Background/Purpose .  The optimal management  of primary central nervous system vasculitis (PCNSV) remains unclear.   Cyclophosphamyde (CYC) in combination with glucocorticoids (GCs) or GCs alone are the most commonly used therapies.  It is not proven if other immunosuppressants, used in other  vasculitides, such as azathioprine (AZA), methotrexate (MTX) and mycophenolate mofetil (MMF), that are less toxic than CYC,  can be used  as induction and/or maintenance  therapies.   The aim of the study was to determine the efficacy and safety of MMF in PCNSV.                      

Methods .  The study  cohort consisted  of 163 consecutive patients with PCNSV seen at Mayo Clinic (Rochester, MN) from 1983 to 2011.  The diagnosis of PCNSV was based on findings of brain or spinal cord biopsy, cerebral angiography, or both.   To assess treatment response, we used the treating physician’s global opinion about the response to therapy. The degree of disability was defined using the modified Rankin scale. Outcomes, relapses, treatment response and ability to discontinue treatment at last follow-up were compared between patients treated with MMF and those receiving other  therapies.          

Results .  159/163 patients were treated at the time of diagnosis: 68 received GCs alone, 72  GCs and CYC,  2  CYC alone, 10  MMF and GCs, 6  AZA and GCs, and 1 rituximab and GCs.  6 more patients were treated with MMF after obtaining remission with CYC .  In total,  16 patients were treated with MMF:  7 males and 9 females with a median age  of 45.5 years (range:  20-72 years). Cerebral biopsy was performed in 10  and  was positive in 9 (3 associated cerebral amyloid angiopathy).  Clinical manifestations at presentation were:  headache (9 patients), altered cognition (9 patients), persistent neurologic deficit or stroke (8 patients),  seizures (6 patients) and  1 had intracranial hemorrhage.  Cerebral MRI showed infarctions in 10 patients (bilateral in 7) and prominent leptomeningeal enhancement in 4 patients. CSF abnormalities were observed in 14/16 patients. The median duration of follow-up was 34 months (range: 10-78 months). Cerebral angiography was positive in 7/11 patients and large vessel involvement was observed in 4 .  The median  dosage of MMF was 2 grams (range: 0.5-3 grams) and the median therapy duration 14.9 months (range: 1-31.6 months).   Rankin disability scores at diagnosis were similar  between patients treated with MMF and those receiving other therapies (Rankin score, 0-3: 68.7% versus  69.9%;  Rankin score, 4-6: 31.3% versus 30.1%; p = 1.000). A significantly lower proportion of patients treated with MMF had severe disability at last follow-up compared to those receiving  other therapies (Rankin score, 4-6:  0 versus 25.1%, p=0.023).  No statistically significant differences were observed in patients treated with MMF compared to those receiving other therapies regarding relapses [7/16 (43.7%) versus 37/143 (25.9%), p = 0.146], ability to discontinue therapy at last follow-up [4/16 (25%)  versus 36/142 (25.3%), p = 1.000], and treatment response [15/15 versus 111/142 (78.1%), p = 0.075]. Only 1 patient suspended MMF for a severe adverse event (leukopenia).                                                                                                                             

Conclusion .  In this retrospective study of a small number of PCNSV patients,  MMF was an effective and safe therapy.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/mycophenolate-mofetil-in-the-treatment-of-primary-central-nervous-system-vasculitis/