Prednisone Treatment in Duchenne Muscular Dystrophy

DeSilva, Shari

doi:10.1001/archneur.1987.00520200022012

Cited by 130 publications

(80 citation statements)

References 20 publications

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“…Five studies involved participation of Ն14 patients whose ages ranged from 3 to 15 years. [33][34][35][36]40 The duration of treatment varied from 6 months to 11 years.…”

Section: Description Of Processmentioning

confidence: 99%

“…Four studies have examined various combinations of daily, alternate-day, or cyclical prednisone treatment (see table E-3 on the Neurology Web site). 33,34,36,40 Two class IV studies (n ϭ 30; 2 mg/kg/day for 2 months followed by alternate-day prednisone at twothirds the original daily dose) have demonstrated that alternate-day prednisone prolongs ambulation. 33,34 In the 12 patients who received long-term treatment, ambulation was prolonged for Ͼ2 years.…”

Section: Description Of Processmentioning

confidence: 99%

“…33,34,36,40 Two class IV studies (n ϭ 30; 2 mg/kg/day for 2 months followed by alternate-day prednisone at twothirds the original daily dose) have demonstrated that alternate-day prednisone prolongs ambulation. 33,34 In the 12 patients who received long-term treatment, ambulation was prolonged for Ͼ2 years. 34 A study of 32 patients, age 6 to 14 years, found that intermittent cyclical daily prednisone (0.75 mg/kg/ day for 10 days each month) had no long-term benefit.…”

Section: Description Of Processmentioning

confidence: 99%

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Practice Parameter: Corticosteroid treatment of Duchenne dystrophy [RETIRED]

Moxley¹,

Ashwal²,

Pandya³

et al. 2005

Neurology

279

183

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Abstract-Background:The Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society develop practice parameters as strategies for patient management based on analysis of evidence. Objective: To review available evidence on corticosteroid treatment of boys with Duchenne dystrophy. Methods: Relevant literature was reviewed, abstracted, and classified. Recommendations were based on a four-tiered scheme of evidence classification, and areas for future research are defined. Results: Seven class I studies and numerous less rigorous trials all demonstrated that corticosteroid treatment for 6 months with prednisone (0.75 or 1.5 mg/kg/day) increased muscle strength, performance, and pulmonary function and significantly slowed the progression of weakness. Two class I trials examined the effect of lower dosage of prednisone (0.30 and 0.35 mg/kg/day), demonstrated lesser but similar benefits, and showed a lower frequency of side effects (e.g., weight gain). The only significant side effects in all class I trials were weight gain and development of a cushingoid facial appearance. One longer-term trial of daily prednisone (0.3 to 0.7 mg/kg/day), a class III study, showed prolongation of functional ability and slower progression of weakness in patients during 3 years of treatment. One class IV, open trial of alternate-day prednisone (2 mg/kg for 2 months, then two-thirds dose every other day) extended ambulation by approximately 2 years in treated compared with untreated patients. Deflazacort, a corticosteroid similar in structure to prednisone, produced similar improvement in muscle strength and function with a similar side effect profile. Conclusions: Prednisone has been demonstrated to have a beneficial effect on muscle strength and function in boys with Duchenne dystrophy and should be offered (at a dose of 0.75 mg/kg/day) as treatment. If side effects require a decrease in prednisone, tapering to dosages as low as 0.3 mg/kg/day gives less robust but significant improvement. Deflazacort (0.9 mg/kg/day) can also be used for the treatment of Duchenne dystrophy in countries in which it is available. Benefits and side effects of corticosteroid therapy need to be monitored. The offer of treatment with corticosteroids should include a balanced discussion of potential risks. NEUROLOGY 2005;64:13-20 Duchenne dystrophy (DD), an X-linked, recessive disorder, with onset before age 5 years, is the most common and severe form of childhood muscular dystrophy.1-3 The specific molecular defect is an absence or marked deficiency of dystrophin, a large membrane-associated protein that is part of the dystrophin-glycoprotein complex.1 Affected boys develop neck flexor, anterior abdominal, hip, and shoulder girdle muscle weakness in early childhood, with loss of ambulation between ages 7 and 12. 4,5 Additional material related to this article can be found on the Neurology Web site. Go to www.neurology.org and scroll down the Death usually occurs in the 20s, with the chance of...

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“…Five studies involved participation of Ն14 patients whose ages ranged from 3 to 15 years. [33][34][35][36]40 The duration of treatment varied from 6 months to 11 years.…”

Section: Description Of Processmentioning

confidence: 99%

Section: Description Of Processmentioning

confidence: 99%

Section: Description Of Processmentioning

confidence: 99%

See 1 more Smart Citation

Practice Parameter: Corticosteroid treatment of Duchenne dystrophy [RETIRED]

Moxley¹,

Ashwal²,

Pandya³

et al. 2005

Neurology

279

183

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“…Comparable studies in which daily doses of steroids were used have reported a mean age of 11.5 [14] and 12.5 years [15], and those in which alternate-day high-dose prednisone (1.3-2.0 mg/kg) were used have reported 14 [16] or 12.2 years [17]. However, a drop-out percentage of 55% was reported during daily prednisone 0.75 mg/kg per day due to obesity or infection-related complications [16].…”

Section: Discussionmentioning

confidence: 99%

Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy

et al. 2009

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Corticosteroids are effective in improving motor function in Duchenne muscular dystrophy (DMD) patients within 6 months-2 years of treatment initiation, but there is as yet no consensus on which treatment scheme is the best. We retrospectively analyzed data of 35 DMD patients who were treated with prednisone 0.75 mg/kg per day intermittently 10 days on/10 days off. Prednisone was started during the ambulant phase at age 3.5-9.7 years (median 6.5 years). The median period of treatment was 27 months (range 3-123 months). The median age at which ambulation was lost was 10.8 years (mean 10.9 years; 95% confidence interval 10.0-11.8 years). Nine patients (26%) had excessive weight gain. Eight boys (21%) had a bone fracture, which was when four of these eight children lost the ability to walk. Treatment was stopped in two obese patients, two hyperactive boys and one patient following a fracture. Our data suggest that prednisone 10 on/10 off has relatively few side effects and extends the ambulant phase by 1 year compared to historical controls.

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“…19,20 Drachman's group repeated their protocol following 16 boys for one or more years, during which time ½ of this group became wheel chair bound and another quarter quickly became chair dependent shortly after discontinuation. 17 Brooke's study used a dose of 1.5mg/kg daily for 6 months in 33 boys, 6 of whom were wheelchair bound and could not perform the time function studies.…”

Section: Corticosteroidsmentioning

confidence: 99%

Therapeutics in Duchenne muscular dystrophy

Strober

2006

NeuroRX

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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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Prednisone Treatment in Duchenne Muscular Dystrophy

Cited by 130 publications

References 20 publications

Practice Parameter: Corticosteroid treatment of Duchenne dystrophy [RETIRED]

Practice Parameter: Corticosteroid treatment of Duchenne dystrophy [RETIRED]

Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy

Therapeutics in Duchenne muscular dystrophy

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