Corticosteroids for the treatment of Duchenne muscular dystrophy

Abstract

Moderate quality evidence from RCTs indicates that corticosteroid therapy in DMD improves muscle strength and function in the short term (twelve months), and strength up to two years. On the basis of the evidence available for strength and function outcomes, our confidence in the effect estimate for the efficacy of a 0.75 mg/kg/day dose of prednisone or above is fairly secure. There is no evidence other than from non-randomised trials to establish the effect of corticosteroids on prolongation of walking. In the short term, adverse effects were significantly more common with corticosteroids than placebo, but not clinically severe. A weekend-only prednisone regimen is as effective as daily prednisone in the short term (12 months), according to low to moderate quality evidence from a single trial, with no clear difference in BMI (low quality evidence). Very low quality evidence indicates that deflazacort causes less weight gain than prednisone after a year's treatment. We cannot evaluate long-term benefits and hazards of corticosteroid treatment or intermittent regimens from published RCTs. Non-randomised studies support the conclusions of functional benefits, but also identify clinically significant adverse effects of long-term treatment, and a possible divergence of efficacy in daily and weekend-only regimens in the longer term. These benefits and adverse effects have implications for future research and clinical practice.

Intermittent glucocorticoid steroid dosing enhances muscle repair without eliciting muscle atrophy

“…Although the side effect profile from GC steroids is extensive, muscle weakness and myopathy can occur in response to chronic GC steroid use and can be extremely debilitating (1). Curiously, chronic GC steroids are used to treat Duchenne muscular dystrophy (DMD), a primary muscle disease, and the use of steroids is associated with prolonged ambulation (2,3).…”

Bone Density in the Obese Child: Clinical Considerations and Diagnostic Challenges

“…The added consequences of corticosteroid treatment are less clear. There is good evidence that corticosteroids can prolong the period of independent ambulation with associated improvements in muscle strength and function[66]. However, the detrimental effects of long-term corticosteroids are cumulative; with greater increases over time being reported in central adiposity, reduced stature, pubertal delay and bone fragility [67, 68].…”

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

“…All patients diagnosed with DMD should have glucocorticoids prescribed (Level of evidence: 1A, Class of Recommendation: A) 32 . Comparisons between the natural history studies in the pre-glucocorticoid era and those after glucocorticoid therapy have demonstrated benefits in the motor function, giving longer independent gait, better core stabilization and upper limb function, prevention of spine deformities, and delaying the settlement of lower limb deformities [32][33][34][35][36][37][38][39] .…”

“…Comparisons between the natural history studies in the pre-glucocorticoid era and those after glucocorticoid therapy have demonstrated benefits in the motor function, giving longer independent gait, better core stabilization and upper limb function, prevention of spine deformities, and delaying the settlement of lower limb deformities [32][33][34][35][36][37][38][39] . The use of glucocorticoids is also responsible for nonmotor benefits, particularly in preserving respiratory function, preventing cardiomyopathy, improving quality of life parameters and prolonging life itself 38,40,41 .…”