Department of Clinical Neurosciences, University of Calgary, Calgary, AB, Canada/Department of Community Health Sciences, University of Calgary, Calgary, AB, Canada.
Department of Neurology, Rijnstate Hospital, Arnhem, The Netherlands.
Mult Scler. 2021 Oct;27(12):1864-1874. doi: 10.1177/1352458520987539. Epub 2021 Jan 19.
The expanded disability status scale (EDSS) is the standard clinical outcome measure in primary progressive multiple sclerosis (PPMS), even though the timed 25-foot walk (T25FW), nine-hole peg test (NHPT) or combinations of these measures may be more useful. The paced auditory serial addition test (PASAT) is a widely used cognitive measure in MS, but little is known about change in PASAT scores over time in PPMS.
The objective of this study is to compare clinical outcome measures in a large PPMS trial data set.
We determined significant worsening events on the EDSS, T25FW and NHPT, and PASAT scores over the course of this 3-year trial. We compared unconfirmed, confirmed and sustained disability worsening and contrasted disability worsening with similarly defined improvement. We examined the association of baseline characteristics with the risk of disability worsening at 12, 24 and 36 months with logistic regression models.
The EDSS and T25FW showed most worsening events, while only few patients worsened on the NHPT. Adding the NHPT to a combined outcome added only few further worsening events. PASAT scores slightly increased over time, possibly due to a practice effect.
Both the EDSS and T25FW, but not NHPT or PASAT, appear to be useful outcome measures in PPMS.
扩展残疾状况量表（EDSS）是原发性进展型多发性硬化症（PPMS）的标准临床疗效指标，尽管定时 25 英尺步行（T25FW）、九孔钉测试（NHPT）或这些措施的组合可能更有用。连续听觉加法测试（PASAT）是 MS 中广泛使用的认知测量方法，但关于 PASAT 分数随时间在 PPMS 中的变化知之甚少。
本研究的目的是比较大型 PPMS 试验数据集的临床疗效测量方法。
我们确定了 EDSS、T25FW 和 NHPT 以及 PASAT 评分在这 3 年试验过程中的显著恶化事件。我们比较了未确认、确认和持续的残疾恶化，并将残疾恶化与同样定义的改善进行了对比。我们使用逻辑回归模型检查了基线特征与 12、24 和 36 个月时残疾恶化风险的相关性。
EDSS 和 T25FW 显示出大多数恶化事件，而只有少数患者在 NHPT 上恶化。将 NHPT 添加到综合结果中仅增加了少数进一步的恶化事件。PASAT 分数随着时间的推移略有增加，可能是由于练习效应。
EDSS 和 T25FW 似乎都是 PPMS 的有用疗效指标，但 NHPT 或 PASAT 则不然。
