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Updated: Nov 11, 2025
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL
Philip A Powell1, Jill Carlton2, Donna Rowen2
1From the School of Health and Related Research (P.A.P., J.C., D.R., J.E.B.), University of Sheffield; Duchenne UK (F.C.), London; and John Walton Dystrophy Research Centre (M.G.), Institute of Genetic Medicine, University of Newcastle and Newcastle Hospitals NHS Foundation Trust, UK. p.a.powell@sheffield.ac.uk.
Objective:
Based on concerns about existing patient-reported outcome measures (PROMs) for assessing quality of life (QoL) in Duchenne muscular dystrophy (DMD), we describe the mixed methods development of a new QoL PROM for use in boys and men with DMD: the DMD-QoL.
Methods:
The DMD-QoL was developed in 3 stages. First, draft items were generated from 18 semistructured qualitative interviews with boys and men with DMD, analyzed using framework analysis. Second, cognitive debriefing interviews with patients (n = 10), clinicians (n = 8), and patients' parents (n = 10) were undertaken, and a reduced item set was selected and refined. Third, psychometric data on the draft items from a cross-sectional online survey (n = 102) and stakeholder input from patients and patients' parents were used to produce the final questionnaire. Patient and public involvement and engagement was embedded throughout the process.
Results:
From an initial draft of 47 items, a revised set of 27 items was produced at stage 2, and this set was further refined at stage 3 to generate the DMD-QoL, a 14-item QoL PROM. The DMD-QoL is designed for use from 7 years of age by proxy report and from 10 years of age by self-report or proxy report. The final measure showed good psychometric properties.
Conclusion:
The DMD-QoL is a new 14-item QoL PROM for boys and men with DMD, with demonstrable content and face validity.
