Objective:

NA

Background:

Rituximab has been used as an alternative first-line induction therapy to cyclophosphamide for primary central nervous system vasculitis (PCNSV).  This study characterized clinical characteristics and induction therapy outcomes in a hospitalized cohort with PCNSV.

Design/Methods:

This was a single-center retrospective case-control study. Kruskal-Wallis test was used to compare median mRS improvement to worst mRS across groups. Intergroup comparisons and post-hoc covariate analysis were done using the Wilcoxon Rank-Sum test.

Results:

47 patients were treated with cyclophosphamide (n= 34, 72%), rituximab (n=8, 17%), or combined induction therapy (n= 5, 11%).  Mean age was 50. 51% of the cohort was female, 57% White, 19% Black, and 8.5% Hispanic. 39/46=85% had multifocal lesions; 13/39=33% had abnormal MRA brain; 18/28=64% abnormal catheter angiogram; 2/10=20% abnormal vessel wall MRI.  Brain biopsy confirmed vasculitis in 27/35=77%. Only 60% (26/43) had CSF pleocytosis, and 17% of patients without pleocytosis still had biopsy-proven vasculitis. Cyclophosphamide led to improved median mRS change compared to rituximab at 6 months (p = 0.015), but not at 12 months (p=0.4201). However, when including only biopsy-confirmed vasculitis cases, there was no difference in induction therapy (p = 0.447). Treatment with cyclophosphamide >3g (n=26) led to a greater change in median mRS compared to <3g (n=7) (-1.5 vs -1.0).   Initiating treatment within 1 month of symptom onset led to better mRS outcomes at 6 months (p= 0.024) and 12 months (p=0.044).   Age less than 50, Black/Hispanic race/ethnicity, necrotizing pathology or ICU admission did not impact mRS outcomes.   No rituximab patients experienced CTCAE greater than 3, whereas 13% of cyclophosphamide patients had grade 4 CTCAE.

Conclusions:

Cyclophosphamide treatment led to improved 6-month mRS in PCNSV compared to rituximab, though this was not significant when including only biopsy-confirmed cases.  Initiating treatment <1 month from symptom onset may lead to improved outcomes. Multicenter prospective randomized controlled trials are warranted.