title: Autologous hematopoietic stem cell transplantation in neuromyelitis optica: a registry study of the EBMT Autoimmune Diseases Working Party.
abstract: 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.
authors: Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Italy greco.raffaella@hsr.it.
authors: Raffaella
authors: R
authors: Greco
authors: Leukemia Immunotherapy Unit, Division of Regenerative Medicine, Stem Cells and Gene Therapy, Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Italy.
authors: Attilio
authors: A
authors: Bondanza
authors: Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Brazil.
authors: Maria Carolina
authors: MC
authors: Oliveira
authors: European Group for Blood and Marrow Transplantation (EBMT) Study Office, Autoimmune Diseases Working Party (ADWP)-EBMT, Hôpital Saint-Antoine, Assistance Publique des Hôpitaux de Paris, France.
authors: Manuela
authors: M
authors: Badoglio
authors: Department of Neuroscience, Uppsala University, Sweden.
authors: Joachim
authors: J
authors: Burman
authors: Department of Clinical Neuroscience, Neuroimmunology Unit, Karolinska Institute Solna, Center for Molecular Medicine, Sweden.
authors: Fredrik
authors: F
authors: Piehl
authors: Hematology Center, Karolinska University Hospital Huddinge, Sweden; Division of Hematology, Karolinska Institutet, Sweden.
authors: Hans
authors: H
authors: Hagglund
authors: Department of Neurology, Charles University in Prague, 1st Faculty of Medicine and General Teaching Hospital, Czech Republic.
authors: Eva
authors: E
authors: Krasulova
authors: Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Brazil.
authors: Belinda Pinto
authors: BP
authors: Simões
authors: Division of Hematology, University Hospital, Sweden.
authors: Kristina
authors: K
authors: Carlson
authors: 1st Department of Medicine, Department of Haematology, Charles University in Prague, Czech Republic/1st Faculty of Medicine and General Teaching Hospital, Czech Republic.
authors: David
authors: D
authors: Pohlreich
authors: EBMT Study Office, Hôpital Saint-Antoine, France.
authors: Myriam
authors: M
authors: Labopin
authors: Department of Haematology, Careggi University Hospital, Italy.
authors: Riccardo
authors: R
authors: Saccardi
authors: Neurology Department, Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Italy.
authors: Giancarlo
authors: G
authors: Comi
authors: Department of Neuroscience, Ophthalmology and Genetics, San Martino Hospital, University of Genoa, Italy.
authors: Gian Luigi
authors: GL
authors: Mancardi
authors: Division of Hematology, San Martino Hospital, Italy.
authors: Andrea
authors: A
authors: Bacigalupo
authors: Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Italy.
authors: Fabio
authors: F
authors: Ciceri
authors: ADWP-EBMT Chair, Internal Medicine and Vascular Pathology Unit, Saint Louis Hospital, Assistance Publique des Hôpitaux de Paris, Paris 7 University, INSERM 976, France.
authors: Dominique
authors: D
authors: Farge
mesh_terms: D000328:Adult; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009471:Neuromyelitis Optica / Q000601:surgery*; D017063:Outcome Assessment, Health Care / Q000379:methods*; D012042:Registries; D014182:Transplantation, Autologous; D055815:Young Adult
keywords: Neuromyelitis optica; autologous stem cell transplantation; disease outcome