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Ann Rheum Dis. 2004 October; 63(10): 1318–1326.
PMCID: PMC1754760

Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity

Abstract

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.

Figure 1
 Reconstitution of lymphocyte subsets in 29 patients with juvenile idiopathic arthritis after autologous stem cell transplantation for 24 months. Error bars = SEM.
Figure 2
 Kaplan–Meier curves showing the proportion of surviving patients and the proportion of event-free surviving patients. An event is defined as either a partial or complete recurrence of disease. Each bar mark represents the maximum follow ...
Figure 3
 Rheumatological follow up before and at three monthly intervals after autologous stem cell transplantation. Error bars = SD. Numbers below the x axis indicate ratios of the available data (number of data available/number of patients with indicated ...

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