Background/Purpose: To determine the response to therapy and long-term outcome of PCNSV we reviewed all cases of PCNSV seen over a 29-year period.   

Methods: The  cohort included 163 consecutive patients diagnosed with PCNSV at Mayo Clinic Rochester over a 29-year period (1983 through 2011). The diagnosis was based on findings of brain or spinal cord biopsy, cerebral angiography, or both. Biopsy specimens were reviewed by a neuropathologist  and angiograms by a neuroradiologist.   Information about clinical findings, laboratory, radiologic imaging, type of, duration of, and response to treatment, number of relapses, functional status at followup, and cause of death were completed for all cases.   

Results: In 75 patients, glucocorticoids (GCs) were the only initial therapeutic agents.  In 66 intravenous pulse GC doses  (median:  5 pulses of methylprednisolone  [1 gm/pulse]) were administered before oral GCs were started. The median initial oral prednisone dose was 60 mg/day. The median duration of prednisone therapy was 10 months, and 75% were treated for 19 months or less.  In 69 patients cyclophosphamide (CYC) (49 had daily oral doses, 23 had intermittent intravenous pulses) and prednisone were the only initial treatment.  The median initial dose of oral CYC was 150 mg/day and the median duration of therapy was 7 months. The median dose of intravenous pulse CYC was 1 gm/month. Other initial treatments included CYC alone (2) azathioprine (median initial dose 100 mg/day) and GCs (6), mycophenolate mophetil and GCs (3),  rituximab and GCs (1), plasma-exchange, CYC and GC therapy (2), and infliximab (1).  Four patients received no specific therapy. In the 75 treated with GCs only, a favorable  response was observed in  83%.   In those with CYC plus GCs, 76% responded favorably. 44 patients had relapses that led to a change in therapy.   The frequency of relapses were significantly lower in the patients treated with CYC compared to those treated with GCs alone (18% vs 39%, p = 0.006). The median follow-up duration of the 163 patients was 12 months (range: 0-13.7 years).  Age- and sex-adjusted survival of the patients with PCNSV was significantly reduced compared to the estimated survival of the US white population (p < 0.001). 25 patients died during follow-up.   The cause of death was cerebral infarction in 10 patients.  Univariate Cox proportional hazards model was used to assess survival  and findings at diagnosis: older age at diagnosis (HZ 1.4) and  cerebral infarction at MRI (HZ 4.4) were associated with poor survival, while gadolinium enhanced cerebral lesions or meninges at MRI (HZ 0.20) with  better survival.  Univariate logistic regression analysis was used to assess association of findings at diagnosis with Rankin score outcomes.  Older age (HZ 1.4) and MRI evidence of cerebral infarction (HZ 3.7) at presentation were associated with a high disability score at last follow-up (Rankin score of 4-6), while patients with gadolinium enhanced cerebral lesions or meninges had lower disability at follow-up (HR 0.35).

Conclusion: Most patients with PCNSV showed a favorable response to therapy. Recognition of findings at diagnosis associated with poor outcomes may aid decisions regarding initial therapy.

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