Steroids in Duchenne muscular dystrophy--deflazacort trial

Neuromuscul Disord. 1991;1(4):261-6. doi: 10.1016/0960-8966(91)90099-e.


We conducted a double blind controlled trial in 28 Duchenne muscular dystrophy (DMD) patients with Deflazacort (DF), an oxazoline derivative of prednisolone which reduces its side-effects. Myometric muscle strength measurements, Scott Score and timed tests showed statistically significant improvement for the treated group (P less than 0.05). Side-effects after 9 months of treatment included mild cushingoid appearance in four patients (28%) and moderate in only one (7%), increased appetite in seven (50%), increased body hair in four (28%), irritability and hyperactivity in three (21%). Increased body weight was not prominent and was controlled with dietary measures. No patient had to be withdrawn from medication. More research and long-term follow-up are needed in order to establish the mechanism of improvement and the consequences of long-term steroid administration in DMD. In this regard DF appears as an alternative to prednisone preserving its benefits but with fewer side-effects.

Publication types

  • Clinical Trial
  • Controlled Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anti-Inflammatory Agents / adverse effects
  • Anti-Inflammatory Agents / therapeutic use*
  • Child
  • Child, Preschool
  • Double-Blind Method
  • Humans
  • Muscular Dystrophies / drug therapy*
  • Prednisolone / adverse effects*
  • Prednisolone / antagonists & inhibitors
  • Pregnenediones / adverse effects
  • Pregnenediones / therapeutic use*


  • Anti-Inflammatory Agents
  • Pregnenediones
  • Prednisolone
  • deflazacort