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利鲁唑用于治疗遗传性共济失调:一项系统评价。

Use of Riluzole for the Treatment of Hereditary Ataxias: A Systematic Review.

作者信息

Ayala Iván Nicolas, Aziz Syed, Argudo Jennifer M, Yepez Mario, Camacho Mikaela, Ojeda Diego, Aguirre Alex S, Oña Sebastian, Andrade Andres F, Vasudhar Ananya, Moncayo Juan A, Hassen Gashaw, Ortiz Juan Fernando, Tambo Willian

机构信息

KER Unit, Mayo Clinic, Rochester, MN 55902, USA.

Shaheed Suhrawardy Medical College, Dhaka 1207, Bangladesh.

出版信息

Brain Sci. 2022 Aug 5;12(8):1040. doi: 10.3390/brainsci12081040.

DOI:10.3390/brainsci12081040
PMID:36009103
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9405857/
Abstract

Ataxia is a constellation of symptoms that involves a lack of coordination, imbalance, and difficulty walking. Hereditary ataxia occurs when a person is born with defective genes, and this degenerative disorder may progress for several years. There is no effective cure for ataxia, so we need to search for new treatments. Recently, interest in riluzole in the treatment of ataxia has emerged. We conducted this systematic review to analyze the safety and efficacy of riluzole for treating hereditary ataxia in recent clinical trials. We conducted a systematic review using PubMed and Google Scholar as databases in search of this relationship. We used the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) and Meta-analysis of Observational Studies in Epidemiology (MOOSE) protocols to conduct this study. For inclusion criteria, we included full-text clinical trials on humans written in English and found three clinical trials. We excluded case reports, literature reviews, systematic reviews, and meta-analyses for this analysis. We aimed to evaluate the Scale for the Assessment and Rating of Ataxia (SARA) score, the International Cooperative Ataxia Rating Scale (ICARS) score, and the safety of the medication. Two out of the three clinical trials showed statistically significant clinical improvement in the ICARS and SARA scores, while the other trial did not show improvement in the clinical or radiological outcomes. The drug was safe in all clinical trials. Overall, the results of this analysis of riluzole for the treatment of hereditary ataxia are encouraging. Further clinical trials are needed to investigate the efficacy of riluzole on hereditary ataxia.

摘要

共济失调是一组症状,包括缺乏协调性、平衡感以及行走困难。遗传性共济失调是指一个人出生时带有缺陷基因,这种退行性疾病可能会持续发展数年。目前尚无治疗共济失调的有效方法,因此我们需要寻找新的治疗手段。最近,人们对利鲁唑治疗共济失调产生了兴趣。我们进行了这项系统评价,以分析利鲁唑在近期临床试验中治疗遗传性共济失调的安全性和有效性。我们以PubMed和谷歌学术为数据库进行系统评价,以探寻这种关系。我们使用系统评价和Meta分析的首选报告项目(PRISMA)以及流行病学观察性研究的Meta分析(MOOSE)方案来开展本研究。纳入标准方面,我们纳入了以英文撰写的关于人类的全文临床试验,共找到三项临床试验。本次分析排除了病例报告、文献综述、系统评价和Meta分析。我们旨在评估共济失调评估与评分量表(SARA)评分、国际协作共济失调评定量表(ICARS)评分以及药物的安全性。三项临床试验中有两项显示ICARS和SARA评分在统计学上有显著的临床改善,而另一项试验在临床或影像学结果上未显示出改善。该药物在所有临床试验中均安全。总体而言,本次利鲁唑治疗遗传性共济失调的分析结果令人鼓舞。需要进一步的临床试验来研究利鲁唑对遗传性共济失调的疗效。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/233559319902/brainsci-12-01040-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/e60ae5377f1e/brainsci-12-01040-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/8bef89acfa7d/brainsci-12-01040-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/233559319902/brainsci-12-01040-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/e60ae5377f1e/brainsci-12-01040-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/8bef89acfa7d/brainsci-12-01040-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ea9a/9405857/233559319902/brainsci-12-01040-g003.jpg

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