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. 2004 Oct;63(10):1318–1326. doi: 10.1136/ard.2003.017798

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

I M de Kleer 1, D Brinkman 1, A Ferster 1, M Abinun 1, P Quartier 1, J van der Net 1, C ten 1, L Wedderburn 1, G Horneff 1, J Oppermann 1, F Zintl 1, H Foster 1, A Prieur 1, A Fasth 1, M A J van Rossum 1, W Kuis 1, N Wulffraat 1
PMCID: PMC1754760  PMID: 15361393

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.

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Figure 1.

Figure 1

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 Reconstitution of lymphocyte subsets in 29 patients with juvenile idiopathic arthritis after autologous stem cell transplantation for 24 months. Error bars = SEM.

Figure 2.

Figure 2

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 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 up of a particular patient.

Figure 3.

Figure 3

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 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 follow up). With the exception of EPM-ROM scores at time points 24, 36, and 48 months post-ASCT, all scores post-ASCT were significantly decreased compared with baseline (Wilcoxon signed-rank test; all p values ⩽0.04). Top left: physician's global assessment of overall wellbeing (visual analogue scale, range 0–10); top right: parent/patient assessment of pain (VAS, range 0–3); middle left: functional disability (child health assessment questionnaire (CHAQ), range 0–3); middle right: number of active joints (0–54); bottom left, number of joints with limited range of motion (paediatric EPM-ROM, range 0–3); bottom right, erythrocyte sedimentation rate (ESR, mm/h).

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