转变)可能具有挑战性。这反过来可能导致错过治疗机会。为了说明这些困难,我们描述了两名HCM患者,他们从典型的非对称性室间隔增厚的高动力阶段进展为射血分数严重降低的终末期心力衰竭。我们根据Olivotto及其同事提出的临床分期,突出了这种复杂遗传性心肌病的不同阶段。通过这种方式,我们旨在为临床医生提供一份实用指南,并希望提高对这种常见心脏病形式的认识。