Abstract

Objective

To assess whether published scales for measuring upper motor neuron burden (UMNB) show longitudinal change in patients with primary lateral sclerosis (PLS). Design: Retrospective calculation of three UMNB scales on a prospectively collected dataset from 53 patients with PLS enrolled in a longitudinal natural history study with at least 2 evaluation visits. UMNB scales were calculated according to their published descriptions. Non-linear trends over time of UMNB scale scores and slopes were calculated for each patient and correlations between UMNB scores and clinical measures were assessed. Results: All three UMNB scales exhibited increasing scores over the first 7.8 years of symptoms, with a flattening in slope after approximately 8 years. A scale used in imaging studies and the UPENN UMNS scale provided a better fit to the dataset than the MGH UMNB scale. All three UMNB scales exhibited moderate correlations with some clinical measures of movement, such as finger-tapping rate and timed gait. Correlations were strongest for the UPENN UMNS, which was also moderately correlated with the revised ALS functional rating scale. Conclusion: In a cohort of PLS patients enrolled in a natural history study, the three UMNB scales exhibited modest linear increases over the first 8 years of symptoms, followed by a plateau. Future clinical trials in PLS should consider stratification of patients by disease duration. UMNB scales may be useful secondary outcomes, but more sensitive primary outcome measures are needed for clinical trials for PLS.

中文翻译：

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原发性侧索硬化中上运动神经元负荷量表的纵向评价

摘要

客观的

评估已发布的测量上运动神经元负荷 (UMNB) 的量表是否显示原发性侧索硬化症 (PLS) 患者的纵向变化。设计：回顾性计算来自 53 名 PLS 患者的前瞻性收集数据集的三个 UMNB 量表，该 PLS 患者参加了至少 2 次评估访问的纵向自然史研究。UMNB 量表是根据他们公布的描述计算的。计算每位患者的 UMNB 量表评分和斜率随时间的非线性趋势，并评估 UMNB 评分和临床测量之间的相关性。结果：在症状出现的前 7.8 年中，所有三个 UMNB 量表的分数都在增加，大约 8 年后斜率趋于平缓。成像研究中使用的量表和 UPENN UMNS 量表比 MGH UMNB 量表更适合数据集。所有三个 UMNB 量表都与一些临床运动测量值表现出中等相关性，例如手指敲击率和定时步态。UPENN UMNS 的相关性最强，这也与修订后的 ALS 功能评定量表中度相关。结论：在参加自然史研究的一组 PLS 患者中，三个 UMNB 量表在症状出现的前 8 年表现出适度的线性增加，然后是平稳期。PLS 的未来临床试验应考虑按病程对患者进行分层。UMNB 量表可能是有用的次要结果，但 PLS 临床试验需要更敏感的主要结果测量。