Hematology and Bone Marrow Transplant Unit, IRCCS San Raffaele Hospital, Milan, Italy; Universitá Vita-Salute San Raffaele, Milan, Italy.
Department of Haematology, Saint Antoine Hospital, INSERM UMR 938, Université Pierre et Marie Curie, Paris, France.
Handb Clin Neurol. 2024;202:295-305. doi: 10.1016/B978-0-323-90242-7.00008-0.
Hematopoietic stem cell transplantation (HSCT) has evolved over the last 25 years as a specific treatment of patients with severe neurologic autoimmune diseases (ADs), through eradication of the pathologic, immunologic memory, and profound immune "resetting." HSCT for ADs is recently facing a unique developmental phase across transplant centers. Data from patients undergoing HSCT and cellular therapies have been captured through the established major transplant registries, such as the European Society for Blood and Marrow Transplantation (EBMT) and the Center for International Blood and Marrow Transplant Research (CIBMTR). The EBMT Autoimmune Diseases Working Party (ADWP) is central to bringing together HSCT and disease-specialist communities. The AD section of the EBMT registry is the largest database of its kind worldwide, reporting more than 3700 transplants. Multiple sclerosis (MS) covers approximately 50% of transplants in AD, HSCT being an integral and standard-of-care part of the treatment algorithm. In the Americas, at least a subset of HSCT is reported to the CIBMTR, as reporting is voluntary. A total of 1400 recipients of autologous HSCT were reported and 1030 were performed for the treatment of neurologic conditions. MS accounts for 96% of all diagnoses among neurologic indications for HSCT. Although the activity of HSCT for MS is low in the United States in relation to its prevalence, the number of transplants has increased in recent years. In contrast, Mexico has reported a sharp increase in the number of these transplants. This chapter provides an overview of the EBMT and CIBMTR registries, then offers the current status and publication outputs in relation to neurologic AD.
造血干细胞移植(HSCT)在过去的 25 年中发展成为治疗严重神经自身免疫性疾病(AD)的特定方法,通过消除病理、免疫记忆和深刻的免疫“重置”。AD 的 HSCT 最近在移植中心面临着独特的发展阶段。接受 HSCT 和细胞治疗的患者的数据已通过已建立的主要移植登记处(如欧洲血液和骨髓移植学会(EBMT)和国际血液和骨髓移植研究中心(CIBMTR))捕获。EBMT 自身免疫疾病工作组(ADWP)是将 HSCT 和疾病专家社区聚集在一起的核心。EBMT 登记处的 AD 部分是全球最大的同类数据库,报告了超过 3700 例移植。多发性硬化症(MS)约占 AD 移植的 50%,HSCT 是治疗算法的重要组成部分和标准护理部分。在美洲,至少有一部分 HSCT 向 CIBMTR 报告,因为报告是自愿的。共报告了 1400 例自体 HSCT 受者,其中 1030 例用于治疗神经系统疾病。MS 占所有神经系统疾病 HSCT 诊断的 96%。尽管与患病率相比,美国 MS 的 HSCT 活动率较低,但近年来移植数量有所增加。相比之下,墨西哥报告称此类移植数量急剧增加。本章概述了 EBMT 和 CIBMTR 登记处,然后提供了与神经系统 AD 相关的当前状况和出版物产出。
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