Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME): A randomized, controlled trial. Issue 3 (27th January 2016)

• Record Type: Journal Article
• Authors: Benatar, Michael; Mcdermott, Michael P.; Sanders, Donald B.; Wolfe, Gil I.; Barohn, Richard J.; Nowak, Richard J.; Hehir, Michael; Juel, Vern; Katzberg, Hans; Tawil, Rabi
• Is Part Of: Muscle & nerve. Volume 53:Issue 3(2016)
• Page Start: 363
• Page End: 369
• Publication Date: 2016-01-27
• Subjects: clinical trial -- ocular myasthenia -- prednisone -- neuromuscular -- steroids; Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.24769
• Languages: English
• ISSNs: 0148-639X

Abstract

ABSTRACT: 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. Muscle Nerve 53: 363–369, 2016