Autologous Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica

Neuromyelitis Optica (NMO) is an immune-mediated astrocytopathy characterized by frequent demyelinating/necrotizing attacks of optic nerves and spinal cord. Treatments are highly toxic and incompletely effective. Fifty percent of patients are blind in one eye or require a mobility aide within 5 years. The immunological features of NMO, coupled with its severity make it an ideal candidate for a trial of autologous non-ablative hematopoietic stem cell transplantation (AHSCT) with the goal of disease remission and freedom from immunosuppressive medications. The objective of this study is to determine if NMO patients, despite maintenance therapy, experience a reduction in relapses and disability without ongoing immunosuppressants after AHSCT. We hypothesize a greater than or equal to 50% reduction in the proportion of patients relapsing 3 years post-transplant.