Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME): A randomized, controlled trial.

INTRODUCTION: In this study we evaluated the safety, tolerability, and efficacy of prednisone in patients with ocular myasthenia gravis (OMG) concurrently treated with pyridostigmine. METHODS: This investigation was a randomized, double-blind, placebo-controlled trial. Participants whose symptoms failed to remit on pyridostigmine were randomized to receive placebo or prednisone, initiated at 10 mg every other day, and titrated to a maximum of 40 mg/day over 16 weeks. The primary outcome measure was treatment failure. RESULTS: Fewer subjects were randomized than the 88 planned. Of the 11 randomized, 9 completed 16 weeks of double-blind therapy. Treatment failure incidence was 100% (95% CI 48%-100%) in the placebo group (n = 5) vs. 17% (95% CI 0%-64%) in the prednisone group, P = 0.02 (n = 6). Median time to sustained minimal manifestation status (MMS) was 14 weeks, requiring an average prednisone dose of 15 mg/day. Adverse events were infrequent and generally mild in both groups. CONCLUSIONS: A strategy of low-dose prednisone with gradual escalation appears to be safe, well-tolerated, and effective in treating OMG.

Duke Scholars

Author Donald Benjamin Sanders Neurology, Neuromuscular Disease

Author Vern Charles Juel Neurology, Neuromuscular Disease

Author Jeffrey Guptill Neurology, Neuromuscular Disease

Author Janice Munn Massey Neurology, Neuromuscular Disease