Measuring quality of life in muscular dystrophy

Carla M. Bann, Richard T. Abresch, Barbara Biesecker, Kristin Caspers Conway, Chad Heatwole, Holly Peay, Peter Scal, Jonathan Strober, Karen Uzark, Jodi Wolff, Marjorie Margolis, Angela Blackwell, Natalie Street, Angela Montesanti, Julie Bolen

Research output: Contribution to journalArticlepeer-review

11 Scopus citations


Objectives: The objectives of this study were to develop a conceptual model of quality of life (QOL) in muscular dystrophies (MDs) and review existing QOL measures for use in the MD population. Methods: Our model for QOL among individuals with MD was developed based on a modified Delphi process, literature review, and input from patients and patient advocacy organizations. Scales that have been used to measure QOL among patients with MD were identified through a literature review and evaluated using the COSMIN (Consensus-Based Standards for the Selection of Health Measurement Instruments) checklist. Results: The Comprehensive Model of QOL in MD (CMQM) captures 3 broad domains of QOL (physical, psychological, and social), includes factors influencing self-reported QOL (disease-related factors, support/resources, and expectations/aspirations), and places these concepts within the context of the life course. The literature review identified 15 QOL scales (9 adult and 6 pediatric) that have been applied to patients with MD. Very few studies reported reliability data, and none included data on responsiveness of the measures to change in disease progression, a necessary psychometric property for measures included in treatment and intervention studies. No scales captured all QOL domains identified in the CMQM model. Conclusions: Additional scale development research is needed to enhance assessment of QOL for individuals with MD. Item banking and computerized adaptive assessment would be particularly beneficial by allowing the scale to be tailored to each individual, thereby minimizing respondent burden.

Original languageEnglish (US)
Pages (from-to)1034-1042
Number of pages9
Issue number10
StatePublished - Mar 10 2015

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Publisher Copyright:
© 2015 American Academy of Neurology.

Copyright 2015 Elsevier B.V., All rights reserved.

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