Journal ArticleDOI
Autologous stem cell transplantation for progressive multiple sclerosis: update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database.
Riccardo Saccardi,Tomas Kozak,Chiara Bocelli-Tyndall,A Fassas,Aristidis Kazis,Eva Havrdova,Enric Carreras,Albert Saiz,Bob Löwenberg,P Aw te Boekhorst,Francesca Gualandi,Harry Openshaw,G Longo,F Pagliai,Luca Massacesi,E Deconink,J Ouyang,F Jz Nagore,Juan Besalduch,I. Lisukov,A Bonini,Elisa Merelli,Shimon Slavin,Alois Gratwohl,Jakob Passweg,Alan Tyndall,Andreas J. Steck,M Andolina,Marco Capobianco,J Ld Martin,Alessandra Lugaresi,Giuseppe Meucci,Ricardo Saez,Richard E. Clark,Mario Fernandez,L. Fouillard,B. Herstenstein,Vladimir Koza,Eleonora Cocco,H Baurmann,G. L. Mancardi +40 more
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TLDR
HSCT was shown as a promising procedure to slow down progression in a subset of patients affected by severe, progressive MS; the safety and feasibility of the procedure can be significantly improved by appropriate patient selection and choice of transplant regimen.Abstract:
Over the last decade, hematopoietic stem cells transplantation (HSCT) has been increasingly used in the treatment of severe progressive autoimmune diseases. We report a retrospective survey of 183 multiple sclerosis (MS) patients, recorded in the database of the European Blood and Marrow Transplantation Group (EBMT). Transplant data were available from 178 patients who received an autologous graft. Overall, transplant related mortality (TRM) was 5.3% and was restricted to the period 1995-2000, with no further TRM reported since then. Busulphan-based regimens were significantly associated with TRM. Clinical status at the time of transplant and transplant techniques showed some correlations with toxicity. No toxic deaths were reported among the 53 patients treated with the BEAM (carmustine, etoposide, cytosine-arabinoside, melphalan)/antithymocyte globulin (ATG) regimen without graft manipulation, irrespective of their clinical condition at the time of the transplant. Improvement or stabilization of neurological conditions occurred in 63% of patients at a median follow-up of 41.7 months, and was not associated with the intensity of the conditioning regimen. In this large series, HSCT was shown as a promising procedure to slow down progression in a subset of patients affected by severe, progressive MS; the safety and feasibility of the procedure can be significantly improved by appropriate patient selection and choice of transplant regimen.read more
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Journal ArticleDOI
Multiple sclerosis: a complicated picture of autoimmunity.
Henry F. McFarland,Roland Martin +1 more
TL;DR: Recent findings obtained with both animal models and patients with multiple sclerosis indicate involvement of a T helper cell with a TH-17 phenotype, in contrast to previous data indicating that T helper type 1 cells are critical.
Journal ArticleDOI
Primary-progressive multiple sclerosis
TL;DR: MRI of the brain and spinal cord, and examination of the CSF, are important investigations for diagnosis; conventional immunomodulatory therapies, such as interferon beta and glatiramer acetate, are ineffective.
Journal ArticleDOI
Mesenchymal stem cells for the treatment of neurodegenerative disease
TL;DR: MSCs transplanted into the brain have been demonstrated to promote functional recovery by producing trophic factors that induce survival and regeneration of host neurons, and proposed regenerative approaches to neurological diseases using MSCs include cell therapies in which cells are delivered via intracerebral or intrathecal injection.
Journal ArticleDOI
Clinical Applications of Blood-Derived and Marrow-Derived Stem Cells for Nonmalignant Diseases
Richard K. Burt,Yvonne Loh,William H. Pearce,Nirat Beohar,Walter G. Barr,Robert M. Craig,Yanting Wen,Jonathan A. Rapp,John A. Kessler +8 more
TL;DR: Stem cells harvested from blood or marrow, whether administered as purified HSCs or mesenchymal stem cells or as an unmanipulated or unpurified product can provide disease-ameliorating effects in some autoimmune diseases and cardiovascular disorders.
Journal ArticleDOI
Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years’ experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases
Dominique Farge,Myriam Labopin,Alan Tyndall,Athanasios Fassas,Gianluigi Mancardi,Jaap Van Laar,Jian Ouyang,Tomas Kozak,John Moore,Ina Kötter,Virginie Chesnel,Alberto M. Marmont,Alois Gratwohl,Riccardo Saccardi +13 more
TL;DR: This largest cohort studied worldwide shows that autologous hematopoietic stem cell transplantation can induce sustained remissions for more than 5 years in patients with severe autoimmune diseases refractory to conventional therapy.
References
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Journal ArticleDOI
Axonal transection in the lesions of multiple sclerosis.
TL;DR: Transected axons are common in the lesions of multiple sclerosis, and axonal transection may be the pathologic correlate of the irreversible neurologic impairment in this disease.
Journal ArticleDOI
Mitoxantrone in progressive multiple sclerosis: a placebo-controlled, double-blind, randomised, multicentre trial.
Hans-Peter Hartung,Richard Gonsette,Nikolaus Konig,Hubert Kwiecinski,Andreas Guseo,Sean P. Morrissey,Hilmar Krapf,Thomas Zwingers +7 more
TL;DR: Mitoxantrone 12 mg/m(2) was generally well tolerated and reduced progression of disability and clinical exacerbations and the frequency of long-term drug-related side-effects.
Journal ArticleDOI
Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients
Paolo A. Muraro,Daniel C. Douek,Amy N. Packer,Katherine Chung,Francisco J. Guenaga,Riccardo Cassiani-Ingoni,Catherine Campbell,Sarfraz Memon,James W. Nagle,Frances T. Hakim,Ronald E. Gress,Henry F. McFarland,Richard K. Burt,Roland Martin +13 more
TL;DR: These data are the first to demonstrate that long-term suppression of inflammatory activity in MS patients who received HSCT does not depend on persisting lymphopenia and is associated with profound qualitative immunological changes that demonstrate a de novo regeneration of the T cell compartment.
Journal ArticleDOI
Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: first results of a pilot study
A Fassas,Achilles Anagnostopoulos,Aristidis Kazis,K. Kapinas,Ioanna Sakellari,V K Kimiskidis,Aliki Tsompanakou +6 more
TL;DR: Autologous HSCT appears feasible in MS; it does not aggravate disability and seems to offer a clinical benefit, however, these observations need confirmation and long-term outcomes will show if benefits counterbalance toxicity and cost.
Journal ArticleDOI
Hematopoietic stem cell transplantation for multiple sclerosis. A retrospective multicenter study.
A Fassas,Jakob Passweg,Achilles Anagnostopoulos,Aristidis Kazis,Tomas Kozak,Eva Havrdova,Enric Carreras,Francesc Graus,Ashwin Kashyap,Harry Openshaw,M. Schipperus,Eric Deconinck,G. L. Mancardi,Alberto M. Marmont,J. Hansz,Marco Rabusin,F. J. Zuazu Nagore,J. Besalduch,T. Dentamaro,L. Fouillard,Bernd Hertenstein,G. La Nasa,Marco Musso,Federico Papineschi,J. M. Rowe,Riccardo Saccardi,Andreas J. Steck,Ludwig Kappos,Alois Gratwohl,Alan Tyndall,Marrow Transplantation +30 more
TL;DR: Autologous HSCT suggest positive early results in the management of progressive MS and is feasible and is being utilised in the planning of future trials to reduce transplant related mortality.
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