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丁苯那嗪对亨廷顿舞蹈症患者减轻舞蹈样动作及改善运动功能的疗效与安全性:一项系统评价

Efficacy and Safety of Tetrabenazine in Reducing Chorea and Improving Motor Function in Individuals With Huntington's Disease: A Systematic Review.

作者信息

Vadlamani Nandini, Ibrahimli Sabina, Khan Farees Ahmad, Castillo Jason A, Amaravadi Kavya Sai Satya, Nalisetty Poornachandra, Khan Safeera

机构信息

Family Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.

Internal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.

出版信息

Cureus. 2024 Oct 14;16(10):e71476. doi: 10.7759/cureus.71476. eCollection 2024 Oct.

Abstract

Huntington's disease (HD) is a hereditary neurodegenerative disorder that causes chorea and motor dysfunction due to a mutation in the Huntingtin (HTT) gene. Tetrabenazine (TBZ) is used to treat HD-related chorea, but its efficacy and safety require further investigation. This systematic review aims to assess the efficacy and safety of TBZ in reducing chorea and improving motor function in HD patients. A comprehensive search was conducted across multiple sources, including PubMed, PubMed Central, Cochrane Library, Wiley Library, and Google Scholar. Medical subject heading (MeSH) terms were used to enhance search precision. Narrative reviews, clinical practice guidelines, open-label trials, and observational studies were included. Data synthesis followed Cochrane's recommendations for narrative synthesis. Evidence from narrative reviews, clinical guidelines, and trials consistently supports TBZ's efficacy in reducing chorea and improving motor function in HD patients. However, potential side effects like sedation and depression have been noted. This review underscores TBZ's positive impact but emphasizes cautious consideration of associated risks, informing clinical management and further research directions.

摘要

亨廷顿舞蹈症(HD)是一种遗传性神经退行性疾病,由于亨廷顿蛋白(HTT)基因突变导致舞蹈症和运动功能障碍。丁苯那嗪(TBZ)用于治疗与HD相关的舞蹈症,但其疗效和安全性仍需进一步研究。本系统评价旨在评估TBZ在降低HD患者舞蹈症和改善运动功能方面的疗效和安全性。通过多个来源进行了全面检索,包括PubMed、PubMed Central、Cochrane图书馆、Wiley图书馆和谷歌学术。使用医学主题词(MeSH)来提高检索精度。纳入了叙述性综述、临床实践指南、开放标签试验和观察性研究。数据合成遵循Cochrane关于叙述性合成的建议。来自叙述性综述、临床指南和试验的证据一致支持TBZ在降低HD患者舞蹈症和改善运动功能方面的疗效。然而,已注意到潜在的副作用,如镇静和抑郁。本综述强调了TBZ的积极影响,但强调要谨慎考虑相关风险,为临床管理和进一步的研究方向提供依据。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cd84/11560395/08068a5e9147/cureus-0016-00000071476-i01.jpg

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