Prednisone in Duchenne Dystrophy

Griggs, Robert C.; Moxley, Richard T.; Mendell, Jerry R.; Fenichel, Gerald M.; Brooke, Michael H.; Pestronk, Alan; Miller, J. Philip

doi:10.1001/archneur.1991.00530160047012

Cited by 249 publications

(49 citation statements)

References 20 publications

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“…In most studies evaluating steroids in DMD, weight gain is an important problem. Studies in which the drug regimen consisted of a daily dose 0.75 mg/ kg per day have reported a weight gain [10% in 75% of the patients [3,4], while we found a comparable weight gain in only 25% of our patients and, more remarkably, only in boys after they got WCB. Since seven of the 11 patients that reached a body weight of ?2 SDS had a body weight of ?1 SD before the start of prednisone, the latter seems to be a risk factor for excessive weight gain.…”

Section: Discussioncontrasting

confidence: 68%

“…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%

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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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Section: Discussioncontrasting

confidence: 68%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

et al. 2009

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“…Griggs and et al 8 reported that boys treated with prednisone were significantly improved in lifting weights at their 6-mo follow-up period. Most of the boys having DMD lost ability to lift weights and raise their hands above their head before the age of 13 yrs.…”

Section: Discussionmentioning

confidence: 98%

“…Of all the therapeutic drugs studied in DMD, only catabolic steroids such as prednisone and deflazacort seem to have potential for improving or preserving functional status. [7][8][9][10][11][12] Nevertheless, there is still variation in perception and practice regarding corticosteroid treatment for DMD.…”

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confidence: 99%

Corticosteroid Treatment and Functional Improvement in Duchenne Muscular Dystrophy

Balaban

Matthews

Clayton

et al. 2005

American Journal of Physical Medicine &Amp; Rehabilitation

161

133

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Intermittent Prednisone Therapy in Duchenne Muscular Dystrophy

Beenakker¹,

Fock²,

Tol³

et al. 2005

Arch Neurol

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Background: Prednisone treatment is used to prolong ambulation in patients with Duchenne muscular dystrophy (DMD). However, since severe adverse effects often accompany prednisone treatment, it is debatable whether the benefits of prednisone treatment outweigh its adverse effects. Objectives: To study the effects of prednisone on muscle function and to determine the extent of steroid-related adverse effects and their influence on the quality of life of ambulant patients with DMD. Design: A randomized, placebo-controlled, crossover trial with 6 months of treatment: prednisone or placebo (0.75 mg/kg daily) during the first 10 days of each month. After a washout period of 2 months, patients received the other regimen for an additional 6 months. Setting: University hospital and rehabilitation center in the Netherlands. Patients: Seventeen ambulant patients with DMD aged 5 to 8 years. Main Outcome Measure: Change in muscle function assessed by timed functional testing: running 9 m, climbing 4 standard-sized stairs, and rising from the floor to a standing position. Results: The increase in time needed to run 9 m (P=.005) and to climb 4 standard-sized stairs (P= .02) was significantly lower during the prednisone period. Conclusions: Prednisone slowed deterioration of muscle function and muscle force in ambulant patients with DMD. Although adverse effects were present, patient quality of life was not affected. Therefore, short-term prednisone treatment can be recommended to preserve motor functions in ambulant patients with DMD.

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Prednisone in Duchenne Dystrophy

Cited by 249 publications

References 20 publications

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

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

Corticosteroid Treatment and Functional Improvement in Duchenne Muscular Dystrophy

Intermittent Prednisone Therapy in Duchenne Muscular Dystrophy

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