Intermittent Prednisone Therapy in Duchenne Muscular Dystrophy

Abstract

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.

Short-term Effects of Corticosteroid Therapy on Cardiac and Skeletal Muscles in Muscular Dystrophies

“…Our results are in agreement with other investigators who recommended the early use of steroid therapy in ambulant patients with DMD. 26 In the studied cases, the lack of clinical signs of heart failure as exertional symptom could be explained by the fact that motor function was already weak and activity was limited. None of them had dyspnea at rest.…”

Short-term Effects of Corticosteroid Therapy on Cardiac and Skeletal Muscles in Muscular Dystrophies

“…Our results are in agreement with other investigators who recommended the early use of steroid therapy in ambulant patients with DMD. 26 In the studied cases, the lack of clinical signs of heart failure as exertional symptom could be explained by the fact that motor function was already weak and activity was limited. None of them had dyspnea at rest.…”

Retrospective cohort study comparing the efficacy of prednisolone and deflazacort in children with muscular dystrophy

“… 6 Various randomized trials have shown to improve muscle function and strength in children treated with prednisolone. 7 8 9 10 11 Deflazacort has shown similar results but with lesser side effects. 12 13 Both deflazacort and prednisolone are equally effective in slowing the progression of disease when given at a dose of 0.9 mg/kg and 0.75 mg/kg, respectively, on daily basis, along with calcium and Vitamin D supplements.…”

Study of Different Modalities of Steroids Use in Children with Duchenne Muscular Dystrophy: An Exploratory Prospective Study

“…Participants reported slightly higher QoL in both groups resulting in signi cant improvement in emotional functioning and total scale at the end of the study. They considered this to be possibly related to the attention given for being incorporated in a clinical trial 32 .…”

Short-term Effects of Corticosteroid Therapy on Cardiac and Skeletal Muscles in Muscular Dystrophies

“…Our results are in agreement with other investigators who recommended the early use of steroid therapy in ambulant patients with DMD. 26 In the studied cases, the lack of clinical signs of heart failure as exertional symptom could be explained by the fact that motor function was already weak and activity was limited. None of them had dyspnea at rest.…”

Retrospective cohort study comparing the efficacy of prednisolone and deflazacort in children with muscular dystrophy

“… 6 Various randomized trials have shown to improve muscle function and strength in children treated with prednisolone. 7 8 9 10 11 Deflazacort has shown similar results but with lesser side effects. 12 13 Both deflazacort and prednisolone are equally effective in slowing the progression of disease when given at a dose of 0.9 mg/kg and 0.75 mg/kg, respectively, on daily basis, along with calcium and Vitamin D supplements.…”

Study of Different Modalities of Steroids Use in Children with Duchenne Muscular Dystrophy: An Exploratory Prospective Study

“…Participants reported slightly higher QoL in both groups resulting in signi cant improvement in emotional functioning and total scale at the end of the study. They considered this to be possibly related to the attention given for being incorporated in a clinical trial 32 .…”

Short-term Effects of Corticosteroid Therapy on Cardiac and Skeletal Muscles in Muscular Dystrophies

“…Our results are in agreement with other investigators who recommended the early use of steroid therapy in ambulant patients with DMD. 26 In the studied cases, the lack of clinical signs of heart failure as exertional symptom could be explained by the fact that motor function was already weak and activity was limited. None of them had dyspnea at rest.…”

Retrospective cohort study comparing the efficacy of prednisolone and deflazacort in children with muscular dystrophy

“… 6 Various randomized trials have shown to improve muscle function and strength in children treated with prednisolone. 7 8 9 10 11 Deflazacort has shown similar results but with lesser side effects. 12 13 Both deflazacort and prednisolone are equally effective in slowing the progression of disease when given at a dose of 0.9 mg/kg and 0.75 mg/kg, respectively, on daily basis, along with calcium and Vitamin D supplements.…”

Study of Different Modalities of Steroids Use in Children with Duchenne Muscular Dystrophy: An Exploratory Prospective Study

“…Participants reported slightly higher QoL in both groups resulting in signi cant improvement in emotional functioning and total scale at the end of the study. They considered this to be possibly related to the attention given for being incorporated in a clinical trial 32 .…”