Efficacy of eculizumab in acute refractory pediatric neuromyelitis optica: A case report
Michael Enriquez, Scott Rosenthal, Loren McLendon, Jeffrey L. Bennett, Amanda L. Piquet, Ryan Kammeyer
Abstract
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.