Tyndall A, Gratwohl A
Department of Rheumatology, Felix Platter Spital, Basel, Switzerland.
Curr Opin Hematol. 1997 Nov;4(6):390-4. doi: 10.1097/00062752-199704060-00005.
The past year of activities represents virtually the first year of activities in hemopoietic blood and marrow transplants in the treatment of severe autoimmune disease. The concept of profound hematoimmunoablation followed by blood or marrow transplants for severe autoimmune disease is not new, but the first publications of such cases treated for autoimmune disease alone occurred only in late 1996. Other case reports followed, and the activity has expanded rapidly such that over 40 treated patients have been described, 35 of whom have been entered into a recently created European League Against Rheumatism/European Group for Blood and Marrow Transplantation database (now extended to include data from Australasia and the United States). Four international meetings have been held, the published proceedings of which have formed the basis for an ongoing international collaborative effort. Although very early, some trends have been observed: the majority of patients (mostly with multiple sclerosis or scleroderma) have clinically improved or stabilized; the toxicity of the procedure (mostly autologous bone marrow transplantation) was as observed previously in other disorders; and better results are suggested in patients achieving significant T-cell depletion, either through conditioning, graft purging, or both. These impressions must be confirmed by prospective comparative studies using a limited number of regimens.
过去一年的活动实际上代表了造血血液和骨髓移植治疗严重自身免疫性疾病的首个活动年。先进行深度血液免疫消融，然后进行血液或骨髓移植来治疗严重自身免疫性疾病的概念并不新鲜，但仅针对自身免疫性疾病进行治疗的此类病例的首批出版物直到1996年末才出现。随后又有其他病例报告，并且该活动迅速扩展，以至于已有超过40例接受治疗的患者被报道，其中35例已被录入最近建立的欧洲抗风湿病联盟/欧洲血液和骨髓移植小组数据库（现扩展至包括来自澳大拉西亚和美国的数据）。已经召开了四次国际会议，其发表的会议记录构成了正在进行的国际合作努力的基础。尽管还非常早期，但已观察到一些趋势：大多数患者（主要是患有多发性硬化症或硬皮病）在临床上病情有所改善或稳定；该治疗程序（主要是自体骨髓移植）的毒性与先前在其他疾病中观察到的情况相同；通过预处理、移植物净化或两者兼用实现显著T细胞清除的患者似乎有更好的结果。这些印象必须通过使用有限数量方案的前瞻性对照研究来证实。
