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斯里兰卡亨廷顿病的临床和遗传特征。

Clinical and genetic features of Huntington disease in Sri Lanka.

机构信息

Human Genetics Unit, Faculty of Medicine, University of Colombo, Sri Lanka.

出版信息

BMC Neurol. 2013 Dec 5;13:191. doi: 10.1186/1471-2377-13-191.

Abstract

BACKGROUND

Huntington disease was one of the first neurological hereditary diseases for which genetic testing was made possible as early as 1993. The study describes the clinical and genetic characteristics of patients with Huntington disease in Sri Lanka.

METHODS

Data of 35 consecutive patients tested from 2007 to 2012 at the Human Genetics Unit, Faculty of Medicine, University of Colombo was analyzed retrospectively. Clinical data and genetic diagnostic results were reviewed. Statistical analysis was performed using descriptive statistics.

RESULTS

Thirty patients had fully penetrant (FP) CAG repeat mutations and 5 had reduced penetrant (RP) CAG repeat mutations. In the FP group mean ages of onset and diagnosis were 37.5 and 40.4 years, while in the RP group it was 63.0 and 64.8 years respectively. The age of diagnosis ranged from 15 to 72 years, with 2 patients with Juvenile onset (<20 years) and 3 with late onset (>60 years) Huntington disease. The symptoms at diagnosis were predominantly motor (32/35 -91%). Three patients had psychiatric and behavioral disorders. The age difference between onset and genetic diagnosis showed significant delay in females compared to males (p < 0.05). Twenty two (62.8%) had a positive family history, with 13/22 (59.1%) showing a paternal inheritance of the disease. In both groups, those with a family history had a significantly lower age of presentation (p < 0.05). The mean CAG repeat length in patients with FP alleles was 44.6 ± 5 and RP alleles was 37.2 ± 1.1. Age of onset and CAG repeat length of the HTT gene showed significant inverse correlation (p < 0.0005, R2 = 0.727).

CONCLUSIONS

The clinical and genetic features seen in patients with Huntington disease in the Sri Lankan study population were similar to that previously reported in literature.

摘要

背景

亨廷顿病是最早可以进行基因检测的神经遗传性疾病之一,早在 1993 年就已经实现了这一检测。本研究描述了在斯里兰卡的亨廷顿病患者的临床和遗传特征。

方法

回顾性分析了 2007 年至 2012 年在科伦坡大学医学院人类遗传学系接受检测的 35 例连续患者的数据。对临床数据和基因诊断结果进行了回顾。使用描述性统计进行了统计分析。

结果

30 例患者存在完全外显(FP)的 CAG 重复突变,5 例患者存在部分外显(RP)的 CAG 重复突变。在 FP 组中,发病和诊断的平均年龄分别为 37.5 岁和 40.4 岁,而在 RP 组中则分别为 63.0 岁和 64.8 岁。诊断年龄范围为 15 至 72 岁,有 2 例患者为青少年发病(<20 岁),3 例患者为晚发性(>60 岁)亨廷顿病。诊断时的症状主要为运动障碍(35 例中有 32 例,占 91%)。3 例患者存在精神和行为障碍。与男性相比,女性的发病和基因诊断之间的年龄差异具有显著的延迟(p<0.05)。22 例(62.8%)有阳性家族史,其中 13 例(59.1%)为疾病的父系遗传。在两组中,有家族史的患者的发病年龄显著更低(p<0.05)。FP 等位基因的患者的 CAG 重复长度平均为 44.6±5,RP 等位基因的患者为 37.2±1.1。发病年龄和 HTT 基因的 CAG 重复长度呈显著负相关(p<0.0005,R2=0.727)。

结论

在斯里兰卡研究人群中,亨廷顿病患者的临床和遗传特征与文献中先前报道的相似。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7447/4029274/bd23d90f1794/1471-2377-13-191-1.jpg

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