Neurology
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Published online before print April 23, 2008
(Neurology 2008, doi:10.1212/01.wnl.0000312374.95186.cc)
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Received July 27, 2007
Accepted January 7, 2008

An international, phase III, randomized trial of mycophenolate mofetil in myasthenia gravis

D. B. Sanders MD*, I. K. Hart FRCP, R. Mantegazza MD, S. S. Shukla MD, Z. A. Siddiqi MD, PhD, M. H.V. De Baets MD, A. Melms MD, M. W. Nicolle MD, DPhil, N. Solomons MD, and D. P. Richman MD

From the Division of Neurology (D.B.S.), Duke University Medical Center, Durham, NC; University Division of Neuroscience (I.K.H.), The Walton Centre for Neurology and Neurosurgery, Liverpool, UK; Department of Neuromuscular Diseases (R.M.), "Carlo Besta" Neurological Institute Foundation, Milan, Italy; Aspreva Pharmaceuticals Inc. (S.S.S.), Basking Ridge, NJ; Division of Neurology/Department of Medicine (Z.A.S.), University of Alberta, Edmonton, AB, Canada; Department of Neurology (M.H.V.D.B.), Academical Hospital, Maastricht, The Netherlands; Neurologische Universitatsklinik (A.M.), Eberhard-Karls-Universitat, Tubingen, Germany; University Hospital LHSC (M.W.N.), University of Western Ontario, London, ON, Canada; Aspreva Pharmaceuticals Corp. (N.S.), Victoria, BC, Canada; and Department of Neurology and the Center for Neuroscience (D.P.R.), University of California, Davis.


* To whom correspondence should be addressed. E-mail: Donald.Sanders{at}Duke.edu.

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.






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