Glucocorticoid Treatment for the Prevention of Scoliosis in Children with Duchenne Muscular Dystrophy: Long-Term Follow-up

Lebel, David E; Corston, John A; McAdam, Laura; Biggar, W. Douglas; Alman, Benjamin A.

doi:10.2106/jbjs.l.01577

Cited by 120 publications

(99 citation statements)

References 20 publications

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“…There is evidence that using steroids in patients with DMD can have numerous positive effects including prolonged independent ambulation, decreased progression of scoliosis, increased muscle strength, respiratory function, cardiac function and quality of life (18,(21)(22)(23)(24)(25)(26). The use of steroid therapy has been associated with a reduction in the incidence of scoliosis in DMD because prolonging ambulation means that some boys will undergo their pubertal growth spurt before loss of ambulation.…”

Section: Interventions Before Surgerymentioning

confidence: 99%

“…The use of steroid therapy has been associated with a reduction in the incidence of scoliosis in DMD because prolonging ambulation means that some boys will undergo their pubertal growth spurt before loss of ambulation. There is also evidence that suggests patients treated with long-term steroids may avoid developing a scoliosis (24).…”

Section: Interventions Before Surgerymentioning

confidence: 99%

“…Other potential complications include the development of cataracts (24,26), the risk of spine and long bone fractures (22,25), and a decreased bone density (25). With regards to cataracts, both articles, while reporting differing incidence rates of cataracts, noted that those that required surgical correction for this was minimal (24,26).…”

Section: Interventions Before Surgerymentioning

confidence: 99%

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Duchenne muscular dystrophy: the management of scoliosis

et al. 2016

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This study summaries the current management of scoliosis in patients with Duchenne Muscular Dystrophy.A literature review of Medline was performed and the collected articles critically appraised. This literature is discussed to give an overview of the current management of scoliosis within Duchenne Muscular Dystrophy.Importantly, improvements in respiratory care, the use of steroids and improving surgical techniques have allowed patients to maintain quality of life and improved life expectancy in this patient group.

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Section: Interventions Before Surgerymentioning

confidence: 99%

Section: Interventions Before Surgerymentioning

confidence: 99%

Section: Interventions Before Surgerymentioning

confidence: 99%

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Duchenne muscular dystrophy: the management of scoliosis

et al. 2016

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“…Comparisons between the natural history studies in the pre-glucocorticoid era and those after glucocorticoid therapy have demonstrated benefits in the motor function, giving longer independent gait, better core stabilization and upper limb function, prevention of spine deformities, and delaying the settlement of lower limb deformities [32][33][34][35][36][37][38][39] . The use of glucocorticoids is also responsible for nonmotor benefits, particularly in preserving respiratory function, preventing cardiomyopathy, improving quality of life parameters and prolonging life itself 38,40,41 .…”

Section: Are Glucocorticoids Recommended For Dmd Patients?mentioning

confidence: 99%

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

Araujo

Carvalho

Cavalcanti³

et al. 2017

Arq. Neuro-Psiquiatr.

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Significant advances in the understanding and management of Duchenne muscular dystrophy (DMD) took place since international guidelines were published in 2010. Our objective was to provide an evidence-based national consensus statement for multidisciplinary care of DMD in Brazil. A combination of the Delphi technique with a systematic review of studies from 2010 to 2016 was employed to classify evidence levels and grade of recommendations. Our recommendations were divided in two parts. We present Part 1 here, where we describe the guideline methodology and overall disease concepts, and also provide recommendations on diagnosis, steroid therapy and new drug treatment perspectives for DMD. The main recommendations: 1) genetic testing in diagnostic suspicious cases should be the first line for diagnostic confirmation; 2) patients diagnosed with DMD should have steroids prescribed; 3) lack of published results for phase 3 clinical trials hinders, for now, the recommendation to use exon skipping or read-through agents.Keywords: muscular dystrophy, Duchenne; practice guideline; consensus; diagnosis; genetic testing; drug therapy; glucocorticoids; utrophin. RESUMOAvanços na compreensão e no manejo da distrofia muscular de Duchenne (DMD) ocorreram desde a publicação de diretrizes internacionais em 2010. Nosso objetivo foi elaborar um consenso nacional baseado em evidências de cuidado multidisciplinar dos pacientes com DMD no Brasil. Utilizamos a técnica de Delphi combinada com revisão sistemática da literatura de 2010 a 2016 classificando níveis de evidência e graus de recomendação. Nossas recomendações foram divididas em duas partes. Apresentamos aqui a parte 1, descrevendo a metodologia utilizada e conceitos gerais da doença, e fornecemos recomendações sobre diagnóstico, tratamento com corticosteroides e novas perspectivas de tratamentos medicamentosos. As principais recomendações: 1) testes genéticos deveriam ser a primeira linha para confirmação de casos suspeitos; 2) pacientes com diagnóstico de DMD devem receber corticosteroides; 3) por enquanto, a falta de publicações de resultados dos ensaios clínicos de fase 3, dificulta recomendações de uso medicamentos que "saltam exons" ou "passam" por código de parada prematura.

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“…Il existe deux protocoles principaux : l'un utilisant le deflazacort (Calcort ® ) à une posologie de 0,9 mg/kg/jour (0,8 mg/kg à partir de 10 ans, 0,55 mg/kg à partir de 15 ans et 0,5 mg/kg au-delà de 18 ans) [3] ; l'autre utilisant la prednisone (Cortancyl ® ) : 0,75 mg/kg/jour pendant 6 mois à 1 an, puis 0,75 mg/kg un jour sur 2 pendant 1 à 2 ans [4]. Les effets primaires de la corticothérapie sont multiples : recul de l'âge de perte de la marche de 12 à 14,5 ans [5] ; amélioration de la fonction ventilatoire [6] ; âge d'apparition de la cardiomyopathie différé (15,2 ans versus 13,1 ans) [7] ; diminution du risque de scoliose (20 % vs 92 %) [8] ; réduction du taux de mortalité [9]. Les effets secondaires observés sont ceux d'une corticothé-rapie classique : fractures osseuses et tassements vertébraux, surpoids, retard statural, cataracte (infraclinique), troubles du comportement, hyperphagie, syndrome cushingoïde, hirsutisme, troubles gastriques [3].…”

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Intérêt des traitements pharmacologiques symptomatiques des maladies neuromusculaires de l’enfant

Cuisset

Hamain²,

Binoche

et al. 2017

Cah. Myol.

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Les traitements pharmacologiques symptomatiques représentent un appoint utile dans la prise en charge des maladies neuromusculaires, qu'ils soient ciblés sur un processus physiopathologique corolaire de la maladie (fibrose musculaire, oxydation), sur un symptôme physique tel que la myotonie, la douleur, ou sur les troubles cognitifs liés à l'atteinte centrale associée (dystrophinopathies, dystrophie myotonique de Steinert). Ils permettent d'améliorer l'espérance et la qualité de vie, en attendant les progrès des thérapies curatives émergentes.La prise en charge thérapeutique des maladies neuromusculaires (MNM) de l'enfant est essentiellement symptomatique et multidisciplinaire, associant les aspects orthopédique, respiratoire, nutritionnel, digestif, cardiaque et la gestion de la douleur. Dans l'attente de la pleine efficience des thérapies curatives innovantes, susceptibles d'aboutir à une méde-cine moléculaire ciblée sur le gène, voire une méde-cine personnalisée spécifique de la mutation génique ou d'autres caractéristiques liées au patient (terrain génétique, pharmacocinétique, physiopathologie...), il paraît donc bénéfique de prescrire, de façon optimisée, pour une maladie neuromusculaire donnée, tous les traitements pharmacologiques symptomatiques possibles. Une telle démarche appliquée dès la fin des années 1980 aux patients atteints de dystrophie musculaire de Duchenne (DMD), a permis de doubler leur espérance de vie en 20 ans. Les suppléments vitaminiques ou nutritionnels, les traitements symptomatiques concernant une complication ciblée sur un seul appareil (par exemple les troubles du transit intestinal) ne seront pas traités ici. PRISE EN CHARGE Cah. Myol. 2017 ; 15 : 30-33

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Glucocorticoid Treatment for the Prevention of Scoliosis in Children with Duchenne Muscular Dystrophy: Long-Term Follow-up

Abstract: The long-term use of the glucocorticoid results in a substantial decreased need for spinal surgery to treat scoliosis.

Cited by 120 publications

References 20 publications

Duchenne muscular dystrophy: the management of scoliosis

Duchenne muscular dystrophy: the management of scoliosis

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

Intérêt des traitements pharmacologiques symptomatiques des maladies neuromusculaires de l’enfant

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