Background

Primary Central Nervous System Vasculitis (PCNSV) is responsible for 3%–5% of strokes before age 50. It presents with clinical, radiological, and pathological variability. Optimal management is unknown given the absence of randomized clinical trials.

Aims of the study

Explore whether tocilizumab, an anti-interleukin-6 monoclonal antibody, is an effective treatment for refractory PCNSV.

Methods

Patients with PCNSV treated with tocilizumab in a single tertiary center were reviewed.

Results

Three patients were identified. In two of them, MRI-revealed ischemic lesions. The other presented with a subcortical hemispheric pseudotumoral lesion. Brain biopsy was inconclusive in two patients. Due to a significant number of relapses and clinical deterioration despite other immunosuppressive drugs, tocilizumab was initiated and induced a long remission period up to 44 months. Observed side effects were a fungic infection, neutropenia and thrombocytopenia (both transitory), and a pulmonary embolism in one of the cases.

Conclusions

Tocilizumab might be a therapeutic option for PCNSV (Class IV evidence), given its efficacy and safety. We propose a novel pathway for diagnosis and therapeutics of PCNSV with the purpose of improving the diagnosis, monitoring, and prognosis of this heterogeneous disorder, setting the framework for future use of tocilizumab in this condition.