Journal of Neurology

, Volume 265, Issue 11, pp 2688–2694 | Cite as

Efficacy and safety of mycophenolate mofetil in progressive multiple sclerosis patients

  • Rami Fakih
  • Marcelo Matiello
  • Tanuja Chitnis
  • James M. StankiewiczEmail author
Original Communication



There are increasingly effective therapies for relapsing forms of multiple sclerosis (MS); however, the options for the progressive patient population are limited. The effect of mycophenolate mofetil (MMF), a disease-modifying agent for several autoimmune diseases, in progressive MS has not been explored effectively. We performed a prospective study to assess the safety and efficacy of MMF in progressive MS patients.


We identified 64 patients enrolled in the comprehensive longitudinal database at the Partners MS Center, who fulfilled our inclusion criteria. They were exposed to MMF for at least 1 year with recorded clinical outcomes. Efficacy was assessed by comparing the absolute relapse rate (ARR), and the mean Expanded Disability Status Scale (EDSS) and timed 25 foot walk (T25FW) test scores before and after MMF treatment.


At the start of MMF, 78% of patients (n = 50) were in the 4–7.5 EDSS range. There was a slight increase in mean EDSS from 5.49 ± 1.65 (n = 48) 1 year before MMF start to 5.85 ± 1.56 (n = 48) 1 year after (p = 0.020). The mean T25FW score increased 1 year before MMF from 12.3 ± 9.6 s (n = 38) to 15.6 ± 12.3 s (n = 38) 1 year after (p = 0.009). The ARR in the 2 years pre-MMF period was 0.30 ± 0.63, which decreased to a 0.09 ± 0.29 (p = 0.022) 2 years post MMF.


MMF did not affect disease progression but did influence relapse rate. We believe that other medication options should be considered before MMF in advanced progressive patients.


Progressive multiple sclerosis Mycophenolate mofetil Disease-modifying therapy Efficacy 



We thank Mariann Polgar and Brian Healy from the Translational Neuroimmunology Research Center.


We thank Merck Serono and the National MS Society Nancy Davis Center Without Walls for their support of the CLIMB study.

Compliance with ethical standards

Conflicts of interest

RF declares no conflict of interests. MM was a consultant for TerumoBCT and Octapharma. TC has received consulting/advisory fees from Biogen and Celgene, and serves on clinical trial advisory committees for Novartis and Sanofi-Genzyme. JS was a consultant for Biogen Idec, Genzyme, Celgene, Bayer, and EMD Serono.

Ethics approval

All subjects gave their informed consent prior to their inclusion in the study. The Partners Institutional Review Board approved this study.


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Copyright information

© Springer-Verlag GmbH Germany, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Harvard Medical School, Brigham and Women’s HospitalPartners Multiple Sclerosis CenterBostonUSA
  2. 2.Harvard Medical SchoolMassachusetts General HospitalBostonUSA

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