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.

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

Tiziana Mongini
Membro del Collaboration Group
;
Federica Ricci
Membro del Collaboration Group
;
2021-01-01

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.
2021
1
8
Duchenne muscular dystrophy; Health related quality of life; Psychosocial.
Craig Campbell a, Elaine McColl, Michael P. McDermott, William B. Martens, Michela Guglieri, Robert C. Griggs, The Muscle Study Group, and TREAT-NMD (Volker Straub, Anne-Marie Childs, Emma Ciafaloni, Perry B Shieh, Stefan Spinty, Russell J Butterfield, Iain Horrocks, Helen Roper, Lorenzo Maggi, Giovanni Baranello, Kevin M Flanigan, Nancy L Kuntz, Adnan Y Manzur, Basil T Darras, Peter Kang, Jean K Mah, Tiziana Mongini, Federica Ricci, Leslie Morrison, Monika Krzesniak-Swinarska, Maja von der Hagen, Richard S Finkel, Ashutosh Kumar, Matthew Wicklund, Craig M McDonald, Erik K Henricson, Ulrike Schara-Schmidt, Ekkehard Wilichowski, Richard J Barohn, Jeffrey Statland, Janbernd Kirschner, Giuseppe Vita, Gian Luca Vita, James F Howard Jr, Imelda Hughes, Hugh J McMillan, Elena Pegoraro, Luca Bello, W Bryan Burnette, Mathula Thangarajh, Taeun Chang.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2318/1806661
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