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经生化检测确诊的13例新的科凯恩综合征病例存在广泛的临床变异性。

Wide clinical variability among 13 new Cockayne syndrome cases confirmed by biochemical assays.

作者信息

Pasquier L, Laugel V, Lazaro L, Dollfus H, Journel H, Edery P, Goldenberg A, Martin D, Heron D, Le Merrer M, Rustin P, Odent S, Munnich A, Sarasin A, Cormier-Daire V

机构信息

Unité de Génétique Clinique, Hôpital Sud, 35203 Rennes, France.

出版信息

Arch Dis Child. 2006 Feb;91(2):178-82. doi: 10.1136/adc.2005.080473.

Abstract

Cockayne syndrome is a multi-systemic, autosomal recessive disease characterised by postnatal growth failure and progressive multi-organ dysfunction. The main clinical features are severe dwarfism (<-2 SD), microcephaly (<-3 SD), psychomotor delay, sensorial loss (cataracts, pigmentary retinopathy, and deafness), and cutaneous photosensitivity. Here, 13 new cases of Cockayne syndrome are reported, which have been clinically diagnosed and confirmed using a biochemical transcription assay. The wide clinical variability, ranging from prenatal features to normal psychomotor development, is emphasised. When cardinal features are lacking, the diagnosis of Cockayne syndrome should be considered when presented with growth retardation, microcephaly, and one of the suggesting features such as enophthalmia, limb ataxia, abnormal auditory evoked responses, or increased ventricular size on cerebral imaging.

摘要

科凯恩综合征是一种多系统常染色体隐性疾病,其特征为出生后生长发育迟缓及进行性多器官功能障碍。主要临床特征包括严重侏儒症(<-2标准差)、小头畸形(<-3标准差)、精神运动发育迟缓、感觉丧失(白内障、色素性视网膜病变和耳聋)以及皮肤光敏性。本文报告了13例新的科凯恩综合征病例,这些病例已通过生化转录分析进行临床诊断和确诊。文中强调了其广泛的临床变异性,范围从产前特征到正常精神运动发育。当缺乏主要特征时,若出现生长发育迟缓、小头畸形以及诸如眼球内陷、肢体共济失调、听觉诱发电位异常或脑成像显示脑室增大等提示性特征之一时,应考虑科凯恩综合征的诊断。

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