Autologous stem cell transplantation for severe juvenile idiopathic arthritis: A multicenter survey

Since 1997 Autologous Stem Cell Transplantation (ASCT) has been applied as an experimental procedure in more than 40 children with refractory Juvenile Idiopathic Arthritis. In order to evaluate feasibility, safety and efficacy we analyzed retrospectively follow up data on 34 children with JIA who were treated with ASCT in 9 different European transplant centers. Rheumatological evaluation was performed by using a modified set of core set criteria. Data were collected on the number of active joints, joints with limited range of motion, physicians global assessment of well-being, parent/patient assessment of pain, functional disability and the erythrocyte sedimentation rate. A response was defined as an improvement of 30% or more in at least three of indicators of disease outcome. The clinical follow up of the children ranged from 12 to 60 months. Eighteen of the 34 patients (53%) with a follow up of 12 to 60 months achieved a drug free complete remission. Seven of these patients had previously failed treatment with anti-Tumor-Necrosis-Factor therapy. Six of the 34 patients (18%) showed a partial response (ranging from 30–70%) and 7 of the 34 patients (21%) showed a complete relapse of disease. Infectious complications were seen frequently. There were three cases (9%) of transplant related mortality due to Macrophage Activation Syndrome (MAS) and two cases of disease related mortality (6%). ASCT in severely ill JIA patients induces a drug-free remission of the disease and a profound increase in general well-being in a substantial proportion of patients. However, the procedure carries a significant mortality risk. In 1999 the protocol was adapted to avoid new cases of MAS. Since then, no more cases of fatal MAS have been reported.