Abstract
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.
Glossary
- AN=
- angiography-negative;
- APNP=
- angiography-positive nonprogressive;
- APP=
- angiography-positive progressive;
- ASA=
- aspirin;
- CA=
- conventional angiography;
- cPACNS=
- childhood primary angiitis of the CNS;
- DSA=
- digital subtraction angiography;
- IVMP=
- IV methylprednisolone;
- MMF=
- mycophenolate mofetil;
- MRA=
- magnetic resonance angiography;
- NASCET=
- North American Symptomatic Carotid Endarterectomy Trial;
- PedsQL=
- Pediatric Quality of Life;
- PGA=
- Physicians Global Assessment;
- PSOM=
- Pediatric Stroke Outcome Measure;
- vWF=
- von Willebrand factor
Footnotes
Go to Neurology.org/NN for full disclosures. Funding information is provided at the end of the article.
The Article Processing Charge was funded by the authors.
- Received January 24, 2019.
- Accepted in final form March 4, 2019.
- Copyright © 2019 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.
This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.
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