A Comparison of Daily and Alternate-Day Prednisone Therapy in the Treatment of Duchenne Muscular Dystrophy

Fenichel, Gerald M.; Mendell, Jerry R.; Moxley, Richard T.; Griggs, Robert C.; Brooke, Michael H.; Miller, J. Phillip; Pestronk, Alan; Robison, Justin; King, Wendy; Signore, L.; Pandya, Shree; Florence, Julaine; Schierbecker, Janine; Wilson, Brad

doi:10.1001/archneur.1991.00530180027012

Cited by 106 publications

(90 citation statements)

References 13 publications

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“…A structured, proactive approach to respiratory management that includes use of assisted cough and nocturnal ventilation has been shown to prolong survival. [64][65][66] Patients with DMD are at risk of respiratory complications as their condition deteriorates due to progressive loss of respiratory muscle strength. These complications include ineffective cough, [67][68][69][70][71][72][73][74][75] nocturnal hypoventilation, sleep disordered breathing, and ultimately daytime respiratory failure.…”

Section: Respiratory Managementmentioning

confidence: 99%

“…Daily administration was more effective than treatment on alternate days. 66 Prednisolone is often used in Europe instead of prednisone. Deflazacort, a similar glucocorticoid available in many countries, but not currently approved Review for use by the US Food and Drug Administration or the CDC in the USA, has been shown to have a similar efficacy at a daily dose of 0·9 mg/kg and has a slightly different chronic risk profi le.…”

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confidence: 99%

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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

Bushby

Finkel

Birnkrant

et al. 2010

The Lancet Neurology

1,655

1,596

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Section: Respiratory Managementmentioning

confidence: 99%

mentioning

confidence: 99%

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

Bushby

Finkel

Birnkrant

et al. 2010

The Lancet Neurology

1,655

1,596

View full text Add to dashboard Cite

“…3). Most randomized studies have evaluated corticosteroid treatment using functional scores, with a surprisingly large variation in outcome depending whether a metric or non-metric measure was chosen; for example, time to run has been evaluated in 10 [7,8], 9 (30 feet) [4,5,9,10,14,24] or 8.54 m (28 feet) [11,25].…”

Section: Discussionmentioning

confidence: 99%

“…In 2005, guidelines were published in the Netherlands advocating that DMD patients should be treated with 0.75 mg/kg prednisone daily for at least the ambulant years [2]. This conclusion was based on trials using daily doses of corticosteroids, which proved to be superior to placebo [3,4] and to prednisone on an alternate-day scheme (1 day on/ 1 day off) [5]. Different doses as well as different intermittent schemes of administration have been tried in clinical trials with the aim of reducing side effects [6][7][8][9][10].…”

Section: Introductionmentioning

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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“…Clinical studies of these regimens suggest that side effects may be reduced, but efficacy compared with daily prednisone is unclear at best. [11][12][13] Deflazacort is a related corticosteroid not approved in the United States but available in Canada and many European countries. Deflazacort has been shown to prolong functional abilities with fewer side effects than prednisone.…”

Section: Neuromuscular Concernsmentioning

confidence: 99%

Approaching a New Age in Duchenne Muscular Dystrophy Treatment

Wagner

2008

Neurotherapeutics

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Summary:Duchenne muscular dystrophy is the most common and severe form of muscular dystrophy. The cornerstones of current treatment include corticosteroids for skeletal muscle weakness, afterload reduction for cardiomyopathy, and noninvasive ventilation for respiratory failure. With these interventions, patients are walking and living longer. However, the current status is still far from adequate. Increased private and federal funding of studies in Duchenne muscular dystrophy has led to a large number of novel agents with propitious therapeutic potential. These include agents that modify dystrophin expression, increase muscle growth and regeneration, and modulate inflammatory responses. Many of these agents are already in clinical trials. Challenges to the development of additional novel therapeutics exist, including lack of validated animal models and lack of adequate biomarkers as surrogate endpoints. However, these challenges are not insurmountable and the next decade will likely see meaningful, new treatment options introduced into the clinical care of patients with Duchenne muscular dystrophy.

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A Comparison of Daily and Alternate-Day Prednisone Therapy in the Treatment of Duchenne Muscular Dystrophy

Cited by 106 publications

References 13 publications

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

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

Approaching a New Age in Duchenne Muscular Dystrophy Treatment

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