Searching for the optimal corticosteroid therapy in DMD

Although long-term corticosteroid therapy is a commonly accepted standard of care in Duchenne muscular dystrophy (DMD), its modalities are still debated. An essai international multicentrique attempted to answer this question by comparing several types of corticosteroids in a population of 196 children.

  • Participants were randomly assigned to one of three groups: daily prednisone (0.75 mg/kg/day), daily deflazacort (0.9 mg/kg/day) and an alternating regimen (intermittent prednisone 0.75 mg/kg for 10 days followed by a 10-day treatment window, and so on);
  • The two continuous daily corticosteroid therapies were the most effective in terms of motor function, no drug, prednisone or deflazacort, was standing out;
  • Changes in the other two endpoints (respiratory function and satisfaction index) were not statistically significant;

The long-awaited results of the trial provide some interesting insights but few definitive conclusions.


Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. Guglieri M, Bushby K, McDermott MP et al. JAMA. 2022 Apr 19;327(15):1456-1468. B_Référence