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伴有心肌病的家族性扩张型心肌病相关 FLNC 致病性变异:患病率及基因型-表型相关性。

FLNC pathogenic variants in patients with cardiomyopathies: Prevalence and genotype-phenotype correlations.

机构信息

APHP, UF Cardiogénétique et Myogénétique Moléculaire et Cellulaire, Service de Biochimie Métabolique, Hôpitaux Universitaires de la Pitié- Salpêtrière- Charles Foix, Paris, France.

Sorbonne Université, UPMC Univ., INSERM, UMR_S 1166 and ICAN Institute for Cardiometabolism and Nutrition, Paris, France.

出版信息

Clin Genet. 2019 Oct;96(4):317-329. doi: 10.1111/cge.13594. Epub 2019 Jul 18.

Abstract

Pathogenic variants in FLNC encoding filamin C have been firstly reported to cause myopathies, and were recently linked to isolated cardiac phenotypes. Our aim was to estimate the prevalence of FLNC pathogenic variants in subtypes of cardiomyopathies and to study the relations between phenotype and genotype. DNAs from a cohort of 1150 unrelated index-patients with isolated cardiomyopathy (700 hypertrophic, 300 dilated, 50 restrictive cardiomyopathies, and 100 left ventricle non-compactions) have been sequenced on a custom panel of 51 cardiomyopathy disease-causing genes. An FLNC pathogenic variant was identified in 28 patients corresponding to a prevalence ranging from 1% to 8% depending on the cardiomyopathy subtype. Truncating variants were always identified in patients with dilated cardiomyopathy, while missense or in-frame indel variants were found in other phenotypes. A personal or family history of sudden cardiac death (SCD) was significantly higher in patients with truncating variants than in patients carrying missense variants (P = .01). This work reported the first observation of a left ventricular non-compaction associated with a unique probably causal variant in FLNC which highlights the role of FLNC in cardiomyopathies. A correlation between the nature of the variant and the cardiomyopathy subtype was observed as well as with SCD risk.

摘要

FLNC 编码细丝蛋白 C 的致病性变异首先被报道可导致肌病,最近与孤立性心脏表型相关。我们的目的是评估 FLNC 致病性变异在不同类型心肌病中的发生率,并研究表型与基因型之间的关系。对 1150 名无关的孤立性心肌病(700 例肥厚型心肌病、300 例扩张型心肌病、50 例限制型心肌病和 100 例左心室致密化不全心肌病)的先证者 DNA 进行了 51 个心肌病致病基因的定制面板测序。在 28 名患者中发现了 1 种 FLNC 致病性变异,其患病率在不同心肌病亚型中从 1%到 8%不等。截断变异总是在扩张型心肌病患者中发现,而错义或框内缺失变异则在其他表型中发现。携带截断变异的患者的个人或家族性心脏性猝死(SCD)病史明显高于携带错义变异的患者(P = 0.01)。本研究首次观察到左心室致密化不全与 FLNC 中独特的可能致病变异相关,这突显了 FLNC 在心肌病中的作用。还观察到变异的性质与心肌病亚型以及 SCD 风险之间存在相关性。

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