Corticosteroids for the Treatment of Duchenne Muscular Dystrophy

The review concluded that corticosteroids, particularly prednisone at 0.75 mg/kg/day, significantly improved muscle strength and function in boys with Duchenne muscular dystrophy (DMD) in the short term (up to 12 months) and strength up to two years. However, there was no strong evidence from randomized controlled trials (RCTs) to confirm the prolongation of walking ability. Adverse effects such as weight gain, behavioral changes, and cushingoid appearance were more common with corticosteroids than placebo but were generally manageable. A weekend-only prednisone regimen was found to be as effective as daily prednisone in the short term, with no clear difference in body mass index (BMI). Deflazacort appeared to cause less weight gain than prednisone after one year, based on very low-quality evidence. Long-term benefits and risks of corticosteroid treatment remain unclear due to insufficient data from RCTs, though non-randomized studies suggested sustained benefits and significant adverse effects over longer periods.