Article

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.
CI - (c) 2013 Mac Keith Press.

Langue : ANGLAIS