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基于全外显子组测序鉴定的 3 型心面皮肤综合征病例:新技术在伴有神经发育迟缓儿童中的应用优势。

Whole exome sequencing based identification of a case of cardiofaciocutaneous syndrome type 3: the benefits of new sequencing technology in children with neurodevelopmental delay.

机构信息

Department of Paediatrics, Midland Regional Hospital, Mullingar, Ireland

Department of Paediatrics, Midland Regional Hospital, Mullingar, Ireland.

出版信息

BMJ Case Rep. 2022 Nov 8;15(11):e251871. doi: 10.1136/bcr-2022-251871.

DOI:10.1136/bcr-2022-251871
PMID:36351669
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9644300/
Abstract

We report the case of a boy with a prolonged diagnostic workup for global developmental delay alongside feeding difficulties, failure to thrive, pulmonary stenosis and macrocephaly. Following a series of diagnostic tests over the first 25 months of life, whole-exome sequencing was performed which diagnosed cardiofaciocutaenous syndrome type 3.Global developmental delay is a common presentation to general paediatric and community paediatric clinics. This prompts the search for an aetiology to describe the child's constellation of symptoms which often consists of a chromosomal microarray, neuroimaging and investigations for an inborn error of metabolism. With developments in genetic testing such as the reducing cost of clinical exome sequencing or whole-exome sequencing, could these testing strategies offer a more comprehensive first line test?This case not only demonstrates the features of cardiofaciocutaneous syndrome type 3 but the added value of modern genetic technologies in the diagnosis of children with global developmental delay.

摘要

我们报告了一例患有全球发育迟缓、喂养困难、生长不良、肺动脉瓣狭窄和大头畸形的男孩病例。在生命的头 25 个月内进行了一系列诊断性检查后,进行了全外显子组测序,诊断为 3 型心面皮肤综合征。全球发育迟缓是普通儿科和社区儿科诊所的常见表现。这促使人们寻找病因来描述孩子的一系列症状,这些症状通常包括染色体微阵列、神经影像学检查和先天性代谢缺陷的检查。随着基因检测技术的发展,如临床外显子组测序或全外显子组测序成本的降低,这些检测策略是否可以提供更全面的一线检测?这个病例不仅展示了 3 型心面皮肤综合征的特征,还展示了现代遗传技术在诊断全球发育迟缓儿童方面的附加价值。

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