Treatment of Duchenne muscular dystrophy with cyclosporin A – A randomized, double-blind, placebo controlled trial

Treatment with steroids has shown to slow down progression of muscle weakness in patients with Duchenne muscular dystrophy (DMD) but its use is limited by side effects. In a randomized, multi-centre, double-blind placebo-controlled trial we investigated the effect of cyclosporin A (CSA) in DMD. 153 patients were randomized to receive either placebo or 4mg/kg CSA. After three months both groups received additional treatment with intermittent prednisone (0.75mg/kg, 10 days on/10 days off) for another 12 months. In each group 73 patients were available for intention to treat analysis. Baseline characteristics were comparable in both groups. There was no significant difference between the two groups concerning primary (manual muscle strength according to MRC) and secondary (myometry, loss of ambulation, side effects) outcome measures. Peak CSA values were measured blindly and ranged from 12–658ng/ml (mean 210ng/ml) in the verum group. In conclusion, CSA does not improve muscle strength as a monotherapy and does not improve the efficacy of intermittent prednisone in DMD. Networks of rare diseases such as the BMBF-funded MD-NET facilitate the conduction of meaningful multicentre trials even in rare diseases.

The study was supported by BMBF, aktion bennie und co e.V., Novartis Pharma, and Deutsche Gesellschaft für Muskelkranke e.V. Participating trial sites were Basel, Berlin, Essen, Freiburg, Göttingen, Kiel, Mainz, Munich, Neuss, and Vienna.