Pediatric Central Nervous System Demyelinating Diseases

Tanuja Chitnis, MD, FAAN p. 793-814 June 2019, Vol.25, No.3 doi: 10.1212/CON.0000000000000730
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PURPOSE OF REVIEW: This article provides an up-to-date summary of the categories, diagnosis, and management of pediatric demyelinating disorders.

Recent Findings: Understanding of the diverse spectrum of pediatric demyelinating disorders, including monophasic and multiphasic forms, has improved. Pediatric multiple sclerosis (MS) is the most common demyelinating disorder in children, and recent genetic and environmental risk research has clarified that pediatric MS is on the same continuum of disease as adult MS. Recent advances in the treatment of pediatric MS include clinical trials leading to regulatory agency–approved treatments. The identification of myelin oligodendrocyte glycoprotein and aquaporin-4 antibodies in children has been a major advance, allowing for appropriate treatment and management of these syndromes.

Summary: Antibody testing is now helping to define subtypes of pediatric demyelinating disorders, including myelin oligodendrocyte glycoprotein–seropositive and aquaporin-4–seropositive cases that are distinct from pediatric MS. Treatments for pediatric MS are being evaluated in clinical trials.

Address correspondence to Dr Tanuja Chitnis, 55 Fruit St, ACC708, Massachusetts General Hospital, Boston, MA 02114, tchitnis@rics.bwh.harvard.edu.

RELATIONSHIP DISCLOSURE: Dr Chitnis serves on scientific advisory boards for Biogen, Celgene Corporation, F. Hoffmann-La Roche Ltd, Novartis AG, and Sanofi Genzyme and as a consultant for Biogen. Dr Chitnis receives research/grant support from the Consortium of Multiple Sclerosis Centers; the Department of Defense; EMD Serono, Inc; the Guthy-Jackson Charitable Foundation; Mallinckrodt Pharmaceuticals; the National Institutes of Health (R01AG057505); the National Multiple Sclerosis Society; Novartis AG; Octave Bioscience; and Verily Life Sciences LLC.

UNLABELED USE OF PRODUCTS/INVESTIGATIONAL USE DISCLOSURE: Dr Chitnis discusses the unlabeled/investigational use of glatiramer acetate, interferon beta, natalizumab, and rituximab for pediatric multiple sclerosis and mycophenolate mofetil for myelin oligodendrocyte glycoprotein antibody–associated disorders and neuromyelitis optica spectrum disorder in children.

© 2019 American Academy of Neurology.