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一例Vulto-van Silfhout-de Vries综合征患者的独特观察

A Unique Observation of a Patient with Vulto-van Silfhout-de Vries Syndrome.

作者信息

Bodunova Natalia, Vorontsova Maria, Khatkov Igor, Baranova Elena, Bykova Svetlana, Degterev Daniil, Litvinova Maria, Bilyalov Airat, Makarova Maria, Sagaydak Olesya, Danishevich Anastasia

机构信息

The Loginov Moscow Clinical Scientific Center, 111123 Moscow, Russia.

National Medical Research Center for Endocrinology, 117292 Moscow, Russia.

出版信息

Diagnostics (Basel). 2022 Aug 4;12(8):1887. doi: 10.3390/diagnostics12081887.

Abstract

Introduction: Vulto-van Silfhout-de Vries Syndrome (VSVS; OMIM#615828) is a rare hereditary disease associated with impaired intellectual development and speech, delayed psychomotor development, and behavioral anomalies, including autistic behavioral traits and poor eye contact. To date, 27 patients with VSVS have been reported in the literature. Materials and Methods: We describe a 23-year-old male patient with autism spectrum disorder (ASD) who was admitted to the gastroenterological hospital with signs of pseudomembranous colitis. ASD was first noted in the patient at the age of 2.5 years. Later, he developed epileptic seizures and important growth retardation. Prior to the hospitalization, chromosomal aberrations, Fragile X syndrome, and aminoacidopathies/aminoacidurias associated with ASD were excluded. Whole-genome sequencing (WGS) was prescribed to the patient at 23 years old. Results: The patient had a heterozygous carrier of “de novo” variant c.662C > T (p.S221L) in exon 4 of the DEAF1 gene. c.662C > T had not been previously described in genomic databases. According to the ACMG criteria, this missense variant was considered to be pathogenic. VSVS was diagnosed in the patient. Conclusions: The phenotype of the patient is very similar to the data presented in the world literature. However, growth retardation and cachexia, which have not been described previously in the articles, are of interest.

摘要

引言

Vulto-van Silfhout-德弗里斯综合征(VSVS;OMIM#615828)是一种罕见的遗传性疾病,与智力发育和语言受损、精神运动发育迟缓以及行为异常有关,包括自闭症行为特征和眼神交流不佳。迄今为止,文献中已报道了27例VSVS患者。材料与方法:我们描述了一名23岁患有自闭症谱系障碍(ASD)的男性患者,他因伪膜性结肠炎症状入住胃肠病医院。该患者在2.5岁时首次被发现患有ASD。后来,他出现了癫痫发作和严重的生长发育迟缓。在住院前,排除了与ASD相关的染色体畸变、脆性X综合征以及氨基酸病/氨基酸尿症。患者在23岁时接受了全基因组测序(WGS)。结果:患者是DEAF1基因第4外显子“从头”变异c.662C>T(p.S221L)的杂合携带者。c.662C>T此前在基因组数据库中未被描述。根据美国医学遗传学与基因组学学会(ACMG)标准,这种错义变异被认为是致病性的。该患者被诊断为VSVS。结论:该患者的表型与世界文献中报道的数据非常相似。然而,文章中此前未描述的生长发育迟缓和恶病质值得关注。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6f48/9406734/3c3b2b40b1d3/diagnostics-12-01887-g001.jpg

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