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一个患有经典型埃勒斯-当洛综合征(cEDS)、骨骼脆性轻度且无血管并发症的家族,由COL1A1基因中的p.Arg312Cys突变引起。

A family with Classical Ehlers-Danlos Syndrome (cEDS), mild bone fragility and without vascular complications, caused by the p.Arg312Cys mutation in COL1A1.

作者信息

Duong June, Rideout Andrea, MacKay Sara, Beis Jill, Parkash Sandhya, Schwarze Ulrike, Horne S Gabrielle, Vandersteen Anthony

机构信息

Queens University Medical School, Kingston, ON, Canada.

IWK Health Centre, Halifax, NS, Dalhousie University Medicine, Canada.

出版信息

Eur J Med Genet. 2020 Feb;63(2):103730. doi: 10.1016/j.ejmg.2019.103730. Epub 2019 Jul 16.

Abstract

The Ehlers-Danlos syndromes (EDS) are heritable disorders of connective tissue (HDCT) with joint hypermobility, skin hyperextensibility and tissue fragility, which were recently re-classified (2017 International Classification). Most patients (>90%) with Classical Ehlers-Danlos syndrome (cEDS) have a mutation in the COL5A1 or COL5A2 genes encoding type V procollagen. A small number of patients with the p.Arg312Cys mutation in COL1A1 have been reported with overlapping features of both cEDS and vascular EDS (vEDS). In this report, we describe two patients from a large family with this mutation and clinical features consistent with cEDS without vascular complications. The proband presented with congenital hip dislocation (previously reported in one patient), the mother of the proband with multiple fractures in childhood, and dental defects (novel findings). The small number of patients reported with this mutation and proportion with vascular complications suggests that vascular surveillance should still be recommended.

摘要

埃勒斯-当洛综合征(EDS)是一种遗传性结缔组织疾病(HDCT),具有关节活动过度、皮肤过度伸展和组织脆弱的特点,最近(2017年国际分类)进行了重新分类。大多数典型埃勒斯-当洛综合征(cEDS)患者(>90%)在编码V型前胶原的COL5A1或COL5A2基因中存在突变。少数COL1A1基因存在p.Arg312Cys突变的患者被报道同时具有cEDS和血管型EDS(vEDS)的重叠特征。在本报告中,我们描述了来自一个大家庭的两名具有该突变且临床特征与cEDS相符但无血管并发症的患者。先证者表现为先天性髋关节脱位(此前在一名患者中报道过),先证者的母亲在儿童期有多处骨折,以及牙齿缺陷(新发现)。报道的具有该突变的患者数量较少以及出现血管并发症的比例表明,仍应建议进行血管监测。

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