Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL

Philip A Powell; Jill Carlton; Donna Rowen; Fleur Chandler; Michela Guglieri; John E Brazier

doi:10.1212/WNL.0000000000011896

Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL

Neurology. 2021 May 11;96(19):e2438-e2450. doi: 10.1212/WNL.0000000000011896. Epub 2021 Mar 30.

Authors

Philip A Powell¹, Jill Carlton², Donna Rowen², Fleur Chandler², Michela Guglieri², John E Brazier²

Affiliations

¹ From 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.
² From 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.

Abstract

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.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adult
Humans
Interview, Psychological / methods
Interview, Psychological / standards*
Male
Middle Aged
Muscular Dystrophy, Duchenne / diagnosis*
Muscular Dystrophy, Duchenne / psychology*
Quality of Life / psychology*
Reproducibility of Results
Surveys and Questionnaires / standards*