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Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality, and transplant related morbidity
  1. I M de Kleer1,
  2. D M C Brinkman2,
  3. A Ferster4,
  4. M Abinun8,
  5. P Quartier9,
  6. J van der Net1,
  7. R ten Cate2,
  8. L R Wedderburn3,
  9. G Horneff5,
  10. J Oppermann6,
  11. F Zintl7,
  12. H E Foster8,
  13. A M Prieur9,
  14. A Fasth10,
  15. M A J van Rossum2,
  16. W Kuis1,
  17. N M Wulffraat1
  1. 1Wilhelmina Children’s Hospital of the University Medical Centre Utrecht, Utrecht, Netherlands
  2. 2Leiden University Medical Centre, Leiden, Netherlands
  3. 3Institute of Child Health and Great Ormond Street Hospital, London WC1, UK
  4. 4University Hospital of Paediatrics, Brussels, Belgium
  5. 5Centre of Paediatrics, Martin-Luther-University, Halle-Wittenberg, Germany
  6. 6Department of Rheumatology, Carl-Thiem Hospital, Cottbus, Germany
  7. 7Department of Haematology/Oncology, Children’s University Hospital, Jena, Germany
  8. 8Department of Child Health and Rheumatology, Newcastle Hospitals NHS Trust, Newcastle Upon Tyne, UK
  9. 9Unité d’Immunologie-Hématologie et Rhumatologie Pédiatriques, Hôpital Necker- Enfants Malades, Paris, France
  10. 10Department of Paediatrics, Göteborg University and the Queen Silvia Children’s Hospital, Gothenburg, Sweden
  1. Correspondence to:
    Dr N M Wulffraat
    Paediatric BMT unit, Suite KC 03.063, University Medical Centre Utrecht, PO box 85090, 3508 AB Utrecht, Netherlands;


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.

  • ASCT, autologous stem cell transplantation
  • ATG, anti-thymocyte immunoglobulin
  • CHAQ, child health assessment questionnaire
  • DMARD, disease modifying anti-rheumatic drug
  • EBMT, European Group for Blood and Marrow Transplantation
  • IAHS, infection associated haemophagocytic syndrome
  • JIA, juvenile idiopathic arthritis
  • NSAID, non-steroidal anti-inflammatory drug
  • TBI, total body irradiation
  • TNF, tumour necrosis factor
  • VAS, visual analogue scale
  • juvenile idiopathic arthritis
  • autologous stem cell transplantation

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