Haemopoietic stem cell transplantation in the treatment of severe autoimmune diseases 2000
Haemopoietic stem cell transplantation in the treatment of severe autoimmune diseases 2000
复制标题
造血干细胞移植治疗严重自身免疫性疾病 2000
DOI：
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发表时间：
2001
影响因子：
27.4
通讯作者：
A. Gratwohl
中科院分区：
文献类型：
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作者：
A. Tyndall;J. Passweg;A. Gratwohl
2000年10月5日至7日在瑞士巴塞尔举行了一次国际会议，来自30个国家的180名与会者参加了会议，目的是评估自体造血干细胞移植治疗严重自身免疫性疾病的现有数据，并决定未来的试验计划。 提供了390名患者的数据：260名来自欧洲/亚洲欧洲/亚洲数据库，87名来自北美(55名来自IBMTR)，39名来自澳大利亚，以及其他4名。主要疾病种类和移植病人数分别为：多发性硬化(MS)127例，系统性硬化(SSC)72例，类风湿关节炎(RA)70例，幼年特发性关节炎(JIA)36例，系统性红斑狼疮(SLE)34例，皮肌炎/多发性肌炎(DM/PM)5例，特发性血小板减少性紫癜(ITP)7例。在所有病例和所有疾病类别中，三分之二的病例出现了临床上显著的 反应，在日本心绞痛和类风湿关节炎中，复发的趋势更明显。治疗与显著的发病率和死亡率有关。在EULAR/EBMT数据库(22个国家的71个中心)中，发现与动员相关的死亡率为1.5%，与手术相关的总死亡率(精算调整为12个月)为9%(可信区间为6-12%)，不同疾病之间存在显著差异。北美的数据也显示出类似的结果。SSC和系统性JIA的死亡率较高，仅有一例RA报告死亡。 在介绍了数据和研讨会讨论后，在几个方面达成了共识：前瞻性随机第三阶段试验现在适用于SSc、MS和RA。SSC(ASTIS试验)的协议已经准备好，MS和RA的概念已经明确。SLE、JIA和血管炎需要进一步的I期和II期数据。会议强调，在通过标准化的EBMT和IBMTR数据表格动员后，有必要继续收集所有病例。
An international meeting took place in Basel, Switzerland from 5 to 7 October 2000 involving 180 participants from 30 countries, with the aim of assessing the existing data on autologous haemopoietic stem cell transplantation (HSCT) in the treatment of severe autoimmune disease, and to decide on future trial planning. Data on 390 patients were presented: 260 from the EBMT/EULAR Basel European/Asian database, 87 from North America (55 from the IBMTR), 39 from Australia, and 4 others. The major disease categories and number of patients receiving transplant were: multiple sclerosis (MS) 127, systemic sclerosis (SSc) 72, rheumatoid arthritis (RA) 70, juvenile idiopathic arthritis (JIA) 36, systemic lupus erythematosus (SLE) 34, dermatomyositis/polymyositis (DM/PM) 5, idiopathic thrombocytopenic purpura (ITP) 7. Single or several cases of other autoimmune diseases were reported. Clinically significant responses were seen in two thirds of all the cases and in all disease categories, with a more accentuated trend towards relapse in JIA and RA. Treatment was associated with a significant morbidity and mortality. In the EULAR/EBMT database (71 centres in 22 countries), a mobilisation associated mortality of 1.5% and an overall procedure related mortality (actuarially adjusted at 12 months) of 9% (confidence interval 6 to 12%) were found, with significant variation between diseases. The North American data showed similar results. Higher mortalities were seen in SSc and systemic JIA, with only one death reported in RA. After presentation of the data and workshop discussion a consensus was reached on several aspects: prospective randomised phase III trials are now appropriate in SSc, MS, and RA. A protocol is ready for SSc (ASTIS Trial), concepts are clear for MS and RA. Further phase I and II data are required in SLE, JIA, and vasculitis. The need for continuing collection of all cases after mobilisation by the standardised EBMT and IBMTR data forms was emphasised.