Allogeneic hematopoietic SCT for patients with autoimmune diseases
Allogeneic hematopoietic SCT for patients with autoimmune diseases
复制标题
DOI：
10.1038/bmt.2008.424
复制
发表时间：
2009-07-01
影响因子：
4.8
通讯作者：
Gratwohl, A.
中科院分区：
文献类型：
--
作者：
Daikeler, T.;Huegle, T.;Gratwohl, A.
异基因造血干细胞移植（HSCT）已被用于治疗个别自身免疫性疾病（AD）患者。为总结现有信息，我们分析了所有因自身免疫性疾病接受异基因HSCT且向欧洲血液和骨髓移植组织（EBMT）数据库报告的患者。确定了35名患者，他们因各种血液系统和非血液系统自身免疫性疾病接受了38次异基因移植。其中4名患者过去曾因常规血液系统适应证接受过异基因HSCT。55%的移植操作使难治性自身免疫性疾病达到完全临床缓解，23%至少达到部分缓解。在最后一次随访时，缓解的中位持续时间为70.7（15.2 - 130）个月。3名患者在HSCT后中位时间12.3个月复发。2年时治疗相关死亡率为22.1%（95%置信区间：7.3 - 36.9%）。2例死亡由自身免疫性疾病进展导致。2年生存率为70%。未发现可预测结果的单一因素。本研究的回顾性性质以及数据的异质性和部分不完整性是其局限性。然而，异基因HSCT可诱导难治性自身免疫性疾病患者缓解。这些数据为谨慎开展前瞻性试验提供了基础。《骨髓移植》（2009年）44卷，27 - 33页；doi:10.1038/bmt.2008.424；2009年1月12日在线发表
Allogeneic hematopoietic SCT (HSCT) ha s been used as treatment for single patients with autoimmune diseases (AD). To summarise currently available information, we analyzed all patients who underwent allogeneic HSCT for AD and who reported to the European Group for Blood and Marrow Transplantation (EBMT) data base. Thirty-five patients receiving 38 allogeneic transplantations for various hematological and non-hematological AD were identified. Four patients had had an allogeneic HSCT for a conventional hematological indication in the past. Fifty-five per cent of the transplantation procedures led to a complete clinical response of the refractory AD and 23% to at least a partial response. The median duration of response at the last follow-up was 70.7 (15.2-130) months. Three patients relapsed at a median of 12.3 months after HSCT. Treatment-related mortality at 2 years was 22.1% (95% CI: 7.3-36.9%). Two deaths were caused by progression of AD. The probability of survival at 2 years was 70%. No single factor predicting the outcome could be identified. The retrospective nature of this study and the heterogeneous, partly incomplete data are its limitations. However, allogeneic HSCT can induce remission in patients suffering from refractory AD. These data provide the basis for carefully conducted prospective trials. Bone Marrow Transplantation (2009) 44, 27-33; doi:10.1038/bmt.2008.424; published online 12 January 2009