Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity
Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity
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DOI：
10.1136/ard.2003.017798
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发表时间：
2004-10-01
影响因子：
27.4
通讯作者：
Wulffraat, NM
中科院分区：
文献类型：
--
作者：
de Kleer, IM;Brinkman, DMC;Wulffraat, NM
目的：评估难治性幼年特发性关节炎（JIA）自体干细胞移植（ASCT）的安全性和有效性。设计：回顾性分析34例JIA患儿在9个不同的欧洲移植中心接受ASCT治疗的随访数据。流变学评价采用了一套修改后的核心标准。结果：术后随访12 ~ 60个月，观察移植后免疫功能恢复情况及感染并发症。34例患者中有18例（53%）在随访12至60个月后达到完全无药缓解。其中7例患者既往接受抗TNF治疗失败。34例患者中有6例（18%）显示部分缓解（范围为30%至70%的改善），7例（21%）对ASCT耐药。感染并发症常见。有三例移植相关死亡率（9%）和两个疾病相关的死亡率（6%.Conclusions：ASCT重症患者JIA诱导的疾病的药物缓解和一个深刻的增加，在一个相当大的比例的患者的总体健康，但程序进行了显着的死亡风险。建议对未来的方案进行以下调整：（1）从预处理方案中消除全身照射;（2）预防性给予抗病毒药物和静脉注射免疫球蛋白，直至CD 4 + T细胞计数正常。
Objective: To evaluate the safety and efficacy of autologous stem cell transplantation ( ASCT) for refractory juvenile idiopathic arthritis (JIA).Design: Retrospective analysis of follow up data on 34 children with JIA who were treated with ASCT in nine different European transplant centres. Rheumatological evaluation employed a modified set of core criteria. Immunological reconstitution and infectious complications were monitored at three month intervals after transplantation.Results: Clinical follow up ranged from 12 to 60 months. Eighteen of the 34 patients (53%) with a follow up of 12 to 60 months achieved complete drug-free remission. Seven of these patients had previously failed treatment with anti-TNF. Six of the 34 patients (18%) showed a partial response ( ranging from 30% to 70% improvement) and seven (21%) were resistant to ASCT. Infectious complications were common. There were three cases of transplant related mortality (9%) and two of disease related mortality (6%).Conclusions: ASCT in severely ill patients with JIA induces a drug-free remission of the disease and a profound increase in general wellbeing in a substantial proportion of patients, but the procedure carries a significant mortality risk. The following adjustments are proposed for future protocols: ( 1) elimination of total body irradiation from the conditioning regimen; ( 2) prophylactic administration of antiviral drugs and intravenous immunoglobulins until there is a normal CD4+ T cell count.