Brain Advance Access originally published online on July 10, 2007
Brain 2007 130(8):2004-2010; doi:10.1093/brain/awm144
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Mycophenolate mofetil as adjunctive therapy for MMN patients: a randomized, controlled trial
Department of Neurology, Rudolf Magnus Institute of Neuroscience, University Medical Centre Utrecht, the Netherlands
Correspondence to: Dr Renske M. Van den Berg-Vos, MD, PhD, Department of Neurology, Rudolf Magnus Institute of Neuroscience, PO Box 85500 3508 GA Utrecht, the Netherlands E-mail: r.vandenberg-vos{at}slaz.nl
Multifocal motor neuropathy (MMN) is an immune-mediated disorder characterized by slowly progressive asymmetrical limb weakness. Treatment with immunoglobulins (IVIg) leads to improvement of muscle strength. Anecdotal evidence suggests that immunosuppressive drugs as adjunctive therapy may be beneficial. Mycophenolate mofetil (MMF) is a potent and safe immunosuppressant. Safety and efficacy of MMF as adjunctive therapy for MMN patients receiving IVIg maintenance treatment were evaluated in a randomized controlled trial. MMN patients responding to IVIg treatment were eligible for randomization. Muscle strength and functional status were assessed at monthly intervals for 1 year. Three months after the start of MMF or placebo treatment, IVIg doses were reduced stepwise, until a deterioration of functioning or decline in muscle strength could be observed. An IVIg dose reduction of 50% during adjunctive treatment was defined as a primary endpoint. Secondary outcome measures were improvement in muscle strength and functional status after 3 months and reduction of anti GM1-IgM titres after 12 months of MMF treatment. Twenty-eight patients were randomized. One patient allocated to MMF reached the primary endpoint of 50% IVIg dose reduction. After 12 months IVIg reduction did not differ significantly between the two treatment groups. Patients did not experience drug toxicity and none of the patients showed significant disease progression after 12 months. Muscle strength and functional scores after 3 months and anti GM1-IgM titres after 12 months did not change. Adjunctive treatment of MMN patients with MMF at a dose of 1 g twice daily is safe but does not alter disease course or allow significant reduction of IVIg doses.
Key Words: multifocal motor neuropathy; immune-mediated neuropathy; immunosuppressant; treatment; immunoglobulins
Abbreviations: MMN, multifocal motor neuropathy; IVIg, intravenous human immunoglobulins; MMF, mycophenolate mofetil; MMT, manual muscle testing; MRC, Muscle Research Council; SES, self-evaluation scale; ELISA, enzyme-linked immunosorbent assay; SAE, Serious adverse events
Received February 28, 2007. Revised May 11, 2007. Accepted May 29, 2007.
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