Burt R K, Traynor A E, Craig R, Marmont A M
Northwestern University Department of Medicine, Chicago, IL, USA.
Bone Marrow Transplant. 2003 Apr;31(7):521-4. doi: 10.1038/sj.bmt.1703868.
Hematopoietic stem cell transplantation (HSCT) is being increasingly utilized for the treatment of a whole spectrum of severe autoimmune diseases refractory to conventional therapy. Although allogeneic HSCT has been followed by durable complete remission in a restricted number of patients with coincidental disease, the autologous procedure is generally preferred because of its lesser toxicity. Most autoimmune diseases are the consequence of a multistep process, mainly originating from the interplay of genetic, environmental, and hormonal factors. It has been postulated that if immunosuppressive regimens can eliminate or effectively reduce the level of autoreactive T and B cells, then regeneration of de novo immunity even in the autologous setting may bypass the initial breakdown of self-tolerance and ensure prolonged disease remission. As mentioned in a recent review of this field, protocol design including conditioning regimen, patient selection, stem cell source and final outcome are likely to be disease-specific. The following is a summary of the 2002 International Bone Marrow Transplantation Registry/American Society of Blood and Bone Marrow Transplantation (IBMTR/ASBMT) satellite symposium in Orlando, Florida on 24 February 2002 on 'Expanding the Promise of Hematopoietic Stem Cell Transplantation in Autoimmune Diseases'.
造血干细胞移植(HSCT)正越来越多地用于治疗一系列对传统疗法难治的严重自身免疫性疾病。尽管在少数患有并发疾病的患者中,异基因造血干细胞移植后出现了持久的完全缓解,但自体移植程序通常因其毒性较小而更受青睐。大多数自身免疫性疾病是一个多步骤过程的结果,主要源于遗传、环境和激素因素的相互作用。据推测,如果免疫抑制方案能够消除或有效降低自身反应性T细胞和B细胞的水平,那么即使在自体移植情况下,新生免疫的再生也可能绕过自身耐受性的最初破坏,并确保疾病长期缓解。正如最近对该领域的一篇综述中所提到的,包括预处理方案、患者选择、干细胞来源和最终结果在内的方案设计可能因疾病而异。以下是2002年2月24日在佛罗里达州奥兰多举行的2002年国际骨髓移植登记处/美国血液和骨髓移植学会(IBMTR/ASBMT)卫星研讨会“扩大造血干细胞移植在自身免疫性疾病中的前景”的总结。