CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy

Escolar, Diana M.; Buyse, Gunnar; Henricson, E.; Leshner, Robert T.; Florence, Julaine; Mayhew, J.; Rocha, Carolina Tesi; Gorni, Ksenija; Pasquali, Livia; Patel, Kantilal M.; McCarter, Robert; Huang, Jennifer; Mayhew, Thomas P.; Bertorini, Tulio E.; Carlo, Jose; Connolly, Anne M.; Clemens, Paula R.; Goemans, Nathalie; Iannaccone, Susan T.; Igarashi, Masanori; Nevo, Yoram; Pestronk, Alan; Subramony, S. H.; Vedanarayanan, Vetta; Wessel, Henry B.

doi:10.1002/ana.20523

“…[3][4][5] Corticosteroid treatment for DMD 6-12 is recommended by an American Academy of Neurology practice parameter.…”

Section: Classification Of Evidencementioning

confidence: 99%

“…[3][4][5] Corticosteroid treatment for DMD [6][7][8][9][10][11][12] is recommended by an American Academy of Neurology practice parameter. 13 Furthermore, a recently published standard of care review emphasized the benefit of corticosteroids for DMD.…”

mentioning

confidence: 99%

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Escolar

¹

,

Hache

²

,

Clemens

³

et al. 2011

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Objective: To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). Methods:A total of 64 boys with DMD who were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months.Results: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone. Conclusions:Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens. Classification of evidence:This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period. Neurology Duchenne muscular dystrophy (DMD) is a progressive muscle disorder due to mutations in the dystrophin gene.1,2 Current treatments can slow disease progression, prolonging ambulation, and improving quality of life and survival. [3][4][5] Corticosteroid treatment for DMD 6-12 is recommended by an American Academy of Neurology practice parameter.13 Furthermore, a recently published standard of care review emphasized the benefit of corticosteroids for DMD.14,15 In a large DMD natural history study currently run by the Cooperative International Neuromuscular Research Group (CINRG), 85% of participants are steroid-treated. 16,17 We hypothesized that weekend prednisone dosing would provide equally effective treatment for DMD as standard daily dosing. Furthermore, corticosteroids might be more widely used in DMD if a dosing regimen had fewer side effects, including less weight gain, less effect on linear Editorial, page 416Podcast Scan this code with your smartphone to access this feature CME Scan this code with your smartphone to access this feature

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“…The other study evaluated creatine to glutamine and placebo in steroid-naïve boys with DMD in a 6-month, double-blinded trial. 4 Supplementation with glutamine is felt to prevent muscle catabolism. This study found no statistically significant effect of either therapy on manual and quantitative muscle strength, although there appeared to be a trend towards less deterioration in all other outcome measures.…”

Section: Supplementsmentioning

confidence: 99%

“…2 Other researchers have reported a good correlation between strength and function in patients with spinal muscular atrophy. 3 Studies have been further confounded due to the question of correlations between age and strength 4 and age and function.…”

Section: Introductionmentioning

confidence: 99%

Therapeutics in Duchenne muscular dystrophy

Strober

¹

2006

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Summary: Duchenne muscular dystrophy (DMD) is a fatal disorder affecting approximately 1 in 3500 live born males, characterized by progressive muscle weakness. Several different strategies are being investigated in developing a cure for this disorder. Until a cure is found, therapeutic and supportive care is essential in preventing complications and improving the afflicted child's quality of life. Currently, corticosteroids are the only class of drug that has been extensively studied in this condition, with controversy existing over the use of these drugs, especially in light of the multiple side effects that may occur. The use of nutritional supplements has expanded in recent years as researchers improve our abilities to use gene and stem cell therapies, which will hopefully lead to a cure soon. This article discusses the importance of therapeutic interventions in children with DMD, the current debate over the use of corticosteroids to treat this disease, the growing use of natural supplements as a new means of treating these boys and provides an update on the current state of gene and stem cell therapies.

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“…2 Other researchers have reported a good correlation between strength and function in patients with spinal muscular atrophy. 3 Studies have been further confounded due to the question of correlations between age and strength 4 and age and function. 1 …”

Section: Introductionmentioning

confidence: 99%

Therapeutics in Duchenne muscular dystrophy

Strober

¹

2006

NeuroRX

View full text Add to dashboard Cite

Summary: Duchenne muscular dystrophy (DMD) is a fatal disorder affecting approximately 1 in 3500 live born males, characterized by progressive muscle weakness. Several different strategies are being investigated in developing a cure for this disorder. Until a cure is found, therapeutic and supportive care is essential in preventing complications and improving the afflicted child's quality of life. Currently, corticosteroids are the only class of drug that has been extensively studied in this condition, with controversy existing over the use of these drugs, especially in light of the multiple side effects that may occur. The use of nutritional supplements has expanded in recent years as researchers improve our abilities to use gene and stem cell therapies, which will hopefully lead to a cure soon. This article discusses the importance of therapeutic interventions in children with DMD, the current debate over the use of corticosteroids to treat this disease, the growing use of natural supplements as a new means of treating these boys and provides an update on the current state of gene and stem cell therapies.

show abstract

CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy

Cited by 95 publications

References 8 publications

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Therapeutics in Duchenne muscular dystrophy

Therapeutics in Duchenne muscular dystrophy

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