Autologous Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica

Neuromyelitis Optica (NMO) is an immune-mediated neurodegenerative disease of the central nervous system characterized by frequent demyelinating/necrotizing attacks of optic nerves and spinal cord leading to significant morbidity and mortality. Treatments are highly toxic and only minimally effective. Fifty percent of patients are blind in one eye or require a mobility aide within 5 years, with a 5-year survival of 68%. The immunological features of NMO, coupled with its severity make it an ideal candidate for a trial of autologous hematopoietic stem cell transplantation (AHSCT) in an effort to achieve remission and freedom from immunosuppressive medications. Our goal was to determine if NMO patients experience a reduction in relapses and associated disability without need for chronic immunosuppressant after AHSCT. We hypothesize => 50% reduction in the proportion of patients relapsing 3 years post-transplant.