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- Daikeler, Thomas, et al.
(author)
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Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: a retrospective study of the EBMT Autoimmune Disease Working Party.
- 2011
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In: Blood. - : American Society of Hematology. - 1528-0020 .- 0006-4971. ; Aug 11:118(6), s. 1693-8
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Journal article (peer-reviewed)abstract
- To specify incidence and risk factors for secondary autoimmune diseases (AD) after HSCT for a primary AD, we retrospectively analysed AD patients treated by HSCT reported to EBMT from 1995 to 2009 with at least one secondary AD (cases) and those without (controls). After autologous HSCT, 29 amongst 347 patients developed at least one secondary AD within 21.9 (0.6-49) months and after allogeneic HSCT, 3 amongst 16 patients. The observed secondary AD included: autoimmune hemolytic anemia (n=3), acquired haemophilia (n=3), autoimmune thrombocytopenia (n=3), antiphospholipid syndrome (n=2), thyroiditis (n=12), blocking TSHR-ab (n=1), Graves' disease (n=2), myasthenia gravis (n=1), rheumatoid arthritis (n=2), sarcoidosis (n=2), vasculitis (n=1), psoriasis (n=1) and psoriatic arthritis (n=1). After autologous HSCT for primary AD, the cumulative incidence of secondary AD was 9.8+/-2 % at 5 years, lupus erythematosus as primary AD and antithymocyte-globulin use plus CD34+ graft selection were important risk factors for secondary AD by multivariate analysis. With a median follow-up of 6.2 (0.54-11) years after autologous HSCT, 26/29 patients with secondary AD were alive, 2 died during their secondary AD (antiphospholipid syndrome, haemophilia) and 1 death was HSCT related. This European multicenter study underlines the need for careful management and follow-up for secondary AD after HSCT.
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2. |
- Greco, Raffaella, et al.
(author)
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Autologous hematopoietic stem cell transplantation in neuromyelitis optica : A registry study of the EBMT Autoimmune Diseases Working Party
- 2015
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In: Multiple Sclerosis Journal. - : SAGE Publications. - 1352-4585 .- 1477-0970. ; 21:2, s. 189-197
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Journal article (peer-reviewed)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.
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