Autologous hematopoietic stem cell transplantatio… [Mult Scler. 2014] – PubMed – NCBI.
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Autologous hematopoietic stem cell transplantation in neuromyelitis optica: A registry study of the EBMT Autoimmune Diseases Working Party.
Author information
- 1Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, via Olgettina 60, 20132 Milano, ItalyHematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Italy.
- 2Leukemia Immunotherapy Unit, Division of Regenerative Medicine, Stem Cells and Gene Therapy, Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Italy.
- 3Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Brazil.
- 4European 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.
- 5Department of Neuroscience, Uppsala University, Sweden.
- 6Department of Clinical Neuroscience, Neuroimmunology Unit, Karolinska Institute Solna, Center for Molecular Medicine, Sweden.
- 7Hematology Center, Karolinska University Hospital Huddinge, Sweden; Division of Hematology, Karolinska Institutet, Sweden.
- 8Department of Neurology, Charles University in Prague, 1st Faculty of Medicine and General Teaching Hospital, Czech Republic.
- 9Division of Hematology, University Hospital, Sweden.
- 101st Department of Medicine, Department of Haematology, Charles University in Prague, Czech Republic/1st Faculty of Medicine and General Teaching Hospital, Czech Republic.
- 11EBMT Study Office, Hôpital Saint-Antoine, France.
- 12Department of Haematology, Careggi University Hospital, Italy.
- 13Neurology Department, Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Italy.
- 14Department of Neuroscience, Ophthalmology and Genetics, San Martino Hospital, University of Genoa, Italy.
- 15Division of Hematology, San Martino Hospital, Italy.
- 16Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Italy.
- 17ADWP-EBMT Chair, Internal Medicine and Vascular Pathology Unit, Saint Louis Hospital, Assistance Publique des Hôpitaux de Paris, Paris 7 University, INSERM 976, France.
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.
© The Author(s), 2014.