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

Lookup NU author(s): Emerita Professor Elaine McCollORCiD, Professor Michela GuglieriORCiD, Professor Volker StraubORCiD


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© 2021 Elsevier B.V.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.

Publication metadata

Author(s): Campbell C, McColl E, McDermott MP, Martens WB, Guglieri M, Griggs RC, Straub V, Childs A-M, Ciafaloni E, Shieh PB, Spinty S, Butterfield RJ, Horrocks I, Roper H, Maggi L, Baranello G, Flanigan KM, Kuntz NL, Manzur AY, Darras BT, Kang P, Mah JK, Mongini T, Ricci F, Morrison L, Krzesniak-Swinarska M, von der Hagen M, Finkel RS, Kumar A, Wicklund M, McDonald CM, Henricson EK, Schara-Schmidt U, Wilichowski E, Barohn RJ, Statland J, Kirschner J, Vita G, Vita GL, Howard JF, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T

Publication type: Article

Publication status: Published

Journal: Neuromuscular Disorders

Year: 2021

Volume: 31

Issue: 11

Pages: 1161-1168

Online publication date: 10/06/2021

Acceptance date: 02/06/2021

ISSN (print): 0960-8966

ISSN (electronic): 1873-2364

Publisher: Elsevier Ltd


DOI: 10.1016/j.nmd.2021.06.001


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