EClinicalMedicine (Mar 2024)
Innovative cellular therapies for autoimmune diseases: expert-based position statement and clinical practice recommendations from the EBMT practice harmonization and guidelines committeeResearch in context
- Raffaella Greco,
- Tobias Alexander,
- Nicoletta Del Papa,
- Fabian Müller,
- Riccardo Saccardi,
- Fermin Sanchez-Guijo,
- Georg Schett,
- Basil Sharrack,
- John A. Snowden,
- Karin Tarte,
- Francesco Onida,
- Isabel Sánchez-Ortega,
- Joachim Burman,
- Cristina Castilla Llorente,
- Ricard Cervera,
- Fabio Ciceri,
- Andrea Doria,
- Jörg Henes,
- James Lindsay,
- Andreas Mackensen,
- Paolo A. Muraro,
- Elena Ricart,
- Montserrat Rovira,
- Tsila Zuckerman,
- Ibrahim Yakoub-Agha,
- Dominique Farge
Affiliations
- Raffaella Greco
- Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Hospital, Vita-Salute San Raffaele University, Milan, Italy; Co-Chair of the Practice Harmonization and Guidelines Committee of EBMT and Chair of the ADWP of the EBMT, Barcelona, Spain; Corresponding author. Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Vita-Salute San Raffaele University, via Olgettina 60, 20132, Milano, Italy.
- Tobias Alexander
- Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Berlin Institute of Health, Department of Rheumatology and Clinical Immunology, Berlin, Germany
- Nicoletta Del Papa
- Scleroderma Clinic, Rheumatology Department, ASST G. Pini-CTO, Università degli Studi di Milano, Milano, Italy
- Fabian Müller
- Department of Internal Medicine 5 - Hematology and Oncology, University Hospital of Erlangen, Erlangen, Germany; Bayrisches Zentrum für Krebsforschung (BZKF) Erlangen, Germany
- Riccardo Saccardi
- Cellular Therapies and Transfusion Medicine Unit, Careggi University Hospital, Florence, Italy
- Fermin Sanchez-Guijo
- Department of Hematology, IBSAL-University Hospital of Salamanca and Department of Medicine, University of Salamanca, Salamanca, Spain
- Georg Schett
- Department of Internal Medicine 3 - Rheumatology and Immunology, FAU Erlangen-Nürnberg and Universitätsklinikum Erlangen, Erlangen, Germany; Deutsches Zentrum Immuntherapie, Universitätsklinikum Erlangen, Friedrich-Alexander University (FAU) Erlangen- Nürnberg, Erlangen, Germany
- Basil Sharrack
- Department of Neuroscience and Sheffield NIHR Translational Neuroscience BRC, Sheffield Teaching Hospitals NHS Foundation Trust & University of Sheffield, Sheffield, England, United Kingdom
- John A. Snowden
- Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Division of Clinical Medicine, School of Medicine and Population Health, The University of Sheffield, Sheffield, UK
- Karin Tarte
- SITI Lab, CHU Rennes, EFS Bretagne, University Rennes, Rennes, France
- Francesco Onida
- Hematology & ASCT Unit, ASST Fatebenefratelli-Sacco, University of Milan, Italy; Co-Chair of the Practice Harmonization and Guidelines Committee of EBMT, Spain
- Isabel Sánchez-Ortega
- Secretary of the Practice Harmonization and Guidelines Committee of EBMT, Barcelona, Spain; EBMT Medical Officer, Executive Office, Barcelona, Spain
- Joachim Burman
- Department of Medical Sciences, Uppsala University, Uppsala, Sweden
- Cristina Castilla Llorente
- Hematologie Department, Gustave Roussy, Villejuif, France
- Ricard Cervera
- Department of Autoimmune Diseases, Reference Centre for Systemic Autoimmune Diseases (UEC, CSUR) of the Catalan and Spanish Health Systems/Member of ERN-ReCONNET, Hospital Clínic, Barcelona, Catalonia, Spain
- Fabio Ciceri
- Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Hospital, Vita-Salute San Raffaele University, Milan, Italy
- Andrea Doria
- Rheumatology Unit, Department of Medicine (DiMED), University of Padua, Padua, Italy
- Jörg Henes
- Center for Interdisciplinary Rheumatology, Immunology and Autoimmune diseases and Department of Internal Medicine II (Haematology, Oncology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, Germany
- James Lindsay
- Department of Gastroenterology, The Royal London Hospital, Barts Health NHS Trust, London, UK; Centre for Immunobiology, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK
- Andreas Mackensen
- Department of Internal Medicine 5 - Hematology and Oncology, University Hospital of Erlangen, Erlangen, Germany; Bayrisches Zentrum für Krebsforschung (BZKF) Erlangen, Germany
- Paolo A. Muraro
- Department of Brain Sciences, Imperial College London, London, UK
- Elena Ricart
- Gastroenterology Department. Hospital Clínic Barcelona. Institut d’Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS). Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEREHD), Barcelona, Spain
- Montserrat Rovira
- BMT Unit, Haematology Department, Institute of Haematology and Oncology, IDIBAPS, Hospital Clinic, University of Barcelona, Barcelona, Spain; Josep Carreras Leukaemia Research Foundation, Spain
- Tsila Zuckerman
- Rambam Health Care Campus and Rappaport Faculty of Medicine, Technion, Haifa, Israel
- Ibrahim Yakoub-Agha
- CHU de Lille, University Lille, INSERM U1286, Infinite, 59000, Lille, France; Chair of the Practice Harmonization and Guidelines Committee of EBMT, Spain
- Dominique Farge
- Internal Medicine Unit (04): CRMR MATHEC, Maladies Auto-immunes et Thérapie Cellulaire, Centre de Référence des Maladies auto-immunes systémiques Rares d’Ile-de-France, AP-HP, St-Louis Hospital Paris-Cite University, France; Department of Medicine, McGill University, Montreal, QC, Canada; Corresponding author. Internal Medicine Unit (04): CRMR MATHEC, Maladies Auto-immunes et Thérapie Cellulaire, AP-HP, St-Louis Hospital, 1 avenue Claude Vellefaux, 75010, Paris, France.
- Journal volume & issue
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Vol. 69
p. 102476
Abstract
Summary: Autoimmune diseases (ADs) are characterized by loss of immune tolerance, high chronicity, with substantial morbidity and mortality, despite conventional immunosuppression (IS) or targeted disease modifying therapies (DMTs), which usually require repeated administration. Recently, novel cellular therapies (CT), including mesenchymal stromal cells (MSC), Chimeric Antigen Receptors T cells (CART) and regulatory T cells (Tregs), have been successfully adopted in ADs. An international expert panel of the European Society for Blood and Marrow Transplantation and the International Society for the Cell and Gene Therapy, reviewed all available evidence, based on the current literature and expert practices, on use of MSC, CART and Tregs, in AD patients with rheumatological, neurological, and gastroenterological indications. Expert-based consensus and recommendations for best practice and quality of patient care were developed to support clinicians, scientists, and their multidisciplinary teams, as well as patients and care providers and will be regularly updated.
