Tyndall A, Passweg J, Gratwohl A
Department of Rheumatology, University Hospital, Basel, Switzerland.
Ann Rheum Dis. 2001 Jul;60(7):702-7. doi: 10.1136/ard.60.7.702.
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.
2000年10月5日至7日,一场国际会议在瑞士巴塞尔举行,来自30个国家的180名参与者出席了会议,会议旨在评估自体造血干细胞移植(HSCT)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。会上展示了390例患者的数据:260例来自欧洲骨髓移植协作组/欧洲抗风湿病联盟(EBMT/EULAR)巴塞尔欧洲/亚洲数据库,87例来自北美(55例来自国际骨髓移植登记处(IBMTR)),39例来自澳大利亚,另有4例。接受移植的患者主要疾病类别及数量如下:多发性硬化症(MS)127例、系统性硬化症(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/多肌炎(DM/PM)5例、特发性血小板减少性紫癜(ITP)7例。还报告了其他自身免疫性疾病的单例或多例病例。所有病例中有三分之二以及所有疾病类别均出现了具有临床意义的反应,JIA和RA的复发趋势更为明显。治疗与显著的发病率和死亡率相关。在欧洲抗风湿病联盟/欧洲骨髓移植协作组数据库(22个国家的71个中心)中,动员相关死亡率为1.5%,总体手术相关死亡率(12个月精算调整后)为9%(置信区间6%至12%),不同疾病之间存在显著差异。北美的数据显示了类似结果。SSc和系统性JIA的死亡率较高,RA仅报告了1例死亡。在展示数据并进行研讨会讨论后,就几个方面达成了共识:目前在SSc、MS和RA中进行前瞻性随机III期试验是合适的。SSc的试验方案已准备就绪(ASTIS试验),MS和RA的概念明确。SLE、JIA和血管炎还需要进一步的I期和II期数据。强调了通过标准化的欧洲骨髓移植协作组和国际骨髓移植登记处数据表格持续收集所有动员后病例的必要性。