Effect of stem cell treatment on functional recovery of spinocerebellar ataxia: systematic review and meta-analysis.

Q3 Medicine Cerebellum and Ataxias Pub Date : 2021-02-25 DOI:10.1186/s40673-021-00130-8
Pablo Andrei Appelt, Kristin Comella, Luciane Aparecida Pascucci Sande de Souza, Gustavo José Luvizutto
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引用次数: 7

Abstract

Background: Spinocerebellar ataxia is a hereditary neurodegenerative disease characterized by changes in balance, locomotion and motor coordination. Stem cell therapies are currently being investigated as an alternative to delay the evolution of the disease, and some experimental studies have investigated the effect of stem cell treatment on spinocerebellar ataxia.

Objectives: The aim of this review was to investigate whether the application of stem cells produced an effect on functional recovery in individuals with spinocerebellar ataxia.

Methods: The studies included in this review investigated the efficacy and safety of a protocol for the application of mesenchymal stem cells extracted from umbilical cord and adipose tissue. Two studies used intrathecal route for application and one study used intravenous route.

Results: Studies have shown clinical improvement in the scores of the ICARS (International Cooperative Ataxia Rating Scale), ADL (Activities of Daily Living Scale), BBS (Berg Balance Scale) and SARA (Scale for the Assessment and Rating of Ataxia), but lacked statistical significance.

Conclusions: There was low evidence for recommending stem cell therapy in individuals with spinocerebellar ataxia, and no statistical difference was observed for improving functional recovery of patients. Further studies are needed with different designs, largest sample sizes and placebo control, to fully understand anticipated outcomes of cellular therapy for spinocerebellar ataxia.

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干细胞治疗对脊髓小脑性共济失调功能恢复的影响:系统回顾和荟萃分析。
背景:脊髓小脑性共济失调是一种遗传性神经退行性疾病,其特征是平衡、运动和运动协调性的改变。目前正在研究干细胞治疗作为延缓疾病发展的替代方法,一些实验研究已经研究了干细胞治疗对脊髓小脑性共济失调的影响。目的:本综述的目的是研究干细胞的应用是否对脊髓小脑性共济失调患者的功能恢复产生影响。方法:本综述中包括的研究调查了从脐带和脂肪组织中提取的间充质干细胞应用方案的有效性和安全性。两项研究使用鞘内途径,一项研究使用静脉途径。结果:研究显示,ICARS(国际合作共济失调评定量表)、ADL(日常生活活动量表)、BBS(伯格平衡量表)和SARA(共济失调评定量表)得分均有临床改善,但缺乏统计学意义。结论:脊髓小脑共济失调患者推荐干细胞治疗的证据较少,在改善患者功能恢复方面无统计学差异。进一步的研究需要不同的设计,最大的样本量和安慰剂对照,以充分了解细胞治疗脊髓小脑性共济失调的预期结果。
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Cerebellum and Ataxias
Cerebellum and Ataxias Medicine-Neurology (clinical)
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