Department of Rheumatology and Clinical Immunology, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Berlin Institute of Health, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy.
Bone Marrow Transplant. 2022 Jul;57(7):1055-1062. doi: 10.1038/s41409-022-01702-w. Epub 2022 May 16.
Autoimmune diseases (ADs) represent a heterogenous group of complex diseases with increasing incidence in Western countries and are a major cause of morbidity. Hematopoietic stem cell transplantation (HSCT) has evolved over the last 25 years as a specific treatment for patients with severe ADs, through eradication of the pathogenic immunologic memory and profound immune renewal. HSCT for ADs is recently facing a unique developmental phase across transplant centers. This review provides a comprehensive overview of the recent evidence and developments in the area, including fundamentals of preclinical research, clinical studies in neurologic, rheumatologic and gastroenterologic diseases, which represent major indications at present, along with evidence of HSCT for rarer indications. Moreover, we describe the interwoven challenges of delivering more advanced cellular therapies, exploiting mesenchymal stem cells, regulatory T cells and potentially CAR-T cell therapies, in patients affected by ADs. Overall, we discuss past and current indications, efficacy, associated risks and benefits, and future directions of HSCT and advanced cellular therapies in the treatment of severe/refractory ADs, integrating the available literature with European Society for Blood and Marrow Transplantation (EBMT) registry data.
自身免疫性疾病（ADs）是一组异质性复杂疾病，在西方国家的发病率不断上升，是发病率的主要原因。造血干细胞移植（HSCT）在过去 25 年中发展成为治疗严重 ADs 患者的一种特殊疗法，通过消除致病性免疫记忆和深刻的免疫更新。最近，ADs 的 HSCT 在移植中心面临着独特的发展阶段。这篇综述提供了该领域最近的证据和发展的全面概述，包括临床前研究的基础、神经、风湿和胃肠疾病的临床研究，这些研究目前是主要的适应症，以及对罕见适应症的 HSCT 的证据。此外，我们描述了在 AD 患者中提供更先进的细胞疗法、利用间充质干细胞、调节性 T 细胞和潜在的 CAR-T 细胞疗法的交织挑战。总体而言，我们讨论了过去和当前的适应症、疗效、相关风险和益处，以及 HSCT 和先进细胞疗法在治疗严重/难治性 ADs 中的未来方向，将现有文献与欧洲血液和骨髓移植学会（EBMT）登记数据相结合。
