Expert Reviews in Molecular Medicine: http://www.expertreviews.org/
DOI: 10.1017/S1462399406000135; 15 November 2006
Nico M. Wulffraat, Ismé M. de Kleer and Berent Prakken (2006) Refractory juvenile idiopathic arthritis: using autologous stem cell transplantation as a treatment strategy. Expert Rev. Mol. Med. Vol. 8, Issue 26, DOI: 10.1017/S1462399406000135

Refractory juvenile idiopathic arthritis: using autologous stem cell transplantation as a treatment strategy

Nico M. Wulffraat ^{a1 c1}, Ismé M. de Kleer ^a1 and Berent Prakken ^a1

^a1 Department of Pediatric Immunology, University Medical Center Utrecht, ‘Wilhelmina Children's Hospital’, 3508 AB Utrecht, The Netherlands.

^c1 Corresponding author: Nico M. Wulffraat, Department of Pediatric Immunology, University Medical Center Utrecht, ‘Wilhelmina Children's Hospital’, PO Box 85090, 3508 AB Utrecht, The Netherlands. Tel: +31 30 2504003; Fax: +31 30 2505350; E-mail: n.wulffraat@umcutrecht.nl

Cellular immune therapy for severe autoimmune diseases can now be considered when such patients are refractory to conventional treatment. The use of autologous stem cell transplantation (ASCT) to treat human autoimmune diseases has been initiated following promising results in a variety of animal models. Anecdotal observations have been made of autoimmune disease remission in patients who have undergone allogeneic bone marrow transplantation as a result of coincidental haematological malignancies. The possibility of inducing immunological self-tolerance by ASCT is particularly attractive as a means for treating juvenile idiopathic arthritis (JIA). In this disease, ASCT restores self-tolerance both through a cell-intrinsic mechanism, involving the reprogramming of autoreactive T cells, and through a cell-extrinsic mechanism, involving a renewal of the immune balance between CD4⁺CD25⁺ regulatory T cells and other T cells. This review describes the clinical results of ASCT performed for this disease and the possible underlying immunological mechanisms.

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