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 K Henricson, Laurent Servais, Giles Campion, Elizabeth Vroom, Valeria Ricotti, Natalie Goemans, Craig M McDonald, Eugenio Mercuri

Research output: Contribution to journalArticle

95 Citations (Scopus)

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

Fingerprint

Duchenne Muscular Dystrophy
Upper Extremity
Patient Advocacy
Activities of Daily Living
Natural History
Psychometrics
Reproducibility of Results
Industry
Outcome Assessment (Health Care)
Clinical Trials

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Developmental Neuroscience

Cite this

Mayhew, A., Mazzone, E. S., Eagle, M., Duong, T., Ash, M., Decostre, V., ... 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

Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy. / Mayhew, Anna; Mazzone, Elena S.; Eagle, Michelle; Duong, Tina; Ash, Maria; Decostre, Valerie; Vandenhauwe, Marlene; Klingels, Katrijn; Florence, Julaine; Main, Marion; Bianco, Flaviana; Henricson, Erik K; Servais, Laurent; Campion, Giles; Vroom, Elizabeth; Ricotti, Valeria; Goemans, Natalie; McDonald, Craig M; Mercuri, Eugenio.

In: Developmental Medicine and Child Neurology, Vol. 55, No. 11, 11.2013, p. 1038-1045.

Research output: Contribution to journalArticle

Mayhew, A, Mazzone, ES, Eagle, M, Duong, T, Ash, M, Decostre, V, Vandenhauwe, M, Klingels, K, Florence, J, Main, M, Bianco, F, Henricson, EK, Servais, L, Campion, G, Vroom, E, Ricotti, V, Goemans, N, McDonald, CM & Mercuri, E 2013, 'Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy', Developmental Medicine and Child Neurology, vol. 55, no. 11, pp. 1038-1045. https://doi.org/10.1111/dmcn.12213
Mayhew, Anna ; Mazzone, Elena S. ; Eagle, Michelle ; Duong, Tina ; Ash, Maria ; Decostre, Valerie ; Vandenhauwe, Marlene ; Klingels, Katrijn ; Florence, Julaine ; Main, Marion ; Bianco, Flaviana ; Henricson, Erik K ; Servais, Laurent ; Campion, Giles ; Vroom, Elizabeth ; Ricotti, Valeria ; Goemans, Natalie ; McDonald, Craig M ; Mercuri, Eugenio. / Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy. In: Developmental Medicine and Child Neurology. 2013 ; Vol. 55, No. 11. pp. 1038-1045.
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