Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

Research output: Contribution to journalResearch articleContributedpeer-review


  • Western University
  • Newcastle University
  • University of Rochester
  • Leeds Teaching Hospitals NHS Trust
  • University of California at Los Angeles
  • Alder Hey Children's NHS Foundation Trust
  • University of Utah
  • The Shrewsbury and Telford Hospital NHS Trust
  • University Hospitals Birmingham NHS Foundation Trust
  • IRCCS Fondazione Istituto Neurologico Carlo Besta - Milano
  • Nationwide Children’s Hospital
  • Ann and Robert H. Lurie Children's Hospital of Chicago
  • Great Ormond Street Hospital for Children NHS Trust
  • Harvard University
  • University of Calgary
  • University of Turin
  • University of New Mexico
  • Nemours Children's Hospital
  • St. Jude Children Research Hospital
  • Pennsylvania State University
  • University of Colorado Denver
  • University of California at Davis
  • University of Duisburg-Essen
  • Children's University Hospital
  • University of Missouri
  • University of Kansas
  • University of Freiburg
  • University of Bonn
  • University of Messina
  • University of North Carolina at Chapel Hill
  • Royal Manchester Children's Hospital
  • University of Ottawa
  • University of Padua
  • Vanderbilt University
  • Virginia Commonwealth University
  • Children's National Medical Center


Knowledge of health related quality of life (HRQOL) in the immediate phase following DMD diagnosis has not been well-characterized. It is important to understand HRQOL early in disease for both clinical care and studies of treatment. The relationship between parent-proxy and child self-report HRQOL and their associations with medical, psycho-social and behavioral symptoms deserve study. In this study HRQOL was measured using the PedsQL inventory in parent/caregiver and corticosteroid-naïve boys (ages 4 to 7 years) participating in the FOR-DMD study. Agreement between the parent-proxy report and the boys’ self-report HRQOL was measured using intraclass correlation coefficients (ICCs). Factors associated with HRQOL, including standardized psychosocial and behavioral measures in this cross-sectional sample, were explored using correlations. The results showed that the level of agreement between 70 dyads of child self-report and parent-proxy ratings of HRQOL was poor for the generic PedsQL total score (ICC=0.48, 95% CI (0.23, 0.66)) and its subscale scores, and was similarly low for the neuromuscular disease module (ICC=0.24, 95% CI (0.00, 0.45)). Parents rated their child's HRQOL as poorer than the children rated themselves in all scales. Psychosocial outcome measures were more highly associated with HRQOL measures than disease severity or patient demographic variables. In the early phases of DMD, child and parent-proxy HRQOL ratings were discordant. In early DMD, psychosocial and behavioral aspects appear to be more relevant to HRQOL than disease severity factors.


Original languageEnglish
Pages (from-to)1161-1168
Number of pages8
JournalNeuromuscular disorders
Issue number11
Publication statusPublished - Nov 2021

External IDs

PubMed 34489153



  • Duchenne muscular dystrophy, Health related quality of life, Psychosocial

Library keywords