Brain biopsy in children with primary small-vessel central nervous system vasculitis.

Objective: Primary angiitis of the central nervous system in childhood (cPACNS) is an immune-mediated inflammatory process directed toward blood vessels in the central nervous system. It has been associated with variable clinical and radiological presentations, and devastating consequences without treatment. Brain biopsy is required for definitive diagnosis.

Treatment of small vessel primary CNS vasculitis in children: an open-label cohort study.

Background: There is no treatment protocol or standardised documentation of neurological outcome for patients with small vessel childhood primary angiitis of the CNS, a rare inflammatory brain disease. We aimed to assess a treatment regimen and describe long-term neurological outcomes in a cohort of children with this disorder.

Methods: We did a single-centre open-label cohort study in children with small vessel childhood primary angiitis of the CNS who were less than 18 years old at diagnosis.