Hügle Thomas, van Laar Jacob M
Musculoskeletal Research Group, Institute of Cellular Medicine, Newcastle University 4th Floor, Catherine Cookson Building, The Medical School, Framlington Place, Newcastle upon Tyne, NE2 4HH UK.
F1000 Med Rep. 2010 Mar 25;2:22. doi: 10.3410/M2-22.
Haematopoietic stem cell transplantation (HSCT) has evolved from an experimental concept to an effective treatment option for severe autoimmune diseases and has a unique ability to restore immune regulation. It is a complex multistep procedure involving the administration of high doses of immunosuppressive medication and transplantation of stem cells. Most HSCT procedures in autoimmune disease have involved autologous stem cells. In the case of allogeneic transplantation, stem cells are derived from peripheral blood or bone marrow of a healthy HLA-matched donor. Allogeneic HSCT has curative potential based on studies in experimental models of autoimmune disease, case reports, and a registry analysis but carries significant risks of rejection and graft-versus-host disease. Unless these risks become manageable, allogeneic HSCT should be offered only if all alternative treatment options have failed, if a patient has a suitable donor, and if a patient still has a chance to benefit significantly from the procedure.
造血干细胞移植（HSCT）已从一个实验性概念发展成为治疗严重自身免疫性疾病的有效选择，并且具有恢复免疫调节的独特能力。它是一个复杂的多步骤过程，包括给予高剂量免疫抑制药物和干细胞移植。自身免疫性疾病的大多数HSCT程序都涉及自体干细胞。在异基因移植的情况下，干细胞来自健康的HLA匹配供体的外周血或骨髓。基于自身免疫性疾病实验模型的研究、病例报告和登记分析，异基因HSCT具有治愈潜力，但存在显著的排斥和移植物抗宿主病风险。除非这些风险可控，否则只有在所有替代治疗方案均失败、患者有合适的供体且患者仍有机会从该程序中显著获益时，才应提供异基因HSCT。
