title: Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy.
abstract: OBJECTIVE
  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).
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  The module was developed using innovative approaches and will be useful for designing clinical trials.
authors.affiliation: Institute of Genetic Medicine, Newcastle Upon Tyne, UK.
authors.forename: Anna
authors.initials: A
authors.lastname: Mayhew
authors.forename: Elena S
authors.initials: ES
authors.lastname: Mazzone
authors.forename: Michelle
authors.initials: M
authors.lastname: Eagle
authors.forename: Tina
authors.initials: T
authors.lastname: Duong
authors.forename: Maria
authors.initials: M
authors.lastname: Ash
authors.forename: Valerie
authors.initials: V
authors.lastname: Decostre
authors.forename: Marlene
authors.initials: M
authors.lastname: Vandenhauwe
authors.forename: Katrijn
authors.initials: K
authors.lastname: Klingels
authors.forename: Julaine
authors.initials: J
authors.lastname: Florence
authors.forename: Marion
authors.initials: M
authors.lastname: Main
authors.forename: Flaviana
authors.initials: F
authors.lastname: Bianco
authors.forename: Erik
authors.initials: E
authors.lastname: Henrikson
authors.forename: Laurent
authors.initials: L
authors.lastname: Servais
authors.forename: Giles
authors.initials: G
authors.lastname: Campion
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Vroom
authors.forename: Valeria
authors.initials: V
authors.lastname: Ricotti
authors.forename: Natalie
authors.initials: N
authors.lastname: Goemans
authors.forename: Craig
authors.initials: C
authors.lastname: McDonald
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000203:Activities of Daily Living; D004185:Disability Evaluation; D006801:Humans; D009069:Movement Disorders / Q000175:diagnosis* / Q000209:etiology; D020388:Muscular Dystrophy, Duchenne / Q000150:complications / Q000473:pathology*; D011594:Psychometrics; D015203:Reproducibility of Results; D012680:Sensitivity and Specificity; D034941:Upper Extremity / Q000503:physiopathology*; D016138:Walking