Strategies for treatment of childhood primary angiitis of the central nervous system

OBJECTIVE: Childhood primary angiitis of the CNS (cPACNS) is a devastating neurologic disease. No standardized treatment protocols exist, and evidence is limited to open-label cohort studies and case reports. The aim of this review is to summarize the literature and provide informed treatment recommendations. METHODS: A scoping review of cPACNS literature from January 2000 to December 2018 was conducted using Ovid, MEDLINE, PubMed, Embase, Cochrane Database of Systematic Reviews, Cochrane Central Register of Controlled Trials, ClinicalTrials.gov, Vasculitis Foundation, European Vasculitis Society, CanVasc, Google Scholar, and Web of Science. Potentially relevant articles were selected for full-text review using the STROBE checklist if they met the following inclusion criteria: (1) reported treatment, (2) addressed pediatrics, (3) focused on the disease of interest, (4) included ≥5 patients, (5) original research, and (6) full-length articles. Reviews, expert opinions, editorials, case reports with <5 patients, articles lacking treatment information, or non-English articles were excluded. A standardized assessment tool measured study quality. Treatment and outcomes were summarized. RESULTS: Of 2,597 articles screened, 7 studies were deemed high quality. No trials were available so no meta-analysis was possible. Overall, treatment strategies recommended are induction with acute antithrombotic therapy subsequently followed by high-dose oral prednisone taper over 3–12 months and long-term platelet therapy. In angiography-positive progressive–cPACNS and angiography-negative–cPACNS, we also recommend 6 months of IV cyclophosphamide therapy, with trimethoprim/sulfamethoxazole as part of induction, and maintenance therapy with mycophenolate mofetil/mycophenolic acid. CONCLUSION: No grade-A evidence exists; however, this review provides recommendations for treatment of cPACNS.