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间质干细胞治疗神经退行性小脑共济失调:系统评价和荟萃分析。

Mesenchymal Stem Cell Therapy in the Treatment of Neurodegenerative Cerebellar Ataxias: a Systematic Review and Meta-analysis.

机构信息

Department of Neurology, CN Centre, All India Institute of Medical Sciences, Room No. 60 GF, New Delhi, 110029, India.

出版信息

Cerebellum. 2023 Jun;22(3):363-369. doi: 10.1007/s12311-022-01403-6. Epub 2022 Apr 22.

Abstract

The objective of this study is to synthesise the findings of clinical studies in order to derive evidence for use of the mesenchymal stem cell (MSC) therapy in the treatment of neurodegenerative cerebellar ataxias. In order to find relevant studies for the systematic review, we searched through Medline (1985 to July 2020), PubMed and Clinical trial register. We included both single-arm and comparative studies in which MSCs were given as intervention in neurodegenerative ataxia patients at any time after the diagnosis. We used Joanna Briggs Institute (JBI) quality scale to evaluate the methodological qualities of the included studies. Our literature search obtained 81 publications. Three articles comprising a total of 47 patients were included in the meta-analysis. None of them were randomised controlled trials (RCTs). Pooled analysis noted that there was a decrease in the Berg Balance Scale (BBS)/Scale for the Assessment and Rating of Ataxia (SARA) score from pre to post assessment; however, the difference was statistically not significant (standardised mean difference (SMD) - 0.20; 95% CI - 0.78 to 0.38). No significant side effects were reported in any of the studies. We did not observe any statistically significant difference in the pooled mean difference in the International Cooperative Ataxia Rating Scale (ICARS) score between pre and post assessment in patients with ataxia after receiving the stem cells (SMD 0.36, 95% CI - 0.08 to 0.81). Our systematic review and meta-analysis concluded that MSC cell therapy appeared safe but provided insufficient evidence to support the use of MSCs to treat patients with neurodegenerative cerebellar ataxia at present. No l RCTs was available in the literature to test efficacy; therefore, well-designed RCTs are needed to ascertain the effectiveness of MSCs in patients with neurodegenerative cerebellar ataxias.

摘要

本研究旨在综合临床研究结果,为间充质干细胞(MSC)治疗神经退行性小脑共济失调提供循证医学证据。为了进行系统评价找到相关研究,我们检索了 Medline(1985 年至 2020 年 7 月)、PubMed 和临床试验注册中心。我们纳入了单臂和对照研究,其中在神经退行性共济失调患者诊断后任何时间给予 MSC 作为干预措施。我们使用 Joanna Briggs 研究所(JBI)质量量表评估纳入研究的方法学质量。我们的文献检索获得了 81 篇文献。三项研究共纳入 47 例患者进行荟萃分析。这些研究均非随机对照试验(RCT)。汇总分析表明,Berg 平衡量表(BBS)/共济失调评估和评分量表(SARA)评分从评估前到评估后有下降,但差异无统计学意义(标准化均数差(SMD)-0.20;95%CI-0.78 至 0.38)。在任何研究中均未报告任何明显的不良反应。我们未观察到接受干细胞治疗后的共济失调患者在国际合作共济失调评分量表(ICARS)评分的汇总平均差值在评估前和评估后之间有统计学上的显著差异(SMD 0.36,95%CI-0.08 至 0.81)。我们的系统评价和荟萃分析得出结论,MSC 细胞治疗似乎安全,但目前提供的证据不足以支持使用 MSC 治疗神经退行性小脑共济失调患者。文献中没有 RCT 来测试疗效;因此,需要精心设计的 RCT 来确定 MSC 对神经退行性小脑共济失调患者的有效性。

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