Glucocorticoid corticosteroids for Duchenne muscular dystrophy

Area of Science:

• Neurology
• Pediatrics
• Pharmacology

Background:

• Duchenne muscular dystrophy (DMD) is a common childhood neuromuscular disorder characterized by progressive muscle wasting and loss of ambulation.
• Prolonging walking ability is a primary therapeutic goal for children diagnosed with DMD.

Purpose of the Study:

• To systematically review the efficacy of glucocorticoid corticosteroids in stabilizing or improving muscle strength and walking ability in boys with DMD.
• To assess short-term and long-term effects and adverse events associated with corticosteroid treatment for DMD.

Main Methods:

• A systematic review and meta-analysis of randomized controlled trials (RCTs) and quasi-randomized trials evaluating glucocorticoids (prednisone, prednisolone, deflazacort) for DMD.
• Searches included major databases (MEDLINE, EMBASE, CINAHL, LILACS) and trial registers, with data extraction and quality assessment performed by two independent reviewers.
• Primary outcome was prolongation of walking; secondary outcomes included muscle strength, functional measures, and adverse events.

Main Results:

• Meta-analysis of four RCTs (249 participants) indicated that glucocorticoids significantly improved muscle strength and function within six months.
• Improvements were observed in measures of mobility, strength, and respiratory function (forced vital capacity).
• One RCT (28 participants) showed stabilization of strength and function for up to two years; the optimal prednisolone dose was 0.75 mg/kg/day daily. Common short-term adverse effects included weight gain and behavioral changes.

Conclusions:

• Randomized controlled studies provide evidence that glucocorticoid therapy improves muscle strength and function in DMD patients in the short term (6 months to 2 years).
• The daily regimen of 0.75 mg/kg/day prednisolone appears most effective, with short-term adverse effects being common but not severe.
• Long-term benefits and risks remain unevaluated by RCTs, though non-randomized studies suggest functional benefits alongside clinically significant long-term adverse effects.