在复发性复发性多发性硬化症中,进展独立于复发活动 (PIRA) 的标准化定义
Jannis Müller1,2,3, Sifat Sharmin1,2, Johannes Lorscheider3
1CORe, Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.
JAMA neurology
|April 14, 2025
概括
进展独立于复发活动 (PIRA) 的定义有所不同,影响了多发性硬化症 (MS) 研究. 建议制定一个标准化的PIRA定义,以提高研究的可比性和对MS患者残疾累积的理解.
科学领域:
- 神经学 神经学
- 临床研究 临床研究
- 多发性硬化症 (MS) 是一种疾病.
背景情况:
- 独立于复发活动的进展 (PIRA) 显著导致复发缓解多发性硬化症 (MS) 的长期残疾.
- 在研究中不一致的PIRA定义阻碍了结果的可比性和对疾病进展的理解.
研究的目的:
- 为了比较360个不同的PIRA定义对发病率和持续性的影响.
- 提出一个标准化的PIRA定义,以提高成员国的研究可比性.
主要方法:
- 从MSBase注册表 (2004-2023) 收集的前性数据的回顾性分析.
- 包括33,203名临床确定的复发性缓解性多发性硬化症患者和至少三个扩展残疾状况量表 (EDSS) 评估.
- 基于基线残疾,确认期,大小和复发自由标准的360个PIRA定义的评估.
主要成果:
- 每十年PIRA发病率从0.141到0.658事件不等,持久性从0.753到0.919不等,高度依赖于使用的定义.
- 基线和确认期的标准显著影响了PIRA检测.
- 提出的定义 (残疾恶化与重置基线,自上次EDSS以来没有复发,在12个月内得到确认) 显示出平衡的发病率和持续性.
结论:
- 选择PIRA定义对MS中报告的发病率和持续率产生重大影响.
- 标准化定义对于提高多发性硬化症研究结果的一致性和可比性至关重要.
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