保加利亚瓦尔纳的亨廷顿舞蹈病二十年:临床趋势和挑战的回顾性单中心研究。

IF 3.2 Q2 CLINICAL NEUROLOGY
Mariya Levkova, Mihael Tsalta-Mladenov, Milena Stoyanova, Mari Hachmeriyan, Lyudmila Angelova, Ara Kaprelyan
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引用次数: 0

摘要

背景:亨廷顿氏病(HD)是一种进行性常染色体显性神经退行性疾病,由HTT基因CAG重复扩增引起。尽管在了解其分子基础方面取得了进展,但包括保加利亚在内的许多国家的流行病学数据仍然有限。本研究旨在介绍20年单中心队列的临床和遗传结果。方法:回顾性分析2004年至2024年在瓦尔纳“St. Marina”大学医院接受HD评估的患者。数据包括人口统计学、CAG重复长度、临床特征、影像学和精神病学评估。统计分析侧重于变量之间的相关性,显著性设置为p < 0.05。结果:79例转诊患者中,43例分子确诊。平均发病年龄为43岁,诊断延迟4年。平均CAG重复长度为44.6,尽管两名有症状的患者的外显率等位基因减少(38和39重复)。72%的病例分别出现认知和精神症状。抑郁症在女性中更为普遍(p = 0.011)。大多数患者有阳性家族史,主要是母亲。结论:我们的研究结果强调了诊断延迟、特定性别的精神脆弱性以及个性化护理的重要性。改善获得遗传咨询和早期诊断的机会对于优化结果至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Two Decades of Huntington's Disease in Varna, Bulgaria: A Retrospective Single-Centre Study of Clinical Trends and Challenges.

Background: Huntington's disease (HD) is a progressive, autosomal dominant neurodegenerative disorder caused by an expanded CAG repeat in the HTT gene. Despite advances in understanding its molecular basis, epidemiological data in many countries, including Bulgaria, remain limited. This study aims to present clinical and genetic findings from a 20-year single-centre cohort. Methods: A retrospective review was conducted of patients evaluated for HD at the University Hospital "St. Marina" in Varna between 2004 and 2024. Data included demographics, CAG repeat length, clinical features, imaging, and psychiatric assessments. Statistical analysis focused on correlations between variables, with significance set at p < 0.05. Results: Out of 79 referred individuals, 43 were molecularly confirmed. The mean age of onset was 43 years, with a four-year diagnostic delay. The average CAG repeat length was 44.6, though two symptomatic patients had reduced penetrance alleles (38 and 39 repeats). Cognitive and psychiatric symptoms were each present in 72% of cases. Depression was significantly more prevalent in women (p = 0.011). Most patients had a positive family history, predominantly maternal. Conclusions: Our findings highlight diagnostic delays, gender-specific psychiatric vulnerabilities, and the importance of personalized care. Improved access to genetic counselling and early diagnosis are essential for optimizing outcomes.

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来源期刊
Neurology International
Neurology International CLINICAL NEUROLOGY-
CiteScore
3.70
自引率
3.30%
发文量
69
审稿时长
11 weeks
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