Autologous haematopoietic stem cell transplantation for rheumatic diseases: best practice recommendations from the EBMT Practice Harmonization and Guidelines Committee

Tobias Alexander^{1

2}, Elisa Roldan^{3

4}, Nicoletta Del Papa⁵, Dominique Farge^{6

7}, Jörg Henes⁸, Zora Marjanovic⁹, Mathieu Puyade¹⁰, John A Snowden^{3

4}, Julia Spierings¹¹, Jeska K de Vries-Bouwstra¹², Francesco Onida¹³, Annalisa Ruggeri¹⁴, Isabel Sánchez-Ortega^{15

16}, Richard Burt¹⁷, Ricard Cervera¹⁸, Andrea Doria¹⁹, John Moore²⁰, Maria Carolina Oliveira^{21

22}, Grégory Pugnet²³, Doron Rimar²⁴, Marc Schmalzing²⁵, Ibrahim Yakoub-Agha²⁶, Raffaella Greco²⁷

Affiliations

¹ Department of Rheumatology and Clinical Immunology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany. tobias.alexander@charite.de.
² German Rheumatism Research Centre, a Leibniz Institute, Berlin, Germany. tobias.alexander@charite.de.
³ Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
⁴ Division of Clinical Medicine, School of Medicine and Population Health, The University of Sheffield, Sheffield, UK.
⁵ Scleroderma Clinic, Rheumatology Dept, ASST G. Pini-CTO, Università degli Studi di Milano, Milano, Italy.
⁶ 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, INSERM UMR1342, IRSL, Paris-Cite University, Paris, France.
⁷ Department of Medicine, McGill University, Montreal, QC, Canada.
⁸ University hospital Tuebingen Tübingen, Internal Medicine II (Hematology, Oncology, clinical immunology and rheumatology) Tübingen, Tübingen, Germany.
⁹ Service d'Hématologie Clinique et Thérapie Cellulaire, Hôpital Saint-Antoine, AP-HP, Paris, Sorbonne University, Paris, France.
¹⁰ CHU de Poitiers, Service de Médecine Interne et Maladies Infectieuses, Poitiers, France.
¹¹ Department of Rheumatology and Clinical Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
¹² Department of Rheumatology, Leiden University Medical Center, Leiden, The Netherlands.
¹³ Hematology & ASCT Unit, ASST Fatebenefratelli-Sacco, University of Milan, Milan, Italy.
¹⁴ Hematology and BMT Unit, San Raffaele Scientific Institute, Milano, Italy.
¹⁵ Secretary of the Practice Harmonization and Guidelines committee of EBMT, Barcelona, Spain.
¹⁶ EBMT Medical Officer, Executive Office, Barcelona, Spain.
¹⁷ Northwestern University (retired), Scripps hematology, La Jolla, CA, USA.
¹⁸ Department of Autoimmune Diseases. Reference Centre for Systemic Autoimmune Diseases, Vasculitis and Autoinflammatory Diseases, Member of ERN-ReCONNET/RITA, Hospital Clínic, University of Barcelona, Barcelona, Catalonia, Spain.
¹⁹ Division of Rheumatology, Department of Medicine DIMED, University of Padua, Padua, Italy.
²⁰ Department of Haematology and Bone Marrow Transplantation, St Vincent's Hospital, Sydney, NSW, Australia.
²¹ Center for Cell-based Therapy, Regional Hemotherapy Center of the Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.
²² Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.
²³ Service de Médecine Interne et Immunologie Clinique, CHU Rangueil, Toulouse cedex 9, France.
²⁴ Rheumatology Unit, Bnai Zion Medical Center, Faculty of Medicine, Technion Israel institute of technology, Haifa, Israel.
²⁵ Department of Internal Medicine 2, Rheumatology/Clinical Immunology, University Hospital Würzburg, Würzburg, Germany.
²⁶ CHU de Lille, Univ Lille, INSERM U1286, Infinite, Lille, France.
²⁷ Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy. greco.raffaella@hsr.it.

PMID: 40830237
DOI: 10.1038/s41409-025-02695-y

Autologous haematopoietic stem cell transplantation for rheumatic diseases: best practice recommendations from the EBMT Practice Harmonization and Guidelines Committee

Tobias Alexander et al. Bone Marrow Transplant. 2025.

. 2025 Aug 20.

doi: 10.1038/s41409-025-02695-y. Online ahead of print.

Authors

Affiliations

¹ Department of Rheumatology and Clinical Immunology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Berlin, Germany. tobias.alexander@charite.de.
² German Rheumatism Research Centre, a Leibniz Institute, Berlin, Germany. tobias.alexander@charite.de.
³ Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
⁴ Division of Clinical Medicine, School of Medicine and Population Health, The University of Sheffield, Sheffield, UK.
⁵ Scleroderma Clinic, Rheumatology Dept, ASST G. Pini-CTO, Università degli Studi di Milano, Milano, Italy.
⁶ 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, INSERM UMR1342, IRSL, Paris-Cite University, Paris, France.
⁷ Department of Medicine, McGill University, Montreal, QC, Canada.
⁸ University hospital Tuebingen Tübingen, Internal Medicine II (Hematology, Oncology, clinical immunology and rheumatology) Tübingen, Tübingen, Germany.
⁹ Service d'Hématologie Clinique et Thérapie Cellulaire, Hôpital Saint-Antoine, AP-HP, Paris, Sorbonne University, Paris, France.
¹⁰ CHU de Poitiers, Service de Médecine Interne et Maladies Infectieuses, Poitiers, France.
¹¹ Department of Rheumatology and Clinical Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
¹² Department of Rheumatology, Leiden University Medical Center, Leiden, The Netherlands.
¹³ Hematology & ASCT Unit, ASST Fatebenefratelli-Sacco, University of Milan, Milan, Italy.
¹⁴ Hematology and BMT Unit, San Raffaele Scientific Institute, Milano, Italy.
¹⁵ Secretary of the Practice Harmonization and Guidelines committee of EBMT, Barcelona, Spain.
¹⁶ EBMT Medical Officer, Executive Office, Barcelona, Spain.
¹⁷ Northwestern University (retired), Scripps hematology, La Jolla, CA, USA.
¹⁸ Department of Autoimmune Diseases. Reference Centre for Systemic Autoimmune Diseases, Vasculitis and Autoinflammatory Diseases, Member of ERN-ReCONNET/RITA, Hospital Clínic, University of Barcelona, Barcelona, Catalonia, Spain.
¹⁹ Division of Rheumatology, Department of Medicine DIMED, University of Padua, Padua, Italy.
²⁰ Department of Haematology and Bone Marrow Transplantation, St Vincent's Hospital, Sydney, NSW, Australia.
²¹ Center for Cell-based Therapy, Regional Hemotherapy Center of the Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.
²² Department of Internal Medicine, Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil.
²³ Service de Médecine Interne et Immunologie Clinique, CHU Rangueil, Toulouse cedex 9, France.
²⁴ Rheumatology Unit, Bnai Zion Medical Center, Faculty of Medicine, Technion Israel institute of technology, Haifa, Israel.
²⁵ Department of Internal Medicine 2, Rheumatology/Clinical Immunology, University Hospital Würzburg, Würzburg, Germany.
²⁶ CHU de Lille, Univ Lille, INSERM U1286, Infinite, Lille, France.
²⁷ Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy. greco.raffaella@hsr.it.

PMID: 40830237
DOI: 10.1038/s41409-025-02695-y

Abstract

Immune-mediated rheumatologic and musculoskeletal diseases (RMDs) comprise a heterogeneous group of systemic conditions that affect the connective tissues of the musculoskeletal system and internal organs. Immune-mediated RMDs are driven by chronic autoimmune responses and typically require continuous or repeated administration of immunosuppressive or biologic disease-modifying drugs. Although generally effective, these therapies can cause both short- and long-term side effects and may fail to control the disease with risk of irreversible tissue damage. For such patients, haematopoietic stem cell transplantation (HSCT) has been successfully employed over the past 30 years, but this procedure requires caution due to significant side effects. To address these aspects, updated recommendations for the use of HSCT in RMDs have been developed in collaboration with an international expert panel from the European Society for Blood and Marrow Transplantation (EBMT). The panel reviewed all available evidence regarding HSCT application since 2004. Based on this review, EBMT expert-based consensus recommendations were formulated to guide best practices and ensure high-quality patient care. These recommendations include detailed indications, contraindications, and cautionary notes specific to each RMD, along with comprehensive protocols for diagnostic work-up. They are intended to support clinicians, scientists, patients, and caregivers in the field of RMDs.

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Conflict of interest statement

Competing interests: TA received Honoria from Abbvie, Amgen, AstraZeneca and GSK study support from Johnson & Johnson. RG discloses speaking honoraria from Biotest, Pfizer, Medac, Neovii, Kyverna and Magenta. RB is CEO of Genani Corporation, Chicago, Illinois. FM received honoraria & Travel Support from BMS, Janssen, Kite/Gilead, Miltenyi, Novartis, Received Research Support from Kite/Gilead. JAS discloses consultancy for BMS, Medac, Vertex and Jazz. JH discloses consultancy for Miltenyi and Neovii. JS discloses research support from Boehringer Ingelheim and Miltenyi. RC discloses consultancy for AstraZeneca, Celgene, GSK, Janssen, ElyLilly, Pfizer, UCB, Rubió and Werfen. PA reports no conflict of interest; he discloses travel support and speaker honoraria from unrestricted educational activities organized by Novartis, Bayer HealthCare, Bayer Pharma, Biogen Idec, Merck-Serono and Sanofi Aventis and consulting to Magenta Therapeutics, Jasper Therapeutics and Cellerys AG. ER discloses speaking honoraria by Janssen Pharmaceutica. AR discloses speaker honoraria from Gilead. The remaining authors have nothing to declare. JM discloses speaker honoria from Medac and Pfizer. JVR discloses speaker honoraria from Janssen-Cilag, Boehringer-Ingelheim, Pfizer, AstraZeneca, BMS, UCB; consultancy honoraria from Abbvie, Janssen, Boehringer- Ingelheim, and grant/research support from ReumaNederland, Janssen-Cilag, Galapagos, Roche; all payments were made to the institution. GP reports no conflict of interest; he discloses travel support and speaker honoraria from Abbvie, Astra-Zeneca, Novartis, Amgen, GSK, Pfizer, Boehringer Ingelheim, Amicus. None of the mentioned conflicts of interest were related to financing of the content of this manuscript. Ethical approval and consent to participate: All methods were performed in accordance with the relevant guidelines and regulations. This study was approved by the Autoimmune Diseases Working Party of the EBMT.

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Autologous haematopoietic stem cell transplantation for rheumatic diseases: best practice recommendations from the EBMT Practice Harmonization and Guidelines Committee

Affiliations

Autologous haematopoietic stem cell transplantation for rheumatic diseases: best practice recommendations from the EBMT Practice Harmonization and Guidelines Committee

Authors

Affiliations

Abstract

Conflict of interest statement

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