Lehalle Daphné, Altunoglu Umut, Bruel Ange-Line, Arnaud Eric, Blanchet Patricia, Choi Jong-Woo, Désir Julie, Kiliç Esra, Lederer Damien, Pinson Lucile, Thauvin-Robinet Christel, Singer Amihood, Thevenon Julien, Callier Patrick, Kayserili Hulya, Faivre Laurence
Equipe GAD, INSERM LNC UMR 1231, Faculté de Médecine, Université de Bourgogne Franche-Comté, Dijon, France.
Centre de Génétique et Centre de Référence Anomalies du Développement et Syndromes Malformatifs de l'Interrégion Est, Centre Hospitalier Universitaire Dijon, Dijon, France.
Am J Med Genet A. 2017 Dec;173(12):3136-3142. doi: 10.1002/ajmg.a.38490.
Frontonasal dysplasias are rare congenital malformations of frontonasal process-derived structures, characterized by median cleft, nasal anomalies, widely spaced eyes, and cranium bifidum occultum. Several entities of syndromic frontonasal dysplasia have been described, among which, to date, only a few have identified molecular bases. We clinically ascertained a cohort of 124 individuals referred for frontonasal dysplasia. We identified six individuals with a similar phenotype, including one discordant monozygous twin. Facial features were remarkable by nasal deformity with creased ridge and depressed or absent tip, widely spaced eyes, almond-shaped palpebral fissures, and downturned corners of the mouth. All had apparently normal psychomotor development. In addition, upper limb anomalies, frontonasal encephalocele, corpus callosum agenesis, choanal atresia, and congenital heart defect were observed. We identified five reports in the literature of patients presenting with the same phenotype. Exome sequencing was performed on DNA extracted from blood of two individuals, no candidate gene was identified. In conclusion, we report six novel simplex individuals presenting with a specific frontonasal dysplasia entity associating recognizable facial features, limb and visceral malformations, and apparently normal development. The identification of discordant monozygotic twins supports the hypothesis of a mosaic disorder. Although previous patients have been reported, this is the first series, allowing delineation of a clinical subtype of frontonasal dysplasia, paving the way toward the identification of its molecular etiology.
额鼻发育异常是源自额鼻突结构的罕见先天性畸形,其特征为正中裂、鼻异常、眼距增宽和隐性颅骨裂。已经描述了几种综合征性额鼻发育异常的实体,其中,迄今为止,只有少数确定了分子基础。我们对124例因额鼻发育异常前来就诊的个体进行了临床确诊。我们确定了6例具有相似表型的个体,包括一对不一致的同卵双胞胎。面部特征显著,表现为鼻畸形,有皱嵴、鼻尖凹陷或缺失、眼距增宽、杏仁状睑裂以及嘴角下垂。所有患者的精神运动发育明显正常。此外,还观察到上肢异常、额鼻脑膨出、胼胝体发育不全、后鼻孔闭锁和先天性心脏缺陷。我们在文献中找到了5例具有相同表型患者的报告。对从两名个体血液中提取的DNA进行了外显子组测序,未发现候选基因。总之,我们报告了6例新的散发病例,这些个体表现出一种特定的额鼻发育异常实体,伴有可识别的面部特征、肢体和内脏畸形,且发育明显正常。不一致的同卵双胞胎的发现支持了镶嵌性疾病的假说。尽管之前已有患者报告,但这是首个系列病例,有助于明确额鼻发育异常的一种临床亚型,为确定其分子病因铺平道路。