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- W2047252912 abstract "Multiple sclerosis (MS) is an autoimmune disease that is characterised by a relapsing-remitting clinical course followed, usually 10–15 years after onset, by progressive clinical deterioration—the secondary-progressive phase of the disease. The available therapies—steroids, interferons, glatiramer acetate, natalizumab, and mitoxantrone—are effective mainly in the relapsing-remitting phase. However, as with other autoimmune diseases, some patients do not respond to the approved drugs, and other possible therapeutic options must be assessed. Intense immunosuppression followed by autologous haemopoietic stem cell transplantation has been used in the past 15 years for patients with severe MS. The rationale behind this procedure is the eradication of potential autoreactive clones in the periphery and the target organ by means of the conditioning regimen, followed by complete immune reconstitution by engraftment of autologous haemolymphopoietic stem cells that have previously been mobilised from the patient's bone marrow. Several phase I and II studies have been published that use diverse conditioning regimens and different inclusion criteria, and these report data on toxicity and the efficacy of the procedure on the basis of MRI findings and clinical outcome. 1 Mancardi G Saccardi R Autologous haematopoietic stem-cell transplantation in multiple sclerosis. Lancet Neurol. 2008; 7: 626-636 Summary Full Text Full Text PDF PubMed Scopus (170) Google Scholar However, the efficacy of autologous haemopoietic stem cell transplantation has not yet been shown because no phase III prospective randomised study with a primary clinical endpoint has been done. The main problem with autologous haemopoietic stem cell transplantation is transplantation-related mortality, which is around 3·3%. 1 Mancardi G Saccardi R Autologous haematopoietic stem-cell transplantation in multiple sclerosis. Lancet Neurol. 2008; 7: 626-636 Summary Full Text Full Text PDF PubMed Scopus (170) Google Scholar Although the mortality risk has decreased in recent years, 1 Mancardi G Saccardi R Autologous haematopoietic stem-cell transplantation in multiple sclerosis. Lancet Neurol. 2008; 7: 626-636 Summary Full Text Full Text PDF PubMed Scopus (170) Google Scholar it is still too high for a disorder which, at least in the short term, is not life threatening per se. High-intensity conditioning regimens have a high mortality risk; 1 Mancardi G Saccardi R Autologous haematopoietic stem-cell transplantation in multiple sclerosis. Lancet Neurol. 2008; 7: 626-636 Summary Full Text Full Text PDF PubMed Scopus (170) Google Scholar therefore, low-intensity treatments, which could be as effective as medium-intensity or high-intensity regimens for immunosuppression but have minor myeloablative effects, are now under investigation. Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II studyNon-myeloablative autologous haemopoietic stem cell transplantation in patients with relapsing-remitting MS reverses neurological deficits, but these results need to be confirmed in a randomised trial. Full-Text PDF" @default.
- W2047252912 created "2016-06-24" @default.
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- W2047252912 date "2009-03-01" @default.
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- W2047252912 title "Further data on autologous haemopoietic stem cell transplantation in multiple sclerosis" @default.
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- W2047252912 doi "https://doi.org/10.1016/s1474-4422(09)70018-3" @default.
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