Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy

Anna Mayhew, Elena S. Mazzone, Michelle Eagle, Tina Duong, Maria Ash, Valerie Decostre, Marlene Vandenhauwe, Katrijn Klingels, Julaine Florence, Marion Main, Flaviana Bianco, Erik Henrikson, Laurent Servais, Giles Campion, Elizabeth Vroom, Valeria Ricotti, Natalie Goemans, Craig Mcdonald, Eugenio Mercuri

Research output: Contribution to journalArticle

104 Scopus citations

Abstract

Aim: 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). Method: 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. Results: 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. Interpretation: The module was developed using innovative approaches and will be useful for designing clinical trials.

Original languageEnglish
Pages (from-to)1038-1045
Number of pages8
JournalDevelopmental Medicine and Child Neurology
Volume55
Issue number11
DOIs
StatePublished - Nov 1 2013

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    Mayhew, A., Mazzone, E. S., Eagle, M., Duong, T., Ash, M., Decostre, V., Vandenhauwe, M., Klingels, K., Florence, J., Main, M., Bianco, F., Henrikson, E., Servais, L., Campion, G., Vroom, E., Ricotti, V., Goemans, N., Mcdonald, C., & Mercuri, E. (2013). Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy. Developmental Medicine and Child Neurology, 55(11), 1038-1045. https://doi.org/10.1111/dmcn.12213