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Mycophenolate mofetil in primary central nervous system vasculitis

Carlo Salvarani1, Robert D Brown2, Teresa J H Christianson3

  • 1Unitá Operativa di Reumatologia, Azienda Ospedaliera-IRCCS di Reggio Emilia, Reggio Emilia, Italy.

Seminars in Arthritis and Rheumatism
|March 25, 2015
PubMed
Abstract

Insights

Mycophenolate mofetil (MMF) shows promise for treating adult primary central nervous system vasculitis (PCNSV). Patients on MMF experienced less disability compared to other treatments, with a favorable safety profile.

Area of Science:

  • Neurology
  • Immunology
  • Rheumatology

Background:

  • Primary central nervous system vasculitis (PCNSV) is a rare condition affecting blood vessels in the brain and spinal cord.
  • Treatment options for PCNSV can be limited, necessitating evaluation of alternative therapies.

Purpose of the Study:

  • To assess the effectiveness and safety of mycophenolate mofetil (MMF) for adult PCNSV.
  • To compare MMF outcomes with other treatment regimens.

Main Methods:

  • Retrospective cohort study of 163 adult PCNSV patients at Mayo Clinic (1983-2011).
  • Comparison of patients treated with MMF versus other therapies, including cyclophosphamide and prednisone.

Main Results:

  • Sixteen patients received MMF, often in combination with glucocorticoids (GCs), achieving favorable responses.
  • MMF-treated patients showed significantly less disability at follow-up compared to other therapies (p=0.023) and cyclophosphamide/prednisone (p=0.017).
  • No significant differences in relapse rates or therapy discontinuation; a trend towards better response with MMF (p=0.075). MMF was well-tolerated, with only one patient discontinuing due to leukopenia.

Conclusions:

  • Mycophenolate mofetil (MMF) appears to be an effective and safe treatment option for adult primary central nervous system vasculitis (PCNSV).
  • MMF may offer a favorable alternative for managing PCNSV, potentially leading to better functional outcomes.
Keywords:
Mycophenolate mofetilPCNSVTherapyVasculitis

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