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 < ). 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 No patient had to be withdrawn from medication.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.
Non-randomised studies support RCT evidence in showing improved functional benefit from corticosteroids. These studies suggest sustained benefit for up to 66 months. Adverse effects were common, although generally manageable. According to a large comparative longitudinal study of daily or intermittent (10 days on, 10 days off) corticosteroid for a mean period of four years, a daily regimen prolongs ambulation and improves functional scores over the age of seven, but with a greater frequency of side effects than an intermittent regimen .
• We are aware that trials of other potential new therapies may start during the course of the study. As steroid treatment is part of the normal standard of care in Duchenne we do not believe that being in this study would prevent your boy from being in another study later if there were one that he were eligible for. For example, if your boy is/will be participating in Summit Pharmaceuticals' or Bristol-Myers Suibb's Duchenne trials, he may also be able to participate in FOR-DMD at the same time. Moreover, although we hope that the majority of boys will finish the whole trial, as with any clinical study, you are entitled to withdraw at any time if you no longer wish to participate.