1
views
0
recommends
+1 Recommend
0 collections
    0
    shares
      • Record: found
      • Abstract: found
      • Article: found
      Is Open Access

      Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE)

      1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 , 11 , 12 , 13 , 14 , 5 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , , 12 , for the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and EBMT (JACIE)

      Bone Marrow Transplantation

      Nature Publishing Group UK

      Peripheral neuropathies, Bone marrow transplantation

      Read this article at

      Bookmark
          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

          Abstract

          These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

          Related collections

          Most cited references 123

          • Record: found
          • Abstract: found
          • Article: not found

          Evaluation of no evidence of disease activity in a 7-year longitudinal multiple sclerosis cohort.

          With multiple and increasingly effective therapies for relapsing forms of multiple sclerosis (MS), disease-free status or no evidence of disease activity (NEDA) has become a treatment goal and a new outcome measure. However, the persistence of NEDA over time and its predictive power for long-term prognosis are unknown.
            Bookmark
            • Record: found
            • Abstract: found
            • Article: not found

            Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients

            Clinical trials have indicated that autologous hematopoietic stem cell transplantation (HSCT) can persistently suppress inflammatory disease activity in a subset of patients with severe multiple sclerosis (MS), but the mechanism has remained unclear. To understand whether the beneficial effects on the course of disease are mediated by lympho-depletive effects alone or are sustained by a regeneration of the immune repertoire, we examined the long-term immune reconstitution in patients with MS who received HSCT. After numeric recovery of leukocytes, at 2-yr follow-up there was on average a doubling of the frequency of naive CD4+ T cells at the expense of memory T cells. Phenotypic and T cell receptor excision circle (TREC) analysis confirmed a recent thymic origin of the expanded naive T cell subset. Analysis of the T cell receptor repertoire showed the reconstitution of an overall broader clonal diversity and an extensive renewal of clonal specificities compared with pretherapy. 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.
              Bookmark
              • Record: found
              • Abstract: found
              • Article: not found

              Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial

              Strong immunosuppression, including chemotherapy and immune-depleting antibodies followed by autologous haemopoietic stem-cell transplantation (aHSCT), has been used to treat patients with multiple sclerosis, improving control of relapsing disease. We addressed whether near-complete immunoablation followed by immune cell depleted aHSCT would result in long-term control of multiple sclerosis.
                Bookmark

                Author and article information

                Contributors
                john.snowden1@nhs.net
                Journal
                Bone Marrow Transplant
                Bone Marrow Transplant
                Bone Marrow Transplantation
                Nature Publishing Group UK (London )
                0268-3369
                1476-5365
                26 September 2019
                26 September 2019
                2020
                : 55
                : 2
                : 283-306
                Affiliations
                [1 ]ISNI 0000 0000 9422 8284, GRID grid.31410.37, Department of Neurology, , Sheffield Teaching Hospitals NHS Foundation Trust, ; Sheffield, UK
                [2 ]ISNI 0000 0004 1936 9262, GRID grid.11835.3e, NIHR Neurosciences Biomedical Research Centre, , University of Sheffield, ; Sheffield, UK
                [3 ]ISNI 0000 0004 1759 9494, GRID grid.24704.35, Cell Therapy and Transfusion Medicine Unit, , Careggi University Hospital, ; Firenze, Italy
                [4 ]ISNI 0000 0001 2218 4662, GRID grid.6363.0, Klinik fur Rheumatologie und Klinische Immunologie, , Charite-Universitatsmedizin, ; Berlin, Germany
                [5 ]ISNI 0000 0001 2308 1657, GRID grid.462844.8, EBMT Paris study office, Department of Haematology, Saint Antoine Hospital, INSERM UMR 938, , Sorbonne University, ; Paris, France
                [6 ]ISNI 0000 0004 1936 9457, GRID grid.8993.b, Department of Neuroscience, , Uppsala University, ; Uppsala, Sweden
                [7 ]ISNI 0000 0001 2300 6614, GRID grid.413328.f, Unité de Médecine Interne, Maladies Auto-immunes et Pathologie Vasculaire (UF 04), , Hôpital St-Louis, AP-HP, ; Paris, France
                [8 ]Centre de Référence des Maladies Auto-Immunes Systémiques Rares d’Ile-de-France, Filière, FAI2R Paris, France
                [9 ]ISNI 0000 0001 2217 0017, GRID grid.7452.4, EA 3518, Université Denis Diderot, ; Paris, France
                [10 ]ISNI 0000 0004 1936 8649, GRID grid.14709.3b, Department of Internal Medicine, , McGill University, ; Montreal, QC Canada
                [11 ]ISNI 0000000417581884, GRID grid.18887.3e, Hematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico, , San Raffaele Scientific Institute, ; Milan, Italy
                [12 ]ISNI 0000 0000 9422 8284, GRID grid.31410.37, Department of Haematology, , Sheffield Teaching Hospitals NHS Foundation Trust, ; Sheffield, UK
                [13 ]GRID grid.239826.4, Kings Health Partners, Department of Haematology, , Guys Hospital, ; London, UK
                [14 ]N.N. Blokhin National Medical Center of Oncology, Institute of Pediatric Oncology and Hematology, Moscow, Russia
                [15 ]ISNI 0000 0001 2151 3065, GRID grid.5606.5, Department of Neuroscience, , University of Genova and Clinical Scientific Institutes Maugeri, ; Genoa, Italy
                [16 ]ISNI 0000 0004 0478 9977, GRID grid.412004.3, Neuroimmunology and MS Research, Neurology Clinic, , University Hospital, ; Zurich, Switzerland
                [17 ]Haematology Department, St. Vincent’s Health Network, Darlinghurst, NSW Australia
                [18 ]ISNI 0000 0001 2113 8111, GRID grid.7445.2, Department of Brain Sciences, , Imperial College London, ; London, UK
                [19 ]BMT Unit, Department of Hematology, IDIBAPS, Hospital Clinic, Institut Josep Carreras, Barcelona, Spain
                [20 ]ISNI 0000 0001 2151 3065, GRID grid.5606.5, Department of Health Sciences (DISSAL), , University of Genoa, ; Genoa, Italy
                [21 ]IRCCS Ospedale Policlinico San Martino, Genoa, Italy
                [22 ]ISNI 0000 0000 9422 8284, GRID grid.31410.37, Department of Haematology, , Sheffield Teaching Hospitals NHS Foundation Trust, ; Sheffield, UK
                [23 ]ISNI 0000 0004 1759 9494, GRID grid.24704.35, Haematology Department, , Careggi University Hospital, ; Florence, Italy
                [24 ]EBMT Executive Office, Barcelona, Spain
                [25 ]ISNI 0000 0004 1757 8562, GRID grid.413181.e, Ospedale Pediatrico, , Azienda Ospedaliero Universitaria Meyer (A.O.U. Meyer), ; Florence, Italy
                [26 ]GRID grid.411258.b, IBSAL, , Hospital Universitario de Salamanca, ; Salamanca, Spain
                [27 ]ISNI 0000 0000 9259 8492, GRID grid.22937.3d, Blood Group Serology and Transfusion Medicine, , Medical University of Vienna, ; Vienna, Austria
                [28 ]ISNI 0000 0000 9422 8284, GRID grid.31410.37, Department of Haematology, , Sheffield Teaching Hospitals NHS Foundation Trust, ; Sheffield, UK
                [29 ]ISNI 0000 0001 2218 4662, GRID grid.6363.0, Klinik fur Rheumatologie und Klinische Immunologie, , Charite-Universitatsmedizin, ; Berlin, Germany
                [30 ]ISNI 0000 0001 0462 7212, GRID grid.1006.7, Great North Children’s Hospital, Institute of Cellular Medicine, , Newcastle University, ; Newcastle upon Tyne, UK
                [31 ]ISNI 0000 0001 2218 4662, GRID grid.6363.0, Department of Haematology, Oncology and Tumor Immunology, , Charité Universitätsmedizin Berlin, ; Berlin, Germany
                [32 ]ISNI 0000 0001 0440 1440, GRID grid.410556.3, Department of Haematology, , Oxford University Hospitals NHS Foundation Trust, ; Oxford, UK
                [33 ]ISNI 0000 0001 2284 9388, GRID grid.14925.3b, Unité de transplantation des cellules souches, , Département d’Hématologie Gustave Roussy, ; Villejuif, France
                [34 ]ISNI 0000 0001 2113 8111, GRID grid.7445.2, Department of Haematology, Hammersmith Hospital, , Imperial College Health Care NHS Trust, ; London, UK
                [35 ]University and University Hospital of Basel, Department of Rheumatology, Basel, Switzerland
                [36 ]Osp. G. Pini, Department of Rheumatology, Milan, Italy
                [37 ]ISNI 0000 0001 2300 6614, GRID grid.413328.f, Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Hôpital St-Louis, AP-HP, ; Paris, France
                [38 ]Centre de Référence des Maladies Auto-Immunes Systémiques Rares d’Ile-de-France, Filière FAI2R, Paris, France
                [39 ]ISNI 0000 0001 2217 0017, GRID grid.7452.4, EA 3518, Université Denis Diderot, ; Paris, France
                [40 ]GRID grid.5963.9, Department of Medicine-Hematology and Oncology, , University of Freiburg, ; Freiburg, Germany
                [41 ]ISNI 0000000417581884, GRID grid.18887.3e, Hematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico, , San Raffaele Scientific Institute, ; Milan, Italy
                [42 ]ISNI 0000 0001 2351 3333, GRID grid.412354.5, Department of Hematology, , Uppsala University Hospital, ; Uppsala, Sweden
                [43 ]ISNI 0000 0001 0196 8249, GRID grid.411544.1, Centre for Interdisciplinary Clinical Immunology, Rheumatology and Auto-inflammatory Diseases, , University Hospital Tuebingen, Department of Internal Medicine II (Oncology, Haematology, Immunology, Rheumatology, Pulmonology), ; Tuebingen, Germany
                [44 ]ISNI 0000 0001 2218 4662, GRID grid.6363.0, Klinik fur Rheumatologie und Klinische Immunologie, , Charite-Universitatsmedizin, ; Berlin, Germany
                [45 ]ISNI 0000 0000 9422 8284, GRID grid.31410.37, Department of Haematology, , Sheffield Teaching Hospitals NHS Foundation Trust, ; Sheffield, UK
                [46 ]ISNI 0000 0000 9422 8284, GRID grid.31410.37, Sheffield Pulmonary Vascular Disease Unit, , Sheffield Teaching Hospitals NHS Foundation Trust, ; Sheffield, UK
                [47 ]GRID grid.239826.4, Kings Health Partners, Department of Haematology, , Guys Hospital, ; London, UK
                [48 ]N.N. Blokhin National Medical Center of Oncology, Institute of Pediatric Oncology and Hematology, Moscow, Russia
                [49 ]ISNI 0000 0004 1937 1100, GRID grid.412370.3, Department of Haematology, , Saint Antoine Hospital, ; Paris, France
                [50 ]Neuroimmunology and MS Research, Neurology Clinic, University Hospital, Zurich, Switzerland
                [51 ]ISNI 0000 0001 2177 138X, GRID grid.412220.7, Department of Clinical Immunology, National Referral Center for Autoimmune Diseases, , Strasbourg University Hospital, ; Strasbourg, France
                [52 ]Haematology Department, St. Vincent’s Health Network, Darlinghurst, NSW Australia
                [53 ]Haematology Department, St. Vincent’s Health Network, Darlinghurst, NSW Australia
                [54 ]ISNI 0000 0004 0623 6380, GRID grid.426412.7, Anthony Nolan Research Institute, ; London, UK
                [55 ]ISNI 0000 0001 2113 8111, GRID grid.7445.2, Department of Brain Sciences, Imperial College London, ; London, UK
                [56 ]ISNI 0000 0004 1937 0722, GRID grid.11899.38, Division of Clinical Immunology, Ribeirão Preto Medical School, , University of São Paulo, ; Ribeirão Preto, Brazil
                [57 ]Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology, Hematology and Transplantation First State Pavlov Medical University of Saint Petersburg, Saint Petersburg, Russia 197022
                [58 ]ISNI 0000 0004 1757 2822, GRID grid.4708.b, Haematology—BMT Centre, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, , Università degli Studi di Milano, ; Milan, Italy
                [59 ]ISNI 0000 0004 1937 0722, GRID grid.11899.38, Division of Hematology, Ribeirão Preto Medical School, , University of São Paulo, ; Ribeirão Preto, Brazil
                [60 ]ISNI 0000 0000 9336 4276, GRID grid.411162.1, Centre Hospitalier Universitaire de Poitiers, ; Poitiers, France
                [61 ]GRID grid.460112.0, University Hospital St. Marina, ; 9010 Varna, Bulgaria
                [62 ]BMT Unit, Department of Hematology, IDIBAPS, Hospital Clinic, Institut Josep Carreras, Barcelona, Spain
                [63 ]ISNI 0000 0004 0641 2823, GRID grid.419319.7, Department of Clinical Haematology, , Manchester Royal Infirmary, ; Manchester, UK
                [64 ]GRID grid.414012.2, Bone Marrow Transplantation Unit, , George Papanicolaou General Hospital, ; Thessaloniki, Greece
                [65 ]ISNI 0000000113287408, GRID grid.13339.3b, Department of Hematology, Oncology and Internal Medicine, , Medical University of Warsaw, ; Warsaw, Poland
                [66 ]ISNI 0000000089452978, GRID grid.10419.3d, Department of Rheumatology, , Leiden University Medical Centre, ; Leiden, Netherlands
                [67 ]GRID grid.477259.a, Division of Hematology and Hematopoietic Stem Cell Transplantation, , Hematology and Stem Cell Transplantation Unit, Clinica FOSCAL, ; Bucaramanga, Colombia
                [68 ]ISNI 0000 0004 0620 3132, GRID grid.417100.3, Divisie Kinderen, Cluster Immunologie, Reumatologie, Hematologie en Infectiologie, , Wilhelmina Kinderziekenhuis, ; Utrecht, Netherlands
                [69 ]Osp. G. Pini, Department of Rheumatology, Milan, Italy
                Article
                684
                10.1038/s41409-019-0684-0
                6995781
                31558790
                © Springer Nature Limited 2019

                Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

                Categories
                Feature
                Custom metadata
                © Springer Nature Limited 2020

                Transplantation

                peripheral neuropathies, bone marrow transplantation

                Comments

                Comment on this article