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经典型半乳糖血症中的弗里德赖希共济失调

Friedreich Ataxia in Classical Galactosaemia.

作者信息

Neville Siobhán, O'Sullivan Siobhan, Sweeney Bronagh, Lynch Bryan, Hanrahan Donncha, Knerr Ina, Lynch Sally Ann, Crushell Ellen

机构信息

National Centre for Inherited Metabolic Disorders, Temple Street Children's University Hospital, Dublin, Ireland.

Department of Metabolic Paediatrics, Royal Hospital for Sick Children, Belfast, UK.

出版信息

JIMD Rep. 2016;26:1-5. doi: 10.1007/8904_2015_477. Epub 2015 Jul 29.

Abstract

Movement disorders such as ataxia are a recognized complication of classical galactosaemia, even in diet-compliant patients. Here, we report the coexistence of classical galactosaemia and Friedreich ataxia (FRDA) in nine children from seven Irish Traveller families. These two autosomal recessive disorders, the loci for which are located on either side of the centromere of chromosome 9, appear to be in linkage disequilibrium in this subgroup. Both conditions are known to occur with increased frequency amongst the Irish Traveller population.Each member of our cohort had been diagnosed with galactosaemia in the neonatal period, and all are homozygous for the common Q188R mutation in the GALT gene. Eight of the nine patients later presented with progressive ataxia, between the ages of 5-13 years. Another child presented in cardiac failure secondary to dilated cardiomyopathy at 7 years of age. He was not ataxic at presentation and, one year from diagnosis, his neurological examination remains normal. The diagnosis of FRDA was confirmed by detecting the common pathogenic GAA expansion in both alleles of the frataxin gene (FXN) in each patient.Neurological symptoms are easily attributed to an underlying diagnosis of galactosaemia. It is important to consider a diagnosis of Friedreich ataxia in a child from the Irish Traveller population with galactosaemia who presents with ataxia or cardiomyopathy.

摘要

共济失调等运动障碍是经典型半乳糖血症公认的并发症,即使是严格遵循饮食治疗的患者也会出现。在此,我们报告了来自7个爱尔兰游民家庭的9名儿童同时患有经典型半乳糖血症和弗里德赖希共济失调(FRDA)。这两种常染色体隐性疾病,其基因座位于9号染色体着丝粒的两侧,在这个亚组中似乎处于连锁不平衡状态。众所周知,这两种疾病在爱尔兰游民人群中的发病率都有所增加。我们队列中的每个成员在新生儿期都被诊断为半乳糖血症,并且都是GALT基因常见的Q188R突变的纯合子。9名患者中有8名在5至13岁之间后来出现进行性共济失调。另一名儿童在7岁时因扩张型心肌病出现心力衰竭。他在就诊时没有共济失调症状,自诊断以来一年,其神经学检查仍正常。通过检测每个患者的frataxin基因(FXN)两个等位基因中常见的致病性GAA扩增,证实了FRDA的诊断。神经症状很容易归因于潜在的半乳糖血症诊断。对于患有半乳糖血症且出现共济失调或心肌病的爱尔兰游民儿童,考虑诊断为弗里德赖希共济失调很重要。

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本文引用的文献

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