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努辛森和Risdiplam对脊柱肌肉缩的安全性和有效性:随机对照试验的系统审查和元分析

Yue Qiao¹, Yuewei Chi¹, Jian Gu¹

¹Department of Neurology, Shengjing Hospital of China Medical University, Shenyang 110055, China.

|October 28, 2023

概括

努辛森和瑞斯迪普拉姆在脊髓肌缩 (SMA) 患者中提供了显著的运动功能的改善,其安全性与安慰剂相似. 需要进一步进行直接比较.

科学领域:

神经学神经学
药理学药理学是指药理学的学科.
临床试验临床试验

背景情况:

脊椎肌肉缩 (SMA) 是一种严重的遗传疾病,影响运动神经元.
努辛森和Risdiplam是SMA的新兴治疗方法,但直接比较的疗效和安全性数据有限.

研究的目的:

系统地审查和元分析努西纳森和RISDIPLAM在治疗SMA患者中的疗效和安全性.
综合随机对照试验 (RCT) 的证据,以告知临床实践.

主要方法:

在Pubmed,Web of Science,Embase和Cochrane的系统文献搜索到2023年7月.
包括6个RCT,涉及728名SMA患者.
使用Review Manager 5.4和Stata 15.0软件进行数据分析.

主要成果:

努辛森显著改善了HFMSE,RULM和HINE-2分数.
里斯迪普拉姆显著改善了HFMSE和MFM32的得分.
与安慰剂相比,这两种药物都没有在统计学上显著地增加不良事件或严重不良事件.

结论:

努西森森和瑞斯迪普拉姆在SMA患者的运动功能的临床上有意义的改善.
副作用的发生率在nusinersen,risdiplam和安慰剂之间是相似的.
需要进一步的研究来直接比较nusinersen和risdiplam的疗效和安全性.

关键词:

有效性有效性有效性.这是一个元分析.没有任何的 nusinersenersen.里斯迪普拉姆的风险在安全方面,安全是安全的.脊柱肌肉缩脊柱肌肉缩脊柱肌肉缩

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