title: An international, phase III, randomized trial of mycophenolate mofetil in myasthenia gravis.
abstract: BACKGROUND
  This prospective, randomized, double-blind, placebo-controlled, phase III trial assessed the efficacy, safety, and tolerability of mycophenolate mofetil (MMF) as a steroid-sparing agent in patients with myasthenia gravis (MG).
  
  
  METHODS
  Patients with acetylcholine receptor antibody-positive class II-IVa MG (MG Foundation of America [MGFA] criteria) taking corticosteroids for at least 4 weeks were randomized to MMF (2 g/day) or placebo for 36 weeks. The primary endpoint was a composite measure defined as achievement of minimal manifestations or pharmacologic remission (MGFA post-intervention status), with reduction of corticosteroid dose on a set schedule. Secondary endpoints included disease severity, quality-of-life scores, and safety.
  
  
  RESULTS
  A total of 44% of MMF-treated (n = 88) and 39% of placebo-receiving (n = 88) patients achieved the primary endpoint (p = 0.541). Improvements in mean quantitative MG, MG activities of daily living, and 36-item Short-Form health survey scores were similar in both groups. Numbers of adverse events were similar in both groups. The most commonly reported adverse events in the MMF-treated group were headache (12.5%) and worsening of MG (11.4%), and in the placebo group, worsening of MG (20.5%) and diarrhea (10.2%).
  
  
  CONCLUSIONS
  Initiation of mycophenolate mofetil (MMF) treatment was not superior to placebo in maintaining myasthenia gravis (MG) control during a 36-week schedule of prednisone tapering. There were no significant differences in the primary or secondary endpoints between the study groups. MMF was well tolerated and adverse events were consistent with previous studies. Experience from this large, international, multicenter, phase III study employing full MG Foundation of America guidelines will aid the design of future MG studies.
authors.affiliation: Division of Neurology, Duke University Medical Center, Durham, NC, USA. Donald.Sanders@Duke.edu
authors.forename: D B
authors.initials: DB
authors.lastname: Sanders
authors.forename: I K
authors.initials: IK
authors.lastname: Hart
authors.forename: R
authors.initials: R
authors.lastname: Mantegazza
authors.forename: S S
authors.initials: SS
authors.lastname: Shukla
authors.forename: Z A
authors.initials: ZA
authors.lastname: Siddiqi
authors.forename: M H V
authors.initials: MH
authors.lastname: De Baets
authors.forename: A
authors.initials: A
authors.lastname: Melms
authors.forename: M W
authors.initials: MW
authors.lastname: Nicolle
authors.forename: N
authors.initials: N
authors.lastname: Solomons
authors.forename: D P
authors.initials: DP
authors.lastname: Richman
mesh_terms: D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007391:International Cooperation; D008297:Male; D008875:Middle Aged; D009157:Myasthenia Gravis / Q000188:drug therapy* / Q000276:immunology; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011241:Prednisone / Q000627:therapeutic use; D011446:Prospective Studies
chemical_list: D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid; D011241:Prednisone