Autologous hematopoietic stem cell transplantation for autoimmune diseases

Autologous hematopoietic stem cell transplantation for autoimmune diseases
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DOI:
10.1038/sj.bmt.1704892
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发表时间:
2005-05-01
影响因子:
4.8
通讯作者:
Tyndall, A
Tyndall, A
中科院分区:
医学3区
文献类型:
--
作者:
Gratwohl, A;Passweg, J;Tyndall, A

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实验数据和早期 I/II 期研究表明,高剂量化疗后进行自体造血干细胞移植 (HSCT) 可以阻止严重自身免疫性疾病的进展。我们评估了 1995 年至 2003 年间接受自体 HSCT 治疗的 473 名严重自身免疫性疾病患者的毒性和疾病反应,这些患者来自欧洲血液和骨髓移植组 (EBMT) 自身免疫性疾病工作组数据库的 110 个中心。评估了生存率、移植相关死亡率、治疗反应和疾病进展。总共有 420 名患者(89%;3 年时 86 +/- 4%,中位随访 20 个月)存活,53 名患者(11%)死于移植相关死亡率(N=31;3 年时 7 +/- 3%)或疾病进展(N=22;3 年时 9 +/- 4%)。在 370 名患者中,299 名可评估反应的患者 (81%) 表现出治疗反应,其中 213 名患者(71% 的反应者)持续出现治疗反应。反应与疾病相关(P40 岁,RR0.29 (0.11-0.82))。疾病进展与疾病相关(P
Experimental data and early phase I/II studies suggest that high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (HSCT) can arrest progression of severe autoimmune diseases. We have evaluated the toxicity and disease response in 473 patients with severe autoimmune disease treated with autologous HSCT between 1995 and 2003, from 110 centers participating in the European Group for Blood and Marrow Transplantation (EBMT) autoimmune disease working party database. Survival, transplant-related mortality, treatment response and disease progression were assessed. In all, 420 patients (89%; 86 +/- 4% at 3 years, median follow-up 20 months) were alive, 53 (11%) had died from transplant-related mortality (N=31; 7 +/- 3% at 3 years) or disease progression (N=22; 9 +/- 4% at 3 years). Of 370 patients, 299 evaluable for response (81%) showed a treatment response, which was sustained in 213 (71% of responders). Response was associated with disease (P40 years, RR0.29 (0.11-0.82)). Disease progression was associated with disease (P