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产前诊断为 ACVRL1 基因突变致静脉性脑-心-视网膜血管发育不良

An ACVRL1 gene mutation presenting as vein of Galen malformation at prenatal diagnosis.

机构信息

Department of Obstetrics and Gynecology, University Hospital Brugmann, Université Libre de Bruxelles, Brussels, Belgium.

Department of Radiology, University Hospital Brugmann, Université Libre de Bruxelles, Brussels, Belgium.

出版信息

Am J Med Genet A. 2020 May;182(5):1255-1258. doi: 10.1002/ajmg.a.61535. Epub 2020 Mar 14.

Abstract

Hereditary hemorrhagic telangiectasia (HHT) is a rare autosomal dominant disease. The diagnostic criteria of HHT, or Curaçao criteria, include the following: recurrent epistaxis or nighttime nose bleeding, mucocutaneous telangiectases, visceral arteriovenous malformation, or an appropriate family history. The diagnosis is classified as definite if three criteria are present, possible if two criteria are present, and unlikely if only one is present. Nowadays, the confirmation of HHT diagnosis is based on molecular genetic studies. It has been showed that only mutations of genes encoding proteins within the transforming growth factor beta signaling pathway were responsible for the manifestation of the disease. The vein of Galen malformation (VOGM) as a presenting sign of HHT is rare. The prenatal diagnosis of HHT is even rarer. Herein, we present a case of prenatally diagnosed case of HHT based on the presence of VOGM in the fetus. To our knowledge, it is the first time that the gene mutation discovered in this case manifested as VOGM in the fetal life.

摘要

遗传性出血性毛细血管扩张症(HHT)是一种罕见的常染色体显性遗传疾病。HHT 的诊断标准,即 Curacao 标准,包括以下内容:反复发作的鼻出血或夜间鼻出血、黏膜皮肤毛细血管扩张、内脏动静脉畸形或适当的家族史。如果存在三个标准,则诊断为明确;如果存在两个标准,则诊断为可能;如果仅存在一个标准,则诊断为不太可能。如今,HHT 的诊断确认基于分子遗传学研究。已经表明,只有编码转化生长因子 β 信号通路蛋白的基因突变才会导致该疾病的表现。以 Galen 静脉畸形(VOGM)为表现的 HHT 很少见。HHT 的产前诊断更为罕见。在此,我们介绍了一例基于胎儿中存在 VOGM 而产前诊断的 HHT 病例。据我们所知,这是首例在此病例中发现的基因突变在胎儿期表现为 VOGM。

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