National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, Maryland 20892, USA.
Ann Rheum Dis. 2011 Dec;70(12):2071-4. doi: 10.1136/ard.2010.148049. Epub 2011 Aug 26.
Autologous haematopoietic stem cell transplantation (AHSCT) has been proposed as a treatment modality which may arrest the autoimmune disease process and lead to sustained treatment-free remissions. Since the first consensus statement in 1997, approximately 200 autologous bone marrow or haematopoietic stem cell transplantations (HSCTs) have been reported worldwide for systemic lupus erythematosus (SLE). The current state of AHSCT in SLE was reviewed at a recent meeting of the autoimmune working party of the European Group for Blood and Marrow Transplantation. There was general agreement among experts in this field that in patients with severe SLE refractory to conventional immunosuppressive treatments, AHSCT can achieve sustained clinical remissions (ranging from 50% to 70% disease-free survival at 5 years) associated with qualitative immunological changes not seen with other forms of treatment. However, this clinical benefit is associated with an increase in short-term mortality in most studies. Improving patient selection, long-term follow-up of patients after AHSCT, optimisation of induction and maintenance treatment together with detailed analysis of the immune system are identified as key areas for future research. Optimally, AHSCT should be compared with conventional treatment in randomised controlled trials. Development of stronger transplant registries, defining a core set of clinical data and standardising biological sample collections would make future collaborations and comparison of studies more feasible.
自体造血干细胞移植(AHSCT)已被提议作为一种治疗方式,可能会阻止自身免疫疾病进程并导致持续无治疗缓解。自 1997 年的第一份共识声明以来,全世界已经报告了大约 200 例自体骨髓或造血干细胞移植(HSCT)用于治疗系统性红斑狼疮(SLE)。在最近举行的欧洲血液和骨髓移植协会自身免疫工作组会议上,对 SLE 中的 AHSCT 现状进行了回顾。该领域的专家普遍认为,对于对传统免疫抑制治疗有抗性的严重 SLE 患者,AHSCT 可以实现持续的临床缓解(5 年时无疾病生存率为 50%至 70%),并伴有其他治疗形式未见的定性免疫变化。然而,这种临床获益与大多数研究中的短期死亡率增加有关。改善患者选择、AHSCT 后患者的长期随访、诱导和维持治疗的优化以及对免疫系统的详细分析被确定为未来研究的关键领域。理想情况下,AHSCT 应与常规治疗进行随机对照试验比较。发展更强的移植登记处,定义一套核心临床数据并标准化生物样本采集将使未来的合作和研究比较更加可行。