Buchholz Maresa, Pfaff Michelle, Iskandar Audrey, Reetz Kathrin, Schulz Jörg B, Grobe-Einsler Marcus, Klockgether Thomas, Michalowsky Bernhard
Patient-Reported Outcomes and Health Economics Research, Deutsches Zentrum für Neurodegenerative Erkrankungen/German Center for Neurodegenerative Diseases (DZNE), Site Rostock/Greifswald, Ellernholzstrasse 1-2, 17489, Greifswald, Germany.
Department of Neurology, RWTH Aachen University, Pauwelsstrasse 30, 52074, Aachen, Germany.
Neurol Ther. 2025 Feb;14(1):379-398. doi: 10.1007/s40120-024-00694-7. Epub 2024 Dec 30.
Friedreich Ataxia (FA) is a multisystem neurodegenerative disease. Affected individuals rely on mobility assistive technologies (MAT) (e.g. wheelchairs) and require long-term treatments and care. To analyse the patients' health-related quality of life (HRQoL), the EuroQol 5 Dimension 3 Level survey (EQ-5D-3L)-a widely used and recommended generic measure-is used in clinical and in health economic studies. Concerns about using the instrument in mobility-impaired individuals who might have difficulties finding appropriate response options for mobility-related items led us to investigate how the 3L dimensions perform in patients with FA using or not using MAT.
Using longitudinal data from 607 patients with FA of the EFACTS study (from baseline to the 3-year follow-up), we analysed the acceptability, distribution properties, validity, and responsiveness of the EQ-5D-3L, focusing on the mobility item. Analyses were stratified for patients without and with different MAT-usage.
We identified that n = 177 patients used no MAT, n = 299 a wheelchair and n = 131 walking aids. The mobility item non-response was highest in wheelchair users (6.8%) and lowest in patients without MAT. Walking aid users showed the least variability, all selecting the mid-response option "some problems" for mobility. The mobility item correlated moderately with disease severity (r = 0.35) and the activities of daily living scale (r = 0.36) in wheelchair users. No correlation exists for walking aid users. The strongest health changes occurred for wheelchair users, the weakest for walking aid users. The mobility dimensions showed the highest amount of no changes.
The EQ-5D-3L's mobility item has limitations in MAT users, particularly in walking aid users, due to a tendency towards mid-responses. These limitations may affect the efficacy and (cost)effectiveness conclusions drawn from interventions and clinical trials with mobility-impaired individuals. Further research is needed to explore the understanding and interpretation of responses of the EQ-5D in patients with FA with mobility support.
ClinicalTrials.gov identifier NCT02069509.
弗里德赖希共济失调（FA）是一种多系统神经退行性疾病。受影响的个体依赖移动辅助技术（MAT）（如轮椅），并需要长期治疗和护理。为了分析患者的健康相关生活质量（HRQoL），欧洲五维度健康量表3级调查问卷（EQ-5D-3L）——一种广泛使用且被推荐的通用测量工具——被用于临床和卫生经济学研究。由于担心在行动不便的个体中使用该工具时，他们可能难以找到与行动相关项目的合适回答选项，我们开展了此项研究，以调查EQ-5D-3L的3级维度在使用或未使用MAT的FA患者中的表现。
我们使用了EFACTS研究中607例FA患者的纵向数据（从基线到3年随访），分析了EQ-5D-3L的可接受性、分布特性、有效性和反应性，重点关注行动项目。分析针对未使用MAT和使用不同MAT的患者进行了分层。
我们确定，n = 177例患者未使用MAT，n = 299例使用轮椅，n = 131例使用助行器。行动项目无回答率在轮椅使用者中最高（6.8%），在未使用MAT的患者中最低。使用助行器的患者变异性最小，所有人在行动方面都选择了中间回答选项“有些问题”。在轮椅使用者中，行动项目与疾病严重程度（r = 0.35）和日常生活活动量表（r = 0.36）呈中度相关。使用助行器的患者不存在相关性。轮椅使用者的健康变化最大，使用助行器的患者最小。行动维度显示无变化的情况最多。
由于倾向于选择中间回答，EQ-5D-3L的行动项目在MAT使用者中存在局限性，尤其是在使用助行器的患者中。这些局限性可能会影响从针对行动不便个体的干预措施和临床试验中得出的疗效和（成本）效益结论。需要进一步研究以探索FA行动不便患者对EQ-5D回答的理解和解释。
ClinicalTrials.gov标识符NCT02069509。
