Alexander Tobias, Roldan Elisa, Del Papa Nicoletta, Farge Dominique, Henes Jörg, Marjanovic Zora, Puyade Mathieu, Snowden John A, Spierings Julia, de Vries-Bouwstra Jeska K, Onida Francesco, Ruggeri Annalisa, Sánchez-Ortega Isabel, Burt Richard, Cervera Ricard, Doria Andrea, Moore John, Oliveira Maria Carolina, Pugnet Grégory, Rimar Doron, Schmalzing Marc, Yakoub-Agha Ibrahim, Greco Raffaella
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.
German Rheumatism Research Centre, a Leibniz Institute, Berlin, Germany.
Bone Marrow Transplant. 2025 Aug 20. doi: 10.1038/s41409-025-02695-y.
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.
免疫介导的风湿性和肌肉骨骼疾病（RMD）是一组异质性的全身性疾病，会影响肌肉骨骼系统和内脏器官的结缔组织。免疫介导的RMD由慢性自身免疫反应驱动，通常需要持续或反复给予免疫抑制或生物疾病改善药物。尽管这些疗法通常有效，但它们可能会导致短期和长期的副作用，并且可能无法控制疾病，存在不可逆组织损伤的风险。对于此类患者，造血干细胞移植（HSCT）在过去30年中已成功应用，但由于存在显著副作用，该手术需要谨慎进行。为了解决这些问题，与欧洲血液和骨髓移植学会（EBMT）的一个国际专家小组合作，制定了关于在RMD中使用HSCT的最新建议。该小组审查了自2004年以来有关HSCT应用的所有现有证据。基于此审查，制定了基于EBMT专家的共识建议，以指导最佳实践并确保高质量的患者护理。这些建议包括每种RMD特有的详细适应症、禁忌症和注意事项，以及全面的诊断检查方案。它们旨在支持RMD领域的临床医生、科学家、患者和护理人员。
