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自动化重分析,一种诊断超罕见疾病的新方法:纤连蛋白 1 相关脊椎干骺端发育不良(SMD-FN1)。

Automated reanalysis, a novel way to diagnose an ultra-rare condition: Fibronectin-1-related spondylometaphyseal dysplasia (SMD-FN1).

机构信息

Clinical Genetics Registrar, Guy's and St Thomas' NHS Foundation Trust (London), Birmingham Women's and Children's Hospital NHS Trust & Birmingham Health Partners.

Medical Student, University of Birmingham, Birmingham.

出版信息

Clin Dysmorphol. 2021 Jul 1;30(3):154-158. doi: 10.1097/MCD.0000000000000369.

DOI:10.1097/MCD.0000000000000369
PMID:33605604
Abstract

We report a further case of spondylometaphyseal dysplasia - corner fracture type due to the fibronectin-1 gene (SMD-FN1) in a child originally thought to have metaphyseal chondrodysplasia-Brussels type (MCD Brussels). We highlight phenotypic differences with the SMD-FN1 published reports. This case is unique in terms of the method of molecular confirmation. Findings from the 100 000 Genomes Project were originally negative (in both tier 1 and 2); however, subsequent reanalysis, initiated by an automated search for new gene-disease associations in PanelApp, highlighted a candidate diagnostic variant. Our child had short stature, facial dysmorphism, spondylometaphyseal dysplasia and corner fractures and a heterozygous de novo missense variant in FN1 (c.675C>G p.(Cys225Trp), which was likely pathogenic. The variant matched the clinical and radiological features and a diagnosis of SMD-FN1 was confirmed. We explore the diagnostic journey of this patient, compare her findings with the previous 15 patients reported with SMD-FN1 and discuss the diagnostic utility of automated reanalysis. We consider differences and similarities between MCD Brussels and SMD-FN1, by reviewing literature on both conditions and assess whether they are in fact the same disorder.

摘要

我们报告了一例由于纤维连接蛋白 1 基因(SMD-FN1)导致的脊椎干骺端发育不良 - 角骨折型的病例,该患者最初被认为患有布鲁塞尔型干骺端软骨发育不良(MCD Brussels)。我们强调了与 SMD-FN1 已发表报告的表型差异。就分子确认方法而言,该病例是独特的。10 万基因组计划的结果最初为阴性(在第 1 层和第 2 层均为阴性);然而,随后由 PanelApp 自动搜索新的基因疾病关联发起的重新分析,突出了一个候选诊断变异。我们的孩子身材矮小、面部畸形、脊椎干骺端发育不良和角骨折,以及 FN1 中的杂合性新生错义变异(c.675C>G p.(Cys225Trp),这可能是致病性的。该变异与临床和影像学特征相匹配,从而确诊为 SMD-FN1。我们探讨了该患者的诊断过程,将她的发现与之前 15 例报道的 SMD-FN1 患者进行了比较,并讨论了自动重新分析的诊断效用。我们通过回顾这两种疾病的文献,来评估布鲁塞尔型干骺端软骨发育不良和脊椎干骺端发育不良 - 角骨折型的差异和相似之处,并评估它们是否实际上是同一疾病。

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Automated reanalysis, a novel way to diagnose an ultra-rare condition: Fibronectin-1-related spondylometaphyseal dysplasia (SMD-FN1).自动化重分析,一种诊断超罕见疾病的新方法:纤连蛋白 1 相关脊椎干骺端发育不良(SMD-FN1)。
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