Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI, USA.
Biol Blood Marrow Transplant. 2012 Oct;18(10):1471-8. doi: 10.1016/j.bbmt.2012.06.003. Epub 2012 Jun 13.
Hematopoietic cell transplantation (HCT) is an emerging therapy for patients with severe autoimmune diseases (AID). We report data on 368 patients with AID who underwent HCT in 64 North and South American transplantation centers reported to the Center for International Blood and Marrow Transplant Research between 1996 and 2009. Most of the HCTs involved autologous grafts (n = 339); allogeneic HCT (n = 29) was done mostly in children. The most common indications for HCT were multiple sclerosis, systemic sclerosis, and systemic lupus erythematosus. The median age at transplantation was 38 years for autologous HCT and 25 years for allogeneic HCT. The corresponding times from diagnosis to HCT were 35 months and 24 months. Three-year overall survival after autologous HCT was 86% (95% confidence interval [CI], 81%-91%). Median follow-up of survivors was 31 months (range, 1-144 months). The most common causes of death were AID progression, infections, and organ failure. On multivariate analysis, the risk of death was higher in patients at centers that performed fewer than 5 autologous HCTs (relative risk, 3.5; 95% CI, 1.1-11.1; P = .03) and those that performed 5 to 15 autologous HCTs for AID during the study period (relative risk, 4.2; 95% CI, 1.5-11.7; P = .006) compared with patients at centers that performed more than 15 autologous HCTs for AID during the study period. AID is an emerging indication for HCT in the region. Collaboration of hematologists and other disease specialists with an outcomes database is important to promote optimal patient selection, analysis of the impact of prognostic variables and long-term outcomes, and development of clinical trials.
造血细胞移植(HCT)是一种新兴的治疗严重自身免疫性疾病(AID)的方法。我们报告了 1996 年至 2009 年期间在北美和南美 64 个移植中心向国际血液和骨髓移植研究中心报告的 368 例 AID 患者接受 HCT 的数据。大多数 HCT 涉及自体移植物(n=339);异基因 HCT(n=29)主要用于儿童。HCT 的最常见适应证是多发性硬化症、系统性硬化症和系统性红斑狼疮。自体 HCT 的中位年龄为 38 岁,异基因 HCT 为 25 岁。从诊断到 HCT 的相应时间分别为 35 个月和 24 个月。自体 HCT 后 3 年总生存率为 86%(95%置信区间[CI],81%-91%)。幸存者的中位随访时间为 31 个月(范围,1-144 个月)。死亡的最常见原因是 AID 进展、感染和器官衰竭。多变量分析显示,在中心行 HCT 少于 5 例的患者(相对风险,3.5;95%CI,1.1-11.1;P=0.03)和在研究期间行 5 至 15 例自体 HCT 治疗 AID 的患者(相对风险,4.2;95%CI,1.5-11.7;P=0.006)的死亡风险高于在研究期间行 HCT 治疗 AID 超过 15 例的患者。AID 是该地区 HCT 的新兴适应证。血液科医生和其他疾病专家与结局数据库合作,对于促进最佳患者选择、分析预后变量和长期结果的影响以及开展临床试验非常重要。