原发性侧索硬化症的疾病进展测量。
Measuring disease progression in primary lateral sclerosis.
机构信息
Department of Neurology, Columbia University Medical Center, New York, NY, USA.
Department of Neurology, University of Pennsylvania, Philadelphia, PA, USA.
出版信息
Amyotroph Lateral Scler Frontotemporal Degener. 2020 Nov;21(sup1):59-66. doi: 10.1080/21678421.2020.1837179.
Quantitative measures of disease severity are essential outcome measures for clinical trials. The slow progression of disease in primary lateral sclerosis (PLS) requires clinical measures that are sensitive to changes occurring within the time frame of a clinical trial. Proposed clinical outcome measures include the PLS functional rating scale (PLSFRS), burden scores derived from clinical examination findings, and quantitative measures of motor performance. The PLSFRS has good inter-rater reliability and showed greater longitudinal change over 6- and 12-months compared to the revised ALS functional rating scale. Examination-based upper motor neuron burden (UMNB) scales also have good reliability, and longitudinal studies are in process. Quantitative measures of strength, dexterity, gait, and speech have the potential to provide objective and precise measures of clinical change, but have been the least studied in persons with PLS.
疾病严重程度的定量测量是临床试验的重要结果衡量指标。原发性侧索硬化症（PLS）的疾病进展缓慢，需要在临床试验时间范围内对变化敏感的临床测量方法。拟议的临床结果衡量指标包括 PLS 功能评定量表（PLSFRS）、源于临床检查结果的负担评分，以及运动表现的定量测量。PLSFRS 具有良好的评分者间信度，与修订后的 ALS 功能评定量表相比，在 6 个月和 12 个月时显示出更大的纵向变化。基于检查的上运动神经元负担（UMNB）量表也具有良好的可靠性，且纵向研究正在进行中。力量、灵巧度、步态和言语的定量测量有可能提供临床变化的客观、精确衡量指标，但在 PLS 患者中研究最少。
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