AANEM News Express

AANEM News Express

Efficacy and Safety of Deflazacort vs Prednisone and Placebo for Duchenne Muscular Dystrophy

Griggs R;  Miller JP;  Greenberg C; Fehlings D; Pestronk A et al. Efficacy and Safety of Deflazacort vs Prednisone and Placebo for Duchenne Muscular Dystrophy. Neurology 87, November 15, 2016.

Submitted by Leigh Maria K. Ramos-Platt, MD, News Science Editorial Board

Griggs  and colleagues presented the data from a study that was completed in 1995. This was a phase III double blind placebo controlled study in 2 phases over the course of 52 weeks and included 196 patients diagnosed with a dystrophinopathy either by genetic testing or muscle biopsy. This multicenter trial initially had 4 groups (to include a placebo group) in the first phase of 12 weeks, then 3 treatment groups (1.2 mg/kg/day daily deflazacort, 0.9 mg/kg/day daily deflazacort, and 0.75 mg/kg/day of daily prednisone) to complete 52 weeks. Patients were between 5-15 years old and could be either ambulatory or nonambulatory.

The purpose of this study was to evaluate the safety and efficacy of deflazacort vs. steroids. The primary endpoint of this study was change from baseline of muscle strength based on the Medical Research Council scale. The results demonstrated that in the first 12 weeks, compared to placebo, all three treatment arms demonstrated increased average muscle strength. The interval from 12-52 weeks demonstrated a greater preservation of muscle strength of the high and low dose deflazacort arms compared to the prednisone arm. Overall, at week 52, there was greater preservation of muscle strength of the high and low dose deflazacort arms  compared to the prednisone arm . The side effect profile was also more favorable with the deflazacort arms. Weight gain was 18% of their body weight in the prednisone arm vs 5% in the deflazacort arms. Behavioral changes were more common in the prednisone arm but Cushingoid appearance, short stature, and cataracts were more common in the deflazacort arms.

Overall, this study does support deflazacort having better efficacy after 52 weeks compared to prednisone. There was no significant efficacy difference between the high and low doses of deflazacort. Thus, the authors concluded that steroids are better than placebo in preservation of motor function and that deflazacort had lower incidence of weight gain and psychiatric side effects.

Comment: Deflazacort, although already widely used in other countries, was recently approved by the FDA specifically for the treatment of boys with Duchene muscular dystrophy (DMD). While this study does demonstrate better efficacy and lower side effects of deflazacort vs prednisone, the data is 20 years old. Endpoints that are used in current trials were not employed with this study (such as the 6-minute walk distance) which the authors did acknowledge. Considering the cost of the drug, physicians need to give careful consideration of data in this article. Another study (for DMD) will have more current information regarding the efficacy of steroid regimes in DMD.
About the AANEM News Science Editorial Board
The board helps to highlight significant, timely science news items for AANEM members. It reviews articles in journals and websites, identifies newsworthy items in the field, and writes article summaries.

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