Institute of Genetic Medicine, Newcastle Upon Tyne, UK.
Dev Med Child Neurol. 2013 Nov;55(11):1038-45. doi: 10.1111/dmcn.12213. Epub 2013 Aug 1.
An international Clinical Outcomes Group consisting of clinicians, scientists, patient advocacy groups, and industries identified a need for a scale to measure motor performance of the upper limb. We report the steps leading to the development of the Performance of the Upper Limb (PUL), a tool specifically designed for assessing upper limb function in ambulant and non-ambulant patients with Duchenne muscular dystrophy (DMD).
The development of the PUL followed a number of steps, from the systematic review and a preliminary study exploring the suitability of the existing measures, to the application of a pilot version in a multicentric setting, with Rasch analysis of the preliminary results, leading to a revised pro forma.
The PUL was specifically designed for DMD, with a conceptual framework reflecting the progression of weakness and natural history of functional decline in DMD. Modern psychometric methods were used to create a scale with robust internal reliability, validity, and hierarchical scalability; males with DMD and their families were involved iteratively throughout the process of the clinician-reported outcome assessment tool development to establish clinical meaningfulness and relevance of individual PUL items to activities of daily living.
The module was developed using innovative approaches and will be useful for designing clinical trials.
一个由临床医生、科学家、患者权益团体和行业代表组成的国际临床结果研究小组，发现需要一个衡量上肢运动表现的量表。我们报告了制定上肢表现量表（PUL）的步骤，该量表专门用于评估杜氏肌营养不良症（DMD）患者的上肢功能，无论其是否能够走动。
PUL 的开发经历了多个步骤，从系统评价和初步研究探索现有测量工具的适用性，到在多中心环境中应用试点版本，并对初步结果进行 Rasch 分析，最终形成修订的草案。
PUL 是专门为 DMD 设计的，其概念框架反映了 DMD 中肌肉无力的进展和功能下降的自然史。现代心理测量学方法被用于创建一个具有强大内部可靠性、有效性和层次可扩展性的量表；DMD 男性及其家属在整个临床医生报告结果评估工具开发过程中反复参与，以确定个体 PUL 项目对日常生活活动的临床意义和相关性。
该模块是使用创新方法开发的，将有助于设计临床试验。
