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四名 Langer-Giedion 综合征患者的长期随访:临床经过和并发症。

Long-term follow-up of four patients with Langer-Giedion syndrome: clinical course and complications.

机构信息

Institute of Medical Genetics, Department of Pediatrics, University of Zürich, Zürich, Switzerland.

出版信息

Am J Med Genet A. 2013 Sep;161A(9):2216-25. doi: 10.1002/ajmg.a.36062. Epub 2013 Aug 2.

Abstract

Long-term observations of individuals with the so-called Langer-Giedion (LGS) or tricho-rhino-phalangeal type II (TRPS2) are scarce. We report here a on follow-up of four LGS individuals, including one first described by Andres Giedion in 1969, and review the sparse publications on adults with this syndrome which comprises ectodermal dysplasia, multiple cone-shaped epiphyses prior to puberty, multiple cartilaginous exostoses, and mostly mild intellectual impairment. LGS is caused by deletion of the chromosomal segment 8q24.11-q24.13 containing among others the genes EXT1 and TRPS1. Most patients with TRPS2 are only borderline or mildly cognitively delayed, and few are of normal intelligence. Their practical skills are better than their intellectual capability, and, for this reason and because of their low self-esteem, they are often underestimated. Some patients develop seizures at variable age. Osteomas on processes of cervical vertebrae may cause pressure on cervical nerves or dissection of cerebral arteries. Joint stiffness is observed during childhood and changes later to joint laxity causing instability and proneness to trauma. Perthes disease is not rare. Almost all males become bald at or soon after puberty, and some develop (pseudo) gynecomastia. Growth hormone deficiency was found in a few patients, TSH deficiency so far only in one. Puberty and fertility are diminished, and no instance of transmission of the deletion from a non-mosaic parent to a child has been observed so far. Several affected females had vaginal atresia with consequent hydrometrocolpos.

摘要

患有所谓的 Langer-Giedion(LGS)或毛发-鼻-指(TRPS2)综合征的个体的长期观察结果较为少见。我们在此报告对 4 例 LGS 个体的随访结果,其中包括 1969 年由 Andres Giedion 首次描述的病例,并回顾了该综合征成人患者的罕见文献,该综合征包括外胚层发育不良、青春期前多发性锥形骨骺、多发性软骨外生骨疣,以及智力障碍大多较轻。LGS 是由 8q24.11-q24.13 染色体片段缺失引起的,该片段包含 EXT1 和 TRPS1 等基因。大多数 TRPS2 患者仅存在轻度至中度认知障碍,少数患者具有正常智力。他们的实际技能优于智力能力,由于这个原因以及由于他们的自尊心较低,他们往往被低估。一些患者在不同年龄段出现癫痫发作。颈椎椎体突起处的骨瘤可能会压迫颈神经或导致大脑动脉夹层。在儿童期观察到关节僵硬,随后会变为关节松弛,导致不稳定和容易受伤。佩吉特病并不罕见。几乎所有男性在青春期或青春期后不久都会脱发,有些会出现(假性)乳腺发育。少数患者存在生长激素缺乏,促甲状腺激素缺乏目前仅见于 1 例。青春期和生育能力受到影响,尚未观察到非镶嵌型父母将缺失传递给子女的情况。一些受影响的女性存在阴道闭锁,继而导致阴道积水和子宫积脓。

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