In the past 5 years, around 350 patients have received haematopoietic stem cell (HSC) transplantation for an autoimmune disease, with 275 of these registered in an international data base in Basel under the auspices of the European League Against Rheumatism (EULAR) and the European Group for Blood and Marrow Transplantation(EBMT). Most patients had either a progressive form of multiple sclerosis (MS; n = 88) or scleroderma (now called systemic sclerosis; n = 55). Other diseases were rheumatoid arthritis (Ra n = 40), juvenile idiopathic arthritis (JIA; n = 30), systemic lupus erythematosus (SLE; n = 20), idiopathic thrombocytopenic purpura (ITP; n = 7) and others. The procedure-related mortality was around 9%, with between-disease differences, being higher in systemic sclerosis and JIA and lower in RA (one death only). Benefit has been seen in around two-thirds of cases. No one regimen was clearly superior to another, with a trend toward more infectious complications with more intense regimens. Prospective, controlled randomized trials are indicated and being planned.

译文

在过去的5年中,约有350名患者因自身免疫性疾病接受了造血干细胞 (HSC) 移植,在欧洲抗风湿病联盟 (EULAR) 和欧洲血液和骨髓移植小组 (EBMT) 的主持下,其中275注册在巴塞尔的国际数据库中。大多数患者患有进行性多发性硬化症 (MS; n = 88) 或硬皮病 (现在称为系统性硬化症; n = 55)。其他疾病包括类风湿关节炎 (Ra n = 40),幼年特发性关节炎 (JIA; n = 30),系统性红斑狼疮 (SLE; n = 20),特发性血小板减少性紫癜 (ITP; n = 7) 等。与手术相关的死亡率约为9%,存在疾病间差异,系统性硬化症和JIA较高,RA较低 (仅1例死亡)。在大约3分之2的病例中看到了好处。没有一种方案明显优于另一种方案,随着更严格的治疗方案,感染并发症的趋势越来越明显。前瞻性,对照随机试验表明,并正在计划中。

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