来自印度的肯尼迪氏病:一个多系统表现的印度队列。

IF 3.4 4区 医学 Q2 CLINICAL NEUROLOGY
Journal of neuromuscular diseases Pub Date : 2025-07-01 Epub Date: 2025-05-05 DOI:10.1177/22143602251325795
Saranya B Gomathy, William L Macken, Nimita Rani, Ayush Agarwal, Rakesh Singh, Megha Dhamne, Sruthi S Nair, Alisha Reyaz, Tanveer Ahmed, Ashwin Dalal, Mayandi Muthulakshmi, Lindsay Wilson, Asish Vijayaraghavan, Rohit Bhatia, Robert D S Pitceathly, Kumarasamy Thangaraj, Mary M Reilly, Padma Mv Srivastava, Michael G Hanna, Venugopalan Y Vishnu
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引用次数: 0

摘要

背景:肯尼迪氏病(KD)是一种罕见的隐性进行性下运动神经元综合征,以肌萎缩为特征,累及40至50岁的成年男性的阑尾或球部肌肉组织。没有来自印度次大陆的描述KD临床遗传和实验室谱的大序列。目的了解KD患者的临床、电生理、代谢和遗传特征。方法对10例遗传确诊的KD患者进行回顾性分析。结果本组患者平均年龄47岁,平均发病年龄41.3±9.9岁。症状出现前的中位持续时间为5(3-12)年。最常见的转诊诊断是ALS。大多数患者表现为对称的肢体近端无力并伴有球症状,并发现有男性乳房发育,下运动神经元(LMN)面部无力,面部和舌部束动。电生理学显示5例患者感觉神经病变,所有患者的慢性神经源性改变与前角细胞病一致。代谢谱显示血糖受损,高脂血症和非酒精性脂肪性肝病的证据。所有患者血清肌酸激酶均升高。基因检测显示中位数为46个CAG重复序列。我们患者的表型与全球数据一致,主要来自欧洲血统的参与者。结论:我们描述了一系列来自印度的多系统累及的KD患者。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Kennedy's disease from India: An Indian Cohort with multisystemic manifestations.

BackgroundKennedy's disease (KD) is a rare, insidiously progressive lower motor neuron syndrome characterised by amyotrophy involving the appendicular or bulbar musculature of adult males in their fourth to fifth decade. There are no large series from the Indian subcontinent describing the clinical-genetic and laboratory spectrum of KD.AimTo describe the clinical, electrophysiologic, metabolic and genetic profile of patients with KD.MethodsWe conducted a retrospective review of ten genetically confirmed KD patients.ResultsThe mean age of the cohort was 47 years, with a mean age of onset of illness at 41.3 ± 9.9 years. The median duration of symptoms before presentation was 5 (3-12) years. The most common referral diagnosis was ALS. The majority presented with symmetric proximal limb weakness with bulbar symptoms and were found to have gynecomastia, lower motor neuron (LMN) facial weakness, and facial and lingual fasciculations. Electrophysiology revealed sensory neuropathy in five patients and chronic neurogenic changes consistent with anterior horn cell disease in all. Metabolic profile showed impaired glycemia, hyperlipidemia and evidence of non-alcoholic fatty liver disease in the majority. All had elevated serum creatine kinase. Genetic testing revealed a median of 46 CAG repeats. The phenotypes of our patients aligned with global data that is predominantly derived from participants of European ancestry.ConclusionWe describe a series of patients with KD from India with significant multisystemic involvement.

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来源期刊
Journal of neuromuscular diseases
Journal of neuromuscular diseases Medicine-Neurology (clinical)
CiteScore
5.10
自引率
6.10%
发文量
102
期刊介绍: The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.
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