Autologous haematopoietic stem cell transplants for autoimmune disease -: feasibility and transplant-related mortality
Autologous haematopoietic stem cell transplants for autoimmune disease -: feasibility and transplant-related mortality
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DOI：
10.1038/sj.bmt.1701987
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发表时间：
1999-10-01
影响因子：
4.8
通讯作者：
Gratwohl, A
中科院分区：
文献类型：
--
作者：
Tyndall, A;Fassas, A;Gratwohl, A
这项正在进行的多中心前瞻性I/II期试验招募了来自全球22个中心的74名患者，其中包括严重自身免疫性疾病患者、35名风湿病患者、31名神经系统疾病患者、5名血液病患者和3名血管疾病患者。他们根据预先确定的标准接受自体外周血或骨髓移植治疗。72例患者接受了73例移植，7例骨髓移植，66例动员外周血干细胞移植。43例患者通过移植物去除T细胞和/或B细胞。全部73例移植成功。5例患者死于移植相关并发症：2例死于出血，3例死于感染，2例死于疾病进展。1年移植相关死亡率为9% (1-17%;95% CI)，与同期欧洲非霍奇金淋巴瘤热敏感复发移植患者的移植相关死亡率为6% （3-9%;95% CI）相当（P = 0.39）。60例患者可评估反应，40例（65%）患者的疾病有所改善。造血干细胞移植对严重难治性自身免疫性疾病患者是可行的。移植相关死亡率与非霍奇金淋巴瘤患者应答性复发的结果相当。三分之二的患者至少表现出一些反应。这些初步数据是有希望的。尽管存在相当大的风险，但比较自体干细胞移植与常规治疗的随机试验是有必要的。
This ongoing multicentre prospective phase I/II trial enrolled 74 consecutive patients from 22 centres world-wide with severe autoimmune disease, 35 with rheumatological disorders, 31 with neurological, five with haematological and three with vasculitides. They were treated with autologous peripheral blood or bone marrow transplants according to predetermined criteria. Two patients died after mobilisation before transplant Seventy-two patients were given 73 transplants, seven bone marrow, and 66 mobilised peripheral blood stem cell transplants. The graft was manipulated to remove T and/or B cells in 43 cases. All 73 transplants engrafted. Five patients died of transplant-related complications: two from bleeding, three from infections, Two patients died of progressive disease, The transplant-related mortality at 1 year of 9% (1-17%; 95% CI) is comparable to the transplant-related mortality of 6% (3-9%; 95% CI) in patients transplanted during the same period in Europe for non-Hodgkin's lymphoma ire sensitive relapse (P = 0.39). Sixty patients are evaluable for response, 40 patients (65%) showed some improvement in their disease. Haematopoietic stem cell transplants are feasible for patients with severe refractory autoimmune disease. Transplant-related mortality is comparable to results in patients with non-Hodgkin's lymphoma in responsive relapse. Two-thirds of the patients show at least some response. These preliminary data are promising. Although associated with considerable risk, randomised trials comparing autologous stem cell transplants to conventional therapy are warranted.