Pediatric Neuromyelitis Optica Spectrum Disorder

Abstract

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a demyelinating disease with a high relapse rate and risk of disability accrual. The condition is an astrocytopathy, with antibodies to the aquaporin-4 (AQP4) water channel being detected in AQP4-IgG seropositive disease. Presentation is uncommon in the pediatric age range, accounting for about 3%-5% of cases. NMOSD is more prevalent in populations of Black or East Asian ancestry. Core clinical syndromes include optic neuritis, acute myelitis, area postrema syndrome, acute brainstem syndrome, acute diencephalic syndrome, and symptomatic cerebral syndrome. First-line treatment options in pediatrics include rituximab, azathioprine, and mycophenolate mofetil. Over half of children with AQP4-IgG seropositive NMOSD develop permanent disability, particularly in visual and motor domains. Novel therapeutic targets in the adult population have been developed and are changing the treatment landscape for this disorder.