Efficacy of eculizumab in acute refractory pediatric neuromyelitis optica: A case report

Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system caused by autoantibodies against the aquaporin-4 (AQP4) water channel. Inflammatory injury is often severe and focused on the optic nerves, spinal cord, and other CNS regions with high AQP4 expression. Acute management includes pulse corticosteroids and plasmapheresis; however, many patients have incomplete recovery. We describe the successful use of eculizumab in treating an acute severe refractory brainstem syndrome in a pediatric patient with NMOSD, highlighting the potential utility of eculizumab as a potent, acute therapy in pediatric NMOSD.