title: Efficacy of prednisone for the treatment of ocular myasthenia (EPITOME): A randomized, controlled trial.
abstract: BACKGROUND
  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.
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Benatar
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Michael P
authors.initials: MP
authors.lastname: Mcdermott
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Donald B
authors.initials: DB
authors.lastname: Sanders
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Gil I
authors.initials: GI
authors.lastname: Wolfe
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Barohn
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Nowak
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Hehir
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Vern
authors.initials: V
authors.lastname: Juel
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Hans
authors.initials: H
authors.lastname: Katzberg
authors.affiliation: Department of Neurology, Miller School of Medicine, University of Miami, 1120 NW 14th Street, CRB 1318, Miami, Florida, 33136, USA.
authors.forename: Rabi
authors.initials: R
authors.lastname: Tawil
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D002800:Cholinesterase Inhibitors / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D009157:Myasthenia Gravis / Q000188:drug therapy*; D011729:Pyridostigmine Bromide / Q000627:therapeutic use*; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D002800:Cholinesterase Inhibitors; D011729:Pyridostigmine Bromide
keywords: clinical trial; neuromuscular; ocular myasthenia; prednisone; steroids