Heart and Lung Center, Helsinki University Hospital, University of Helsinki, Helsinki, Finland.
Blueprint Genetics, Helsinki, Finland.
BMC Med Genet. 2020 Jan 31;21(1):19. doi: 10.1186/s12881-020-0955-z.
Dilated cardiomyopathy (DCM) is a condition characterized by dilatation and systolic dysfunction of the left ventricle in the absence of severe coronary artery disease or abnormal loading conditions. Mutations in the titin (TTN) and lamin A/C (LMNA) genes are the two most significant contributors in familial DCM. Previously mutations in the desmoplakin (DSP) gene have been associated with arrhythmogenic right ventricular cardiomyopathy (ARVC) and more recently with DCM.
We describe the cardiac phenotype related to a DSP mutation which was identified in ten unrelated Finnish index patients using next-generation sequencing. Sanger sequencing was used to verify the presence of this DSP variant in the probands' relatives. Medical records were obtained, and clinical evaluation was performed.
We identified DSP c.6310delA, p.(Thr2104Glnfs*12) variant in 17 individuals of which 11 (65%) fulfilled the DCM diagnostic criteria. This pathogenic variant presented with left ventricular dilatation, dysfunction and major ventricular arrhythmias. Two patients showed late gadolinium enhancement (LGE) and myocardial edema on cardiac magnetic resonance imaging (MRI) that may suggest inflammatory process at myocardium.
The patients diagnosed with DCM showed an arrhythmogenic phenotype as well as SCD at young age supporting the recently proposed concept of arrhythmogenic cardiomyopathy. This study also demonstrates relatively low penetrance of truncating DSP variant in the probands' family members by the age of 40. Further studies are needed to elucidate the possible relations between myocardial inflammation and pathogenic DSP variants.
扩张型心肌病(DCM)是一种以左心室扩张和收缩功能障碍为特征的疾病,不存在严重的冠状动脉疾病或异常负荷条件。肌联蛋白(TTN)和核纤层蛋白 A/C(LMNA)基因突变是家族性 DCM 的两个最重要的致病因素。先前,桥粒芯蛋白(DSP)基因突变与致心律失常性右室心肌病(ARVC)有关,最近与 DCM 有关。
我们使用下一代测序技术,在 10 名无关的芬兰索引患者中描述了与 DSP 基因突变相关的心脏表型。使用 Sanger 测序验证该 DSP 变体在先证者亲属中的存在。获取了病历并进行了临床评估。
我们在 17 个人中发现了 DSP c.6310delA,p.(Thr2104Glnfs*12)变体,其中 11 人(65%)符合 DCM 的诊断标准。这种致病性变体表现为左心室扩张、功能障碍和主要室性心律失常。两名患者在心脏磁共振成像(MRI)上显示出晚期钆增强(LGE)和心肌水肿,这可能表明心肌存在炎症过程。
诊断为 DCM 的患者表现出心律失常表型以及年轻时的 SCD,支持最近提出的心律失常性心肌病概念。本研究还表明,在 40 岁之前,截断性 DSP 变体在先证者家族成员中的外显率相对较低。需要进一步研究阐明心肌炎症与致病性 DSP 变体之间的可能关系。