A Pilot Trial of Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optic Spectrum Disorder

Abstract Background Autologous hematopoietic stem cell transplantation (AHSCT) has become a standard treatment in multiple sclerosis. The role of AHSCT for neuromyelitis optica spectrum disorder (NMO/NMOSD) is unclear. We studied AHSCT in NMO/NMOSD patients who have failed conventional immunosuppressive therapy. Methods Eligible patients received AHSCT with cyclophosphamide, rabbit antithymocyte globulin, and rituximab and followed for ≥ five years. The primary outcome was relapse-free status at year three. Additional outcomes included relapse status at year five, relapse rate, EDSS, MRI activity, and overall survival. Results Between 2010-2016, three patients were enrolled. One patient has had no evidence of disease activity over 10 years, one had improvement in relapse rate and EDSS but did have a breakthrough clinically and radiologically requiring rituximab at year five, and the third died at year 3.5 due to uncontrollable NMOSD relapses and accumulation of marked disability. Conclusion In our trial, AHSCT appeared safe, and moderately effective with two of three patients showing improvement in disease activity and disability. Future studies should be undertaken to determine the ideal AHSCT conditioning and the characteristics of patients likely to enter long-term remission.