治疗滞后:在渐进的多发性硬化症中,治疗效果是否延迟?
Noemi Montobbio1, Francesca Bovis1, Alessio Signori1
1Department of Health Sciences (DISSAL), University of Genova, Genova, Italy.
Multiple sclerosis (Houndmills, Basingstoke, England)
|April 15, 2024
概括
在渐进性多发性硬化症 (MS) 试验中,通过重新分析数据,发现了延迟治疗效应. 这一发现表明,多发性硬化症的治疗可能比通常检测到的更晚地显示好处.
科学领域:
- 神经学 神经学
- 临床试验 临床试验
- 生物统计学 生物统计学
背景情况:
- 对渐进性多发性硬化症 (MS) 的随机临床试验 (RCT) 通常无法显示治疗对残疾进展的显著影响.
- 这种缺乏意义可能源于治疗疗效的延迟开始.
- 基线特征可能会影响这种延迟.
研究的目的:
- 调查延迟治疗效果是否解释了渐进性多发性硬化症再试验试验中的非显著发现.
- 分析基线特征对治疗效益时间的影响.
主要方法:
- 重新分析了两项试验 (SPECTRIMS和PROMISE) 的数据,这些试验涉及进步性多发性硬化症中的干扰素-β和格拉提拉-乙酸盐.
- 应用一个依赖时间的考克斯模型,允许延迟治疗效果 (t0).
- 模拟治疗延迟作为基线扩展残疾状态量表 (EDSS) 评分的函数.
主要成果:
- 一个依赖时间的考克斯模型在两个研究中都显示出显著的治疗益处,当延迟被纳入时 (SPECTRIMS:t0=2.5年,HR=0.65;PROMISE:t0=2年,HR=0.65).
- 在合并的数据集中,依赖EDSS的延迟效应显示出显著的好处 (HR=0.68,p<0.001).
- 假设延迟治疗效果改善了模型的适应性,这表明治疗效益显著,尽管转移了.
结论:
- 纳入延迟治疗效果假设显著改善了对渐进性MS RCT数据的分析.
- 这种方法发现了标准分析中没有明显的显著治疗益处,这表明需要在试验设计和解释中考虑延迟疗效.
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