Background/Purpose: To evaluate long-term treatment and outcomes of a large cohort of patients with primary central nervous system vasculitis (PCNSV)

Methods: The study cohort consisted of 191 consecutive patients seen at Mayo Clinic (Rochester, MN) over 35-years (1983 to 2017). The follow-up and treatment of all PCNSV patients were updated until June 2018. The diagnosis of PCNSV was based on findings of brain or spinal cord biopsy, cerebral angiography, or both. Biopsy specimens were reviewed by a neuropathologist, and neurological imaging by a neuroradiologist. We used the treating physician’s global opinion to assess response to therapy. Disability was defined using the modified Rankin scale. Outcomes, relapses, treatment response and ability to suspend treatment at last follow-up were analyzed (mean follow-up length: 54.2+73.6 months).

Results: Treatment at diagnosis in 186/191 patients were as follows: 72 prednisone (PDN) alone, 90 PDN and cyclophosphamide (CYC), 2 CYC alone, 13 mycophenolate mofetil (MMF) and PDN, 6 azathioprine (AZA) and PDN, 1 chlorambucil and PDN, 1 rituximab (RTX) and PDN and 1 infliximab and PDN. A favorable response was observed in 83.1% of patients treated with PDN alone, in 81.2% of patients treated with CYC and PDN, and in 94.7% of patients treated with MMF or AZA and PDN. Relapses were observed in 30.4% of patients (13.1% had at least 2 flares), and 34.6% of patients discontinued therapy by the time of the last followup visit. Patients with prominent gadoliniun-enhanced cerebral or meningeal lesions had more frequently relapses (OR 1.76), while no significant differences in relapse rates were observed comparing patients treated with only PDN versus those associating PDN and CYC. High disability scores (Rankin score of 4-6) were equally frequent at last followup in patients with relapsing disease (34.5%) and in those without relapses (30.1%). Large vessel involvement (OR 2.80) and cerebral infarction on imaging at diagnosis (OR 3.92) were associated with a poor treatment response. Cerebral infarction at diagnosis (OR 1.76) were also associated with continued treatment at last followup. Higher disability scores at last followup visit were associated with increasing age at diagnosis (OR 1.48) and cerebral infarctions on imaging (OR 2.09). No differences in mortality or in high disability scores at last follow-up (Rankin score 4-6) were observed comparing patients initially treated with PDN plus CYC (HR 0.740, OR 1.450) and PDN plus AZA or MMF (HR 0.538, OR 0.486) to those treated with PDN alone.  26 patients treated with MMF at the beginning or during followup had less frequent high disability scores (Rankin score of 4-6) at last followup than those treated with other drugs initially (15.3% vs 34.5%, p = 0.06) , while patients given MMF had more relapses (53.8% vs 27.5%, p = 0.01). Three patients were treated with RTX for a disease flare and one as initial treatment. In 3 patients RTX was associated with a marked reduction in the number of flares (from 9 before starting RTX to 2 after).

Conclusion: The majority of patients with PCNSV responded to treatment. Cerebral infarctions at diagnosis were associated with poor response to treatment and worst outcomes. MMF and RTX appears to be effective therapy for PCNSV.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/adult-primary-central-nervous-system-vasculitis-treatment-and-course-a-long-term-follow-up-study/