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杂合性FGFR1突变可能是导致骨舌发育不全不完全形式的原因,其仅表现为透射性骨病变和牙齿滞留。

Heterozygous FGFR1 mutation may be responsible for an incomplete form of osteoglophonic dysplasia, characterized only by radiolucent bone lesions and teeth retentions.

作者信息

Marzin Pauline, Baujat Geneviève, Gensburger Déborah, Huber Céline, Bole Christine, Panuel Michel, Finidori Georges, De la Dure Muriel, Cormier-Daire Valérie

机构信息

Department of Medical Genetics, INSERM UMR 1163, Paris Descartes-Sorbonne Paris Cité University, IMAGINE Institute, Necker Enfants Malades Hospital, Paris, France.

Department of Rheumatology, Edouard Herriot Hospital, Lyon, France.

出版信息

Eur J Med Genet. 2020 Feb;63(2):103729. doi: 10.1016/j.ejmg.2019.103729. Epub 2019 Jul 15.

Abstract

Non-ossifying fibromas are seen in different disorders recognizable by specific features. Indeed, osteoglophonic dysplasia (OD) is characterized by radiolucent bone lesions associated with severe short stature, dysmorphism and failure of dental eruption. This syndrome is caused by heterozygous activating mutations in the immunoglobulin-like D3 domain of the FGFR1 gene, encoding a tyrosine kinase. Here, we report three patients from the same family presenting with radiolucent bone lesions and teeth retentions. Exome sequencing allowed identification of a novel mutation c.917C > T, p. Pro306Leu in exon 7 of the FGFR1 gene. Our patients present with normal stature and no severe dysmorphism. This report describes a mild form of OD and expands the phenotype related to FGFR1 mutations. These findings emphasize the need to consider FGFR1 variants in the case of multiple non-ossifying bone lesions associated with dental eruption anomalies.

摘要

非骨化性纤维瘤可见于具有特定特征的不同疾病中。事实上,骨舌发育异常(OD)的特征是与严重身材矮小、畸形和牙齿萌出障碍相关的透光性骨病变。该综合征由编码酪氨酸激酶的FGFR1基因免疫球蛋白样D3结构域中的杂合激活突变引起。在此,我们报告了来自同一家庭的三名患者,他们表现出透光性骨病变和牙齿滞留。外显子组测序确定了FGFR1基因第7外显子中的一个新突变c.917C>T,p.Pro306Leu。我们的患者身材正常,无严重畸形。本报告描述了一种轻度形式的OD,并扩展了与FGFR1突变相关的表型。这些发现强调,在出现与牙齿萌出异常相关的多发性非骨化性骨病变时,需要考虑FGFR1变异。

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