Mycophenolate mofetil in primary central nervous system vasculitis.

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.