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初级进展性多发性硬化症患者子类中的疾病修饰性治疗和残疾进展:来自MS大数据网络的结果

Johannes Lorscheider^1,2, Alessio Signori³, Suvitha Subramaniam⁴

¹Department of Neurology, University Hospital Basel, Basel, Switzerland johannes.lorscheider@usb.ch.

Journal of neurology, neurosurgery, and psychiatry

|December 6, 2024

概括

改变疾病的治疗方法 (DMT) 对初级渐进性多发性硬化症 (PPMS) 的有效性有限. 然而,DMT可能会减少具有侵略性疾病轨迹的PPMS患者的残疾恶化.

科学领域:

神经学神经学
临床研究临床研究
流行病学流行病学

背景情况:

对于初级渐进性多发性硬化症 (PPMS) 的疾病修饰治疗 (DMT) 的有效性通常是有限的.
在现实环境中确定可能受益于DMT的PPMS患者的子组至关重要.

研究的目的:

调查DMT对PPMS患者残疾恶化的影响.
根据残疾轨迹对PPMS患者进行分层,以确定潜在的治疗益处.

主要方法:

利用大MS数据网络,合并来自多个国际MS注册表的数据.
应用倾向性比分匹配,以创建可比的治疗和未治疗PPMS患者组.
通过使用隐性类混合模型定义的子组内的时间到反复事件分析分析了残疾结果.

主要成果:

在接受治疗和未接受治疗的PPMS患者之间,确认残疾恶化 (CDW) 没有显著差异.
在接受治疗的PPMS患者中发现CDW的风险降低,这些患者的残疾轨迹较为激烈 (HR 0.68,p=0.014).

结论:

根据先前的研究,DMT通常不会改善PPMS中残疾恶化的情况.
一部分PPMS患者,特别是那些具有积极预测残疾轨迹的患者,可能会从DMT中受益于残疾恶化.

关键词:

多发性硬化多发性硬化

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