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戈谢病的围产期致死型是否比经典2型戈谢病更常见?

Is the perinatal lethal form of Gaucher disease more common than classic type 2 Gaucher disease?

作者信息

Stone D L, van Diggelen O P, de Klerk J B, Gaillard J L, Niermeijer M F, Willemsen R, Tayebi N, Sidransky E

机构信息

Clinical Neuroscience Branch, National Institute of Mental Health, National Institutes of Health, Bethesda, MD 20892-4405, USA.

出版信息

Eur J Hum Genet. 1999 May-Jun;7(4):505-9. doi: 10.1038/sj.ejhg.5200315.

Abstract

In recent years there has been increased recognition of a severe perinatal lethal form of Gaucher disease, the inherited deficiency of lysosomal glucocerebrosidase. We previously reported a case of severe type 2 Gaucher disease which was seen in a medical center in Rotterdam and now present three new cases from two other families seen at the same center. Mutational analyses of these cases revealed two novel mutations, H311R and V398F, located in exons 8 and 9, respectively. The identification of four cases of lethal type 2 Gaucher disease in a single center seems to be a function of increased awareness of this phenotype, rather than of geographic clustering. The actual incidence of lethal type 2 Gaucher disease may be underestimated, as many cases may have been misclassified as collodion babies or hydrops of unknown cause.

摘要

近年来,人们越来越认识到戈谢病存在一种严重的围产期致死型,即溶酶体葡萄糖脑苷脂酶的遗传性缺乏。我们之前报道过一例严重的2型戈谢病病例,该病例出现在鹿特丹的一家医疗中心,现在我们呈现另外两个家族的三例新病例,这些病例也来自同一中心。对这些病例的突变分析揭示了两个新的突变,分别位于第8和第9外显子的H311R和V398F。在单一中心识别出四例致死型2型戈谢病病例,这似乎是对该表型认识提高的结果,而非地理聚集所致。致死型2型戈谢病的实际发病率可能被低估了,因为许多病例可能被误诊为先天性鱼鳞病样红皮病婴儿或不明原因的水肿。

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