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左心室致密化不全心肌病中的原纤蛋白-1基因突变

Fibrillin-1 Gene Mutations in Left Ventricular Non-compaction Cardiomyopathy.

作者信息

Parent John J, Towbin Jeffrey A, Jefferies John L

机构信息

Riley Hospital for Children at Indiana University Health, Indiana University School of Medicine, 705 Riley Hospital Drive, RR 127, Indianapolis, IN, 46202, USA.

Division of Pediatric Cardiology, The Heart Institute, Le Bonheur Children's Hospital, Memphis, TN, USA.

出版信息

Pediatr Cardiol. 2016 Aug;37(6):1123-6. doi: 10.1007/s00246-016-1404-9. Epub 2016 May 9.

Abstract

Left ventricular non-compaction cardiomyopathy (LVNC) is a unique cardiomyopathy with a current yield of about 30-40 % in identifying a causative gene mutation. A retrospective review of all patients with LVNC at our institution was performed and genetic testing was reviewed. Echocardiographic and cardiac magnetic resonance imaging was reviewed to corroborate the reported phenotype. We present a series of patients with LVNC dilated phenotype associated with fibrillin-1 gene mutations. Fifty-one patients were identified as having LVNC with reduced left ventricular function and/or left ventricular dilation. We retrospectively reviewed gene testing in this cohort when available and identified 5 patients (10 %) with an FBN1 gene mutation. Syndrome breakdown as follows: 3 with Marfan, 1 with Shprintzen-Goldberg, and 1 with no identifiable syndrome. Derangements in fibrillin-1 may impact the compaction process resulting in LVNC. Although causation has not been proven by our report, it certainly raises interest in a possible mechanistic relationship between fibrillin-1 and LVNC given the increased prevalence of Marfan syndrome and fibrillin-1 gene mutations in this cohort.

摘要

左心室心肌致密化不全心肌病(LVNC)是一种独特的心肌病,目前在识别致病基因突变方面的检出率约为30%-40%。我们对本机构所有LVNC患者进行了回顾性研究,并对基因检测结果进行了分析。回顾了超声心动图和心脏磁共振成像结果以证实所报告的表型。我们报告了一系列具有LVNC扩张型表型且与原纤蛋白-1基因突变相关的患者。51例患者被确诊为LVNC伴左心室功能降低和/或左心室扩张。我们对该队列中可获得基因检测结果的患者进行了回顾性分析,发现5例(10%)患者存在FBN1基因突变。综合征分类如下:3例患有马方综合征,1例患有施普伦策尔-戈德堡综合征,1例无明确综合征。原纤蛋白-1的紊乱可能会影响致密化过程,从而导致LVNC。尽管我们的报告尚未证实因果关系,但鉴于该队列中马方综合征和原纤蛋白-1基因突变的患病率增加,这无疑引发了人们对原纤蛋白-1与LVNC之间可能存在的机制关系的兴趣。

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