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


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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.

Original languageEnglish (US)
Pages (from-to)363-369
Number of pages7
JournalMuscle and Nerve
Issue number3
StatePublished - Mar 1 2016


  • Clinical trial
  • Neuromuscular
  • Ocular myasthenia
  • Prednisone
  • Steroids

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)


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