Bsteh Gabriel, Marti Stefanie, Krajnc Nik, Traxler Gerhard, Salmen Anke, Hammer Helly, Leutmezer Fritz, Rommer Paulus, Di Pauli Franziska, Chan Andrew, Berger Thomas, Hegen Harald, Hoepner Robert
Department of Neurology, Medical University of Vienna, Vienna, Austria; Comprehensive Center for Clinical Neurosciences and Mental Health, Medical University of Vienna, Vienna, Austria.
Department of Neurology, Inselspital, Bern University Hospital and University of Bern, Switzerland.
Mult Scler Relat Disord. 2025 Jan;93:106215. doi: 10.1016/j.msard.2024.106215. Epub 2024 Dec 6.
Different definitions of disability progression by Expanded Disability Status Scale (EDSS) may influence frequency and/or time to event.
In this multicenter cohort study, we included PPMS patients with follow-up ≥24 months and ≥3 available EDSS scores overall (≥1 per year). We applied 672 definitions of disability progression including different minimal EDSS increase, required confirmation and fixed/roving-baseline score.
We analyzed follow-up periods from 131 PPMS patients (median age at baseline 53.0 years [45.0 - 63.0], 51.9 % female, median follow-up 3.9 years [2.6 - 6.0], median baseline EDSS 4.0 [2.5 - 6.0]). The most sensitive definition of a progression event was an unconfirmed increase of ≥0.5 points with a roving baseline (81.8 % event rate). The least sensitive definition was an increase of ≥1.0 points with a fixed baseline, minimal distance to reference 48 weeks, and confirmed at ≥48 weeks (28.4 % event rate). Comparing roving vs. fixed baseline over all cutoffs and confirmation definitions, average time to progression was 227 days shorter applying the roving baseline (1405 days [550 - 2653] vs. 1632 days [760 - 2653]).
Different definitions of disability progression result in significantly differing rates of disability progression, which may influence study results and create confusion in clinical practice.
扩展残疾状态量表（EDSS）对残疾进展的不同定义可能会影响事件发生的频率和/或时间。
在这项多中心队列研究中，我们纳入了随访时间≥24个月且总体上有≥3个可用EDSS评分（每年≥1个）的继发进展型多发性硬化（PPMS）患者。我们应用了672种残疾进展的定义，包括不同的最小EDSS增加量、所需的确认以及固定/移动基线评分。
我们分析了131例PPMS患者的随访期（基线时的中位年龄为53.0岁[45.0 - 63.0]，51.9%为女性，中位随访时间为3.9年[2.6 - 6.0]，基线EDSS中位数为4.0[2.5 - 6.0]）。进展事件最敏感的定义是移动基线时未经确认的增加≥0.5分（事件发生率为81.8%）。最不敏感的定义是固定基线时增加≥1.0分，与参考值的最小间隔为48周，并在≥48周时得到确认（事件发生率为28.4%）。在所有临界值和确认定义下比较移动基线与固定基线，应用移动基线时进展的平均时间短227天（1405天[550 - 2653]对1632天[760 - 2653]）。
残疾进展的不同定义导致残疾进展率存在显著差异，这可能会影响研究结果并在临床实践中造成混淆。
