为手机测步态数据设定多发性硬化症的临床显著变化阈值。
Establishing the Minimal Clinically Important Difference (MCID) for Smartphone-Derived Gait Measures in Multiple Sclerosis
- 通过多锚点法计算手机步行测试的步态指标变化。
- 发现步速下降0.16米/秒等六项关键变化阈值。
- 适合用于远程监测和新药临床试验评估。
背景：数字健康技术可实现多发性硬化症（MS）患者频繁、远程的步态监测。但要区分日常波动与真实疾病进展，需明确最小临床重要差异（MCID）。目前针对数字步态指标的阈值研究有限。目的：建立反映MS进展的数字步态测量MCID。方法：在CONSONANCE（NCT03523858）研究中，采用智能手机每日远程进行两分钟步行测试，收集进展期MS患者的数字步态数据。基于锚定法，计算第96周时在蒂姆25英尺行走测试、行走评分、扩展残疾状态量表或12项多发性硬化症步行量表上出现临床显著恶化的患者，其数字步态指标的中位数变化，并通过三角验证确定MCID估计值。结果：243例进展期MS患者（女性125例，占比51%；平均年龄49.3岁，标准差9.3；平均EDSS 4.8，标准差1.4）在基线及第96周有可用数据。各锚点间中位数变化一致。三角验证得出的MCID为：步速下降0.16米/秒，步速按行走时间缩放后下降0.18米/秒，步时增加0.06秒，步长减少0.07米，总步数减少28步，总行走距离减少24米。结论：这些MCID为解读有意义的步态变化提供了框架，有助于将数字测量整合进MS疗效评估。不仅可支持新型临床试验终点，还可实现客观的现实世界监测，推动个性化患者管理。
原文摘要 · Abstract (English)
Background: Digital health technologies allow for frequent, remote gait monitoring in people with multiple sclerosis (MS). However, to differentiate daily variability from actual disease progression in longitudinal data, established minimal clinically important differences (MCID) are required. Currently, there is limited literature defining these thresholds for digital gait metrics. Objective: To establish MCIDs for digital gait measures reflecting progression in MS. Methods: Digital gait measures were captured via daily, remote, smartphone-based Two-Minute Walk Tests in CONSONANCE (NCT03523858), a phase 3b study of ocrelizumab in progressive MS. Using an anchor-based approach, median changes from baseline at Week 96 on digital gait measures were computed for patients showing clinically meaningful worsening on either Timed 25-Foot Walk, Ambulation Score, Expanded Disability Status Scale, or 12-item Multiple Sclerosis Walking Scale. These changes were subsequently triangulated to derive the MCID estimates. Results: 243 patients with progressive MS (female: n=125 (51%); mean [SD] age: 49.3 [9.3]; mean [SD] EDSS: 4.8 [1.4]) had digital gait data available at baseline and Week 96. Median changes were generally consistent across anchors. Triangulated MCIDs are: Step Velocity = -0.16 m/s, Step Velocity Scaled to Walking Time = -0.18 m/s, Step Duration = 0.06 s, Step Length = -0.07 m, Total Number of Steps = -28, and Total Distance Walked = -24 m. Conclusion: These MCIDs provide a framework for interpreting meaningful gait changes and integrating digital measures into MS outcome evaluation. Beyond facilitating novel clinical trial endpoints to evaluate treatment efficacy, they enable objective, real-world monitoring to advance personalized patient care.
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