1. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy
- Author
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Giuseppe Vita, Helen Roper, Tiziana Mongini, Leslie Morrison, Nancy L. Kuntz, Russell J. Butterfield, Perry B. Shieh, Hugh J. McMillan, Erik K Henricson, Adnan Y. Manzur, Elaine McColl, Federica Ricci, Ulrike Schara-Schmidt, Anne-Marie Childs, Mathula Thangarajh, Gian Luca Vita, Peter B. Kang, Imelda Hughes, Jean K. Mah, Iain Horrocks, Michela Guglieri, Richard S. Finkel, William B. Martens, James F. Howard, Luca Bello, Taeun Chang, Elena Pegoraro, Kevin M. Flanigan, W. Bryan Burnette, Matthew Wicklund, Maja von der Hagen, Craig M. McDonald, Richard J. Barohn, Giovanni Baranello, Robert C. Griggs, Jeffrey Statland, Michael P. McDermott, Stefan Spinty, Ekkehard Wilichowski, Lorenzo Maggi, Emma Ciafaloni, Ashutosh Kumar, Janbernd Kirschner, Volker Straub, Monika Krzesniak-Swinarska, Craig Campbell, and Basil T. Darras
- Subjects
Male ,Parents ,0301 basic medicine ,Duchenne muscular dystrophy ,Neuromuscular disease ,Psychometrics ,Intraclass correlation ,Patient demographics ,Medizin ,Disease ,03 medical and health sciences ,Health related quality of life ,Psychosocial ,0302 clinical medicine ,Disease severity ,Surveys and Questionnaires ,medicine ,Humans ,Child ,Genetics (clinical) ,business.industry ,medicine.disease ,humanities ,Muscular Dystrophy, Duchenne ,Cross-Sectional Studies ,030104 developmental biology ,Caregivers ,Neurology ,Child, Preschool ,Pediatrics, Perinatology and Child Health ,Quality of Life ,Self Report ,Neurology (clinical) ,business ,030217 neurology & neurosurgery ,Clinical psychology - Abstract
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.
- Published
- 2021