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

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 sy...

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Published in:	Muscle & nerve Vol. 53; no. 3; pp. 363 - 369
Main 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
Format:	Journal Article
Language:	English
Published:	United States Blackwell Publishing Ltd 01-03-2016 Wiley Subscription Services, Inc
Subjects:	Aged Aged, 80 and over Cholinesterase Inhibitors - therapeutic use clinical trial Dose-Response Relationship, Drug Double-Blind Method Female Follow-Up Studies Humans Male Middle Aged Myasthenia Gravis - drug therapy neuromuscular ocular myasthenia prednisone Pyridostigmine Bromide - therapeutic use Retrospective Studies Severity of Illness Index steroids Treatment Outcome clinical trial ocular myasthenia neuromuscular steroids prednisone
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Summary:	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
Bibliography:	National Institutes of Health/NCATS Institutional Clinical and Translational Science Award - No. UL1TR000001 istex:6A80210ADF867014E3F7182A88FBBAE1B341B938 FDA Orphan Products Development - No. R01FD003710 ark:/67375/WNG-TM97596V-P ArticleID:MUS24769 This study was supported by FDA Orphan Products Development (R01FD003710) to M.B. and by a National Institutes of Health/NCATS Institutional Clinical and Translational Science Award (UL1TR000001 to R.J.B.). See Appendix for listing of MSG participants. ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 ObjectType-Undefined-3
ISSN:	0148-639X 1097-4598 1097-4598
DOI:	10.1002/mus.24769