Table_2_The Measurement Performance of the Parkinson's Disease Activities of Daily Living, Interference, and Dependence Instrument.DOCX

Parkinson's disease is a neurodegenerative disease that can be associated with motor fluctuations that result in substantial negative impact to an individual's activities of daily living. Understanding the patient's perspective about the impact of Parkinson's disease therapies is an important part of drug development and shared treatment decision-making. The objective of this research was to examine the structure, scoring, internal consistency, test-retest reliability, and concurrent and known groups validity of the Parkinson's Disease Activities of Daily Living, Interference and Dependence (PD-AID) instrument, a new, patient-reported outcomes instrument, developed to assess the clinical benefit of Parkinson's disease treatment from the patient's perspective. This was a non-interventional study among persons with mild-to-moderate Parkinson's disease currently using and responding to L-Dopa. The structure of the measure was confirmed applying item response theory to data from baseline, supporting 4 candidate scores. Baseline Patient Global Impression of Severity ratings facilitated known-groups analysis. Data from all participants were used to estimate test-retest reliability. Concurrent validity was assessed using correlations with related measures. Participants (n = 94) were mean age 69 years (mean time since diagnosis 6.9 years); 34 experienced L-Dopa-related dyskinesia. Psychometric models supported 4 candidate scoring regimes for the PD-AID. All exhibited adequate reliability and validity characteristics and strong internal consistency. Correlations with reference measures were in the expected direction and range of magnitude. Analyses supported the PD-AID as fit-for-purpose, producing psychometrically sound scores. Further research to confirm the measurement properties of the PD-AID in an expanded sample and to establish thresholds for meaningful score changes is recommended.

帕金森病（Parkinson's Disease, PD）是一种神经退行性疾病，常伴随运动波动，可对患者的日常生活活动造成显著负面影响。了解患者对帕金森病治疗方案影响的主观认知，是药物研发与共同制定治疗决策的重要组成部分。本研究旨在考察新型患者报告结局量表——帕金森病日常生活活动、干扰与依赖量表（Parkinson's Disease Activities of Daily Living, Interference and Dependence, PD-AID）的结构、计分方式、内部一致性、重测信度，以及同时效度与已知群体效度；该量表专为从患者视角评估帕金森病治疗的临床获益而开发。
本研究为一项非干预性研究，纳入当前正在使用左旋多巴（L-Dopa）且对其应答良好的轻至中度帕金森病患者。通过对基线数据应用项目反应理论（Item Response Theory, IRT）验证了该量表的结构，支持4种候选计分方案。基线患者整体印象严重程度评分（Baseline Patient Global Impression of Severity）助力完成已知群体效度分析。本研究纳入所有受试者的数据以评估重测信度，同时效度则通过与相关测量工具的相关性分析进行评估。本研究共纳入94名受试者，平均年龄69岁，确诊至入组的平均病程为6.9年；其中34名受试者出现左旋多巴相关异动症。
心理计量学模型验证了PD-AID的4种候选计分体系，所有计分体系均表现出良好的信度、效度特征与优异的内部一致性。与参照测量工具的相关性符合预期的方向与强度范围。分析结果表明PD-AID符合临床使用需求，其计分结果具备可靠的心理计量学属性。本研究建议后续开展进一步研究，在更大样本中验证PD-AID的测量属性，并确立具有临床意义的评分变化阈值。