Potashman Michele H, Popoff Evan, Powell Lauren C, Beiner Melissa Wolfe, Mackenzie Ainsley, Coric Vlad, Subramony Sub, Synofzik Matthis, Schmahmann Jeremy, L'Italien Gilbert
Biohaven Pharmaceuticals, Inc, 215 Church Street, New Haven, CT, 06510, USA.
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada.
Neurol Ther. 2025 Apr;14(2):527-545. doi: 10.1007/s40120-024-00708-4. Epub 2025 Jan 13.
The Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a valid, highly utilized measure for assessing ADL impacts in patients with Friedreich ataxia. We provide evidence of the psychometric validity of the FARS-ADL in two cohorts of patients with spinocerebellar ataxia (SCA).
Using data from a cohort of real-world subjects with SCA (recruited at Massachusetts General Hospital [MGH]; n = 33) and a phase 3 trial of troriluzole in adults with SCA (NCT03701399 [Study 206]; n = 217), comprising a subset of patients with the SCA3 genotype (n = 89), the psychometric measurement properties and minimal change thresholds of the FARS-ADL were examined.
Ceiling effects for the FARS-ADL were absent within the MGH cohort while floor effects were observed for eight of nine items. Excellent internal consistency reliability was observed (α = 0.88; α = 0.86-0.87), and item-to-total correlations were acceptable (r = 0.55-0.89 per item). Convergent and divergent validity were supported with strong correlations demonstrated between FARS-ADL and scales measuring similar concepts (Neuro-QOL [Upper], Neuro-QOL [Lower], PROM-ADL, PROM-PHYS, and FARS-FUNC; all P < 0.001) and weaker correlations shown between measures of differing constructs. A two- to three-point threshold for meaningful changes was supported as 0.5 × SD = 2.43, SEM = 2.19. Mean changes from baseline for subjects classified as "improved," "no change," or "deteriorated" were -0.54, 0.22, and 1.47, respectively. Similar trends were observed in the Study 206 all-SCA and SCA3 cohorts.
Psychometric evaluation showed that the FARS-ADL performed well on analyses examining the reliability and validity of the measure and can detect meaningful changes in patients with SCA, including those with SCA3.
ClinicalTrials.gov identifier, NCT03701399 (Study 206).
弗里德赖希共济失调日常生活活动评定量表（FARS - ADL）是一种有效且被广泛应用的工具，用于评估弗里德赖希共济失调患者日常生活活动的影响。我们提供了FARS - ADL在两组脊髓小脑共济失调（SCA）患者中的心理测量效度证据。
使用来自一组现实世界的SCA受试者（在马萨诸塞州总医院[MGH]招募；n = 33）以及一项曲鲁唑治疗成人SCA的3期试验（NCT03701399[研究206]；n = 217）的数据，其中包括一部分SCA3基因型患者（n = 89），对FARS - ADL的心理测量特性和最小变化阈值进行了检查。
MGH队列中未观察到FARS - ADL的天花板效应，而九个项目中有八个观察到地板效应。观察到极好的内部一致性信度（α = 0.88；α = 0.86 - 0.87），且项目与总分的相关性可接受（每个项目r = 0.55 - 0.89）。FARS - ADL与测量相似概念的量表（神经生活质量量表[上肢]、神经生活质量量表[下肢]、患者报告结局 - 日常生活活动量表、患者报告结局 - 身体功能量表和FARS - 功能量表；所有P < 0.001）之间显示出强相关性，支持了收敛效度和区分效度，而不同结构的测量之间显示出较弱的相关性。支持有意义变化的两点到三点阈值为0.5×标准差 = 2.43，标准误 = 2.19。分类为“改善”“无变化”或“恶化”的受试者从基线的平均变化分别为 - 0.54、0.22和1.47。在研究206的所有SCA和SCA3队列中观察到类似趋势。
心理测量评估表明，FARS - ADL在检验该测量工具的信度和效度的分析中表现良好，并且可以检测SCA患者（包括SCA3患者）中有意义的变化。
ClinicalTrials.gov标识符，NCT03701399（研究206）。
