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Prednisone and Deflazacort in Duchenne Muscular Dystrophy: Do They Play a Different Role in Child Behavior and
Susan Sienko1, Cathleen Buckon1, Eileen Fowler2
1Clinical Research, Shriners Hospitals for Children, Portland, Oregon, USA.
Insights
This study found no overall increase in behavioral issues for boys with Duchenne Muscular Dystrophy using prednisone or deflazacort. However, specific behavioral differences emerged between the two steroid treatments, impacting quality of life.
Area of Science:
- Pediatric Neurology
- Child Psychology
- Musculoskeletal Disorders
Background:
- Duchenne Muscular Dystrophy (DMD) is a progressive genetic disorder affecting muscle function.
- Corticosteroids like prednisone and deflazacort are commonly used to manage DMD, but their impact on behavior and quality of life is not fully understood.
- Ambulant boys with DMD often experience behavioral and psychosocial challenges.
Purpose of the Study:
- To compare the effects of prednisone and deflazacort on child behavior and psychosocial quality of life in ambulant boys with DMD.
- To assess behavioral differences between steroid-naïve boys and those on prednisone or deflazacort.
- To identify factors influencing psychosocial quality of life in boys with DMD.
Main Methods:
- Prospective natural-history study involving 67 ambulant boys with DMD.
- Parents completed the Child Behavior Checklist (CBCL) for behavioral assessment.
- Parents and boys completed the PedsQL™4.0 for quality of life evaluation.
- Participants were categorized into prednisone, deflazacort, or steroid-naïve groups.
Main Results:
- Boys with DMD exhibited higher rates of general behavioral problems compared to age-matched peers.
- No significant differences in overall CBCL syndrome scales were found between treatment groups.
- Boys on deflazacort showed more withdrawn behaviors; boys on prednisone showed more aggressive behaviors.
- Age and behavioral issues significantly predicted psychosocial quality of life variance.
Conclusions:
- Steroid use (prednisone or deflazacort) was not associated with increased overall behavioral problems in boys with DMD.
- Specific behavioral patterns differed between prednisone and deflazacort groups.
- Behavioral assessment and management are critical for improving psychosocial quality of life in boys with DMD.
Abstract:
The aim of this study was to determine whether prednisone and deflazacort play a different role in child behavior and perceived health related psychosocial quality of life in ambulant boys with Duchenne Muscular Dystrophy. As part of a prospective natural-history study, parents of sixty-seven ambulant boys with DMD (27 taking prednisone, 15 taking deflazacort, 25 were steroid naïve) completed the Child Behavior Checklist (CBCL) for assessment of behavioral, emotional and social problems and both parents and boys with DMD completed the PedsQL™4.0 generic core scale short form. Boys with DMD had higher rates of general behavioral problems than age-matched peers. No significant differences were found among the groups for any of the CBCL syndrome scales raw scores, including internalizing and externalizing behaviors; however, on average boys taking deflazacort demonstrated more withdrawn behaviors than those taking prednisone, while on average the boys taking prednisone demonstrated more aggressive behaviors than boys taking deflazacort. Age, internalizing and externalizing behaviors accounted for 39 and 48% of the variance in psychosocial quality of life for both parents and boys with DMD, respectively. Overall, the use of steroids was not associated with more behavioral problems in boys with DMD. As behavior played a significant role in psychosocial quality of life, comprehensive assessment and treatment of behavioral problems is crucial in this population.
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