BACKGROUND: Neuromyelitis optica (NMO) is an inflammatory autoimmune disorder of the central nervous system, hallmarked by pathogenic anti-aquaporin 4 antibodies. NMO prognosis is worse compared with multiple sclerosis. OBJECTIVE: The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) conducted a retrospective survey to analyze disease outcome following autologous stem cell transplantation (ASCT). METHODS: This retrospective multicenter study assessed the efficacy and safety of ASCT in 16 patients suffering from refractory NMO reported to the EBMT registry between 2001 and 2011. RESULTS: Fifteen patients were successfully mobilized with cyclophosphamide (Cy) and G-CSF, one with G-CSF alone. All patients received an unmanipulated autologous peripheral blood stem cell graft, after conditioning with BEAM plus anti-thymocyte globulin (ATG, n = 9 patients), thiotepa-Cy (n = 3) or Cy (200 mg/kg) plus ATG (n = 4). After a median follow-up of 47 months, three of 16 cases were progression and treatment free, while in the remaining 13 patients further treatments were administered for disability progression or relapse after ASCT. Altogether, relapse-free survival at three and five years was 31% and 10%, respectively, while progression-free survival remained 48% at three and five years. CONCLUSIONS: In these NMO patients, highly resistant to conventional treatment, ASCT allows for temporary control of the disease, despite a tendency to progress or relapse in the long term.

1st Department of Medicine Department of Haematology Charles University Prague Czech Republic 1st Faculty of Medicine and General Teaching Hospital Czech Republic

ADWP EBMT Chair Internal Medicine and Vascular Pathology Unit Saint Louis Hospital Assistance Publique des Hôpitaux de Paris Paris 7 University INSERM 976 France

Department of Clinical Neuroscience Neuroimmunology Unit Karolinska Institute Solna Center for Molecular Medicine Sweden

Department of Haematology Careggi University Hospital Italy

Department of Internal Medicine Ribeirão Preto Medical School University of São Paulo Brazil

Department of Neurology Charles University Prague 1st Faculty of Medicine and General Teaching Hospital Czech Republic

Department of Neuroscience Ophthalmology and Genetics San Martino Hospital University of Genoa Italy

Department of Neuroscience Uppsala University Sweden

Division of Hematology San Martino Hospital Italy

Division of Hematology University Hospital Sweden

EBMT Study Office Hôpital Saint Antoine France

European Group for Blood and Marrow Transplantation EBMT Hôpital Saint Antoine Assistance Publique des Hôpitaux de Paris France

Hematology and Bone Marrow Transplantation Unit San Raffaele Scientific Institute Italy

Hematology Center Karolinska University Hospital Huddinge Sweden; Division of Hematology Karolinska Institutet Sweden

Leukemia Immunotherapy Unit Division of Regenerative Medicine Stem Cells and Gene Therapy Vita Salute San Raffaele University San Raffaele Scientific Institute Italy

Neurology Department Vita Salute San Raffaele University San Raffaele Scientific Institute Italy

References provided by Crossref.org

Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases