Tyndall Alan, Daikeler Thomas
Department of Rheumatology, University Hospital, Basel, Switzerland.
Acta Haematol. 2005;114(4):239-47. doi: 10.1159/000088415.
Ten years have passed since the first published consensus statement on the use of hematopoietic stem cell transplantation (HSCT) in the treatment of severe autoimmune disease (AD) appeared. During that time, around 700 patients suffering from severe AD have undergone HSCT in the frame of phase I/II clinical trials from over 20 countries including the US. The majority have received an autologous HSCT using one of a limited number of regimens, consistent with the original consensus statement. Long-term drug-free remissions, remission then relapse, no response and treatment-related mortality (TRM) were seen in all the subgroups of AD. An overall TRM of 7% was observed, with marked variation between ADs, i.e. 11% in systemic lupus erythematosus (SLE) and only 1 patient in rheumatoid arthritis (RA). Phase III prospective, comparative randomized trials are running or being planned in multiple sclerosis (MS), systemic sclerosis (SSc), SLE and RA. Basic science programs are also being undertaken to study the immunological mechanisms underlying the clinical events observed.
自首份关于使用造血干细胞移植（HSCT）治疗严重自身免疫性疾病（AD）的共识声明发表以来，已经过去了十年。在此期间，来自包括美国在内的20多个国家的约700名患有严重AD的患者在I/II期临床试验框架内接受了HSCT。大多数患者使用了有限数量的方案之一接受了自体HSCT，这与最初的共识声明一致。在AD的所有亚组中均观察到长期无药缓解、缓解后复发、无反应以及治疗相关死亡率（TRM）。观察到总体TRM为7%，不同AD之间存在显著差异，即系统性红斑狼疮（SLE）为11%，而类风湿关节炎（RA）仅有1例患者。针对多发性硬化症（MS）、系统性硬化症（SSc）、SLE和RA的III期前瞻性、比较随机试验正在进行或正在计划中。也正在开展基础科学项目以研究观察到的临床事件背后的免疫机制。
