Raffaella Greco1, Attilio Bondanza2, Maria Carolina Oliveira3, Manuela Badoglio4, Joachim Burman5, Fredrik Piehl6, Hans Hagglund7, Eva Krasulova8, Belinda Pinto Simões3, Kristina Carlson9, David Pohlreich10, Myriam Labopin11, Riccardo Saccardi12, Giancarlo Comi13, Gian Luigi Mancardi14, Andrea Bacigalupo15, Fabio Ciceri16, Dominique Farge17. 1. Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Italy greco.raffaella@hsr.it. 2. Leukemia Immunotherapy Unit, Division of Regenerative Medicine, Stem Cells and Gene Therapy, Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Italy. 3. Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Brazil. 4. 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. 5. Department of Neuroscience, Uppsala University, Sweden. 6. Department of Clinical Neuroscience, Neuroimmunology Unit, Karolinska Institute Solna, Center for Molecular Medicine, Sweden. 7. Hematology Center, Karolinska University Hospital Huddinge, Sweden; Division of Hematology, Karolinska Institutet, Sweden. 8. Department of Neurology, Charles University in Prague, 1st Faculty of Medicine and General Teaching Hospital, Czech Republic. 9. Division of Hematology, University Hospital, Sweden. 10. 1st Department of Medicine, Department of Haematology, Charles University in Prague, Czech Republic/1st Faculty of Medicine and General Teaching Hospital, Czech Republic. 11. EBMT Study Office, Hôpital Saint-Antoine, France. 12. Department of Haematology, Careggi University Hospital, Italy. 13. Neurology Department, Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Italy. 14. Department of Neuroscience, Ophthalmology and Genetics, San Martino Hospital, University of Genoa, Italy. 15. Division of Hematology, San Martino Hospital, Italy. 16. Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Italy. 17. ADWP-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.
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: Raffaella Greco; Tobias Alexander; Joachim Burman; Nicoletta Del Papa; Jeska de Vries-Bouwstra; Dominique Farge; Jörg Henes; Majid Kazmi; Kirill Kirgizov; Paolo A Muraro; Elena Ricart; Montserrat Rovira; Riccardo Saccardi; Basil Sharrack; Emilian Snarski; Barbara Withers; Helen Jessop; Claudia Boglione; Ellen Kramer; Manuela Badoglio; Myriam Labopin; Kim Orchard; Selim Corbacioglu; Per Ljungman; Malgorzata Mikulska; Rafael De la Camara; John A Snowden Journal: Bone Marrow Transplant Date: 2021-05-24 Impact factor: 5.483