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一例女性 Fabry 病杂合子患者,伴多发血管瘤而无血管角质瘤。

A case of multiple angiomas without any angiokeratomas in a female heterozygote with Fabry disease.

机构信息

Department of Dermatology and Allergy Biederstein, Technical University of Munich, Munich, Germany.

出版信息

Australas J Dermatol. 2010 Feb;51(1):36-8. doi: 10.1111/j.1440-0960.2009.00590.x.

Abstract

Fabry disease is a rare, X-chromosome-linked lysosomal storage disease caused by a deficient alpha-galactosidase A enzyme. The disease manifests primarily in affected hemizygous males and to some extent in heterozygous females ('carrier'). A 45-year-old female Fabry disease patient without angiokeratomas but with numerous angiomas is presented. Her leukocyte alpha-galactosidase A activity was reduced (0.35 nmol/min/mg protein; normal range: 0.4-1). The analysis of her alpha-galactosidase A gene (exon 1-7) showed the transition c.427 G>A. An intrafamilial follow-up search detected a reduced leukocyte alpha-galactosidase A activity in her father, who suffered exclusively from coronary heart disease. Our case report underlines the possible wide range of clinical signs in Fabry disease patients, sometimes complicated by missing typical lesions (e.g. angiokeratomas). In oligosymptomatic Fabry disease cases, genetic analysis is recommended.

摘要

法布里病是一种罕见的 X 连锁溶酶体贮积病,由缺乏α-半乳糖苷酶 A 酶引起。该病主要表现为受累的半合子男性,在一定程度上也表现为杂合子女性(“携带者”)。本文报道了一位 45 岁的女性法布里病患者,她没有血管角质瘤,但有许多血管瘤。她的白细胞α-半乳糖苷酶 A 活性降低(0.35 nmol/min/mg 蛋白;正常范围:0.4-1)。对其α-半乳糖苷酶 A 基因(外显子 1-7)的分析显示 c.427 G>A 转换。家族内随访发现其父亲白细胞α-半乳糖苷酶 A 活性降低,仅患有冠心病。本病例报告强调了法布里病患者临床表现的广泛范围,有时伴有典型病变缺失(如血管角质瘤)。在寡症状性法布里病病例中,建议进行基因分析。

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