High-dose immunosuppressive therapy with PBPC support in the treatment of poor risk multiple sclerosis
Tomas Kozak,Eva Havrdova,Pitha J,Evžen Gregora,Robert Pytlik,J Maaloufová,Helena Mareckova,Kobylka P,S Vodvárková +8 more
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TL;DR: PBPC mobilisation was safe with no serious adverse effects, and without significant aggravation of disability, in 11 patients with secondary progressive MS and eight patients were grafted after high-dose BEAM chemotherapy with either in vitro or in vivo T cell depletion.
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Abstract: High-dose immunoablative chemotherapy with autologous haematopoietic cell support might be beneficial in the treatment of intractable forms of MS. We mobilised PBPC in 11 patients with secondary progressive MS and finally eight patients were grafted after high-dose BEAM chemotherapy with either in vitro or in vivo T cell depletion. Median EDSS and SNRS scores at the time of inclusion were 6.5 (6.5-7.5) and 56 (44-65), respectively. PBPC mobilisation was safe with no serious adverse effects, and without significant aggravation of disability. One patient improved significantly (by 1.0 point on EDSS) after the mobilisation. Two mobilisation failures were observed. No life-threatening events occurred during the transplantation. All grafted patients, except one, at least stabilised their disability status. One patient improved significantly (by 1.5 points on EDSS), two patients improved slightly (by 0.5 points on EDSS), one patient worsened by 1.0 point on the EDSS in 10 months. Improvement occurred with a delay of 2-4 months. Median EDSS and SNRS of grafted patients at the last follow up were 6.5 (5.5-8.5) and 64 (39-73), respectively with median follow-up of 8.5 months. Further follow-up is needed to determine the disease course after complete immune reconstitution. Bone Marrow Transplantation (2000) 25, 525-531.
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Citations
Collection of hematopoietic stem cells from patients with autoimmune diseases
Richard K. Burt,Athanasios Fassas,John A. Snowden,John A. Snowden,J.M. van Laar,Tomas Kozak,Nico M Wulffraat,Richard A. Nash,Cynthia E. Dunbar,Renate Arnold,G. Prentice,Sarah J. Bingham,Alberto M. Marmont,Peter A. McSweeney +13 more
TL;DR: When corrected for patient weight and apheresis volume, progenitor cell yields tended to vary by underlying disease, prior medication history and mobilization regimen, and Trends in the approaches to, and results of, progensitor cell mobilization are suggested by this survey.
Hematopoietic Stem Cell Transplantation for Multiple Sclerosis
Athanasios Fassas,Richard K. Burt +1 more
Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial
Richard K. Burt,Roumen Balabanov,Joachim Burman,Basil Sharrack,John A. Snowden,Maria Carolina Oliveira,Jan Fagius,John W. Rose,Flavia Nelson,Amilton Antunes Barreira,Kristina Carlson,Xiaoqiang Han,Daniela A. Moraes,Amy Morgan,Kathleen Quigley,Kimberly Yaung,Regan Buckley,Carri Alldredge,Allison M. Clendenan,Michelle A. Calvario,Jacquelyn Henry,Borko Jovanovic,Irene Helenowski +22 more
TL;DR: In this preliminary study of patients with relapsing-remitting MS, nonmyeloablative HSCT, compared with DMT, resulted in prolonged time to disease progression, with significant differences in time to progression estimated as hazard ratios.
Haemopoietic stem cell transplantation in autoimmune diseases: a European perspective.
TL;DR: A comprehensive update on the efficacy and toxicity of HSCT in severe autoimmune disease is provided and future directions in the context of other evolving therapies are discussed.
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.
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Intermittent cyclophosphamide pulse therapy in progressive multiple sclerosis Final report of the Northeast Cooperative Multiple Sclerosis Treatment Group
Howard L. Weiner,G. A. Mackin,E. J. Orav,David A. Hafler,David M. Dawson,Y. LaPierre,Robert M. Herndon,James R. Lehrich,Stephen L. Hauser,A. Turel,Marc Fisher,G. Birnbaum,J. McArthur,R. Butler,M. Moore,B. Sigsbee,A. Safran +16 more
TL;DR: The findings support a role for immunosuppression in the treatment of MS, begin to identify variables that may explain differences between studies of immunOSuppression with cyclophosphamide in progressive MS, and suggest that intermittent pulse therapy is an important method for the Treatment of progressive MS and perhaps for earlier stages of MS as well.
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