Klingels K, Mayhew A G, Mazzone E S, Duong T, Decostre V, Werlauff U, Vroom E, Mercuri E, Goemans N M
Department of Rehabilitation Sciences, KU Leuven, Leuven, Belgium.
Rehabilitation Research Center (REVAL), Biomed, Hasselt University, Diepenbeek, Belgium.
Dev Med Child Neurol. 2017 Feb;59(2):224-231. doi: 10.1111/dmcn.13277. Epub 2016 Sep 26.
To develop a patient-reported outcome measure (PROM) assessing upper limb function related to activities of daily living (ADL) that cannot be observed in a clinical setting, specifically for patients with Duchenne muscular dystrophy (DMD) across a wide age range, applicable in the different stages of the disease.
The developmental process was based on US Food and Drug Administration guidelines. This included item generation from a systematic review of existing tools and expert opinion on task difficulty and relevance, involving individuals with DMD. Cultural aspects affecting ADL were taken into consideration to make this tool applicable to the broad DMD community. Items were selected in relation to a conceptual framework reflecting disease progression covering the full range of upper limb function across different ADL domains.
After pilot testing and iterative Rasch analyses, redundant or clinically irrelevant items were removed. The final questionnaire consists of 32 items covering four domains of ADL (food, self-care, household and environment, leisure and communication). Test-retest reliability was excellent.
A DMD-specific upper limb PROM was developed on the basis of clinical relevance and psychometric robustness. Its main purpose is to document the patient self-reported natural history of DMD and assess the efficacy of interventions.
开发一种患者报告结局量表（PROM），用于评估与日常生活活动（ADL）相关的上肢功能，这些功能在临床环境中无法观察到，特别是针对广泛年龄范围内的杜氏肌营养不良症（DMD）患者，适用于疾病的不同阶段。
开发过程基于美国食品药品监督管理局的指南。这包括从对现有工具的系统评价以及关于任务难度和相关性的专家意见中生成条目，涉及DMD患者。考虑到影响ADL的文化因素，以使该工具适用于广泛的DMD群体。根据反映疾病进展的概念框架选择条目，该框架涵盖不同ADL领域的全范围上肢功能。
经过预试验和迭代拉施分析，去除了冗余或临床无关的条目。最终问卷由32个条目组成，涵盖ADL的四个领域（饮食、自我护理、家庭与环境、休闲与交流）。重测信度极佳。
基于临床相关性和心理测量稳健性开发了一种特定于DMD的上肢PROM。其主要目的是记录患者自我报告的DMD自然病史并评估干预措施的疗效。
