Neuromyelitis optica is an uncommon inflammatory demyelinating CNS disorder that is distinct from multiple sclerosis with respect to clinical, laboratory, neuroimaging, and prognostic characteristics. Autoantibodies that target aquaporin-4 are highly specific for neuromyelitis optica and have helped define a spectrum of disease beyond the classic definition of acute transverse myelitis and optic neuritis. Accumulating evidence supports the pathogenic potential that these autoantibodies possess in relation to the unique vasculocentric immunopathology of the disease. Current treatment strategies therefore include the use of corticosteroids and plasmapheresis for acute attacks and general or humoral immunosuppression for attack prevention. Ongoing research will focus on establishing the pathogenic mechanisms of the disease, in part derived from newly reported animal models, and testing-focused treatment strategies that evolve from this knowledge.
Note: Text referenced in the Quintessentials Preferred Responses, which appear later in this issue, is indicated in yellow shading throughout this article.
Relationship Disclosure: Dr Wingerchuk has received research support from Alexion, Genentech, Genzyme, and the National Multiple Sclerosis Society.
Unlabeled Use of Products/Investigational Use Disclosure: Dr Wingerchuk discusses the unlabeled use of corticosteroids, plasmapheresis, and several chronic immunotherapies for the treatment of neuromyelitis optica.