Autologous stem cell transplantation: a possible treatment for refractory juvenile chronic arthritis?

OBJECTIVE In adults, autologous stem cell transplantation (ASCT) has been described recently as a possible treatment for severe autoimmune disease refractory to conventional treatment. We report here the four first children with severe forms of juvenile chronic arthritis (JCA) treated with ASCT. METHODS We studied three children with systemic JCA and one child with polyarticular JCA. Unprimed bone marrow was harvested 1 month prior to ASCT. T-cell depletion of the graft was performed with CD2 and CD3 antibodies. We used a preparative regimen of antithymocyte globulin (ATG; 20 mg/kg), cyclophosphamide (Cy; 200 mg/kg) and low-dose total body irradiation (TBI; 4 Gy). Methotrexate (MTX) and cyclosporin A (CsA) were stopped before ASCT; prednisone was tapered after 2 months. RESULTS After ASCT, our patients showed an anti-inflammatory-drug-free follow-up of 6-18 months with a marked decrease in joint swelling, pain and morning stiffness. The erythrocyte sedimentation rate (ESR), C-reactive protein (CRP) and haemoglobin (Hb) returned to near-normal values within 6 weeks. Despite T-cell depletion, there was a very rapid immune reconstitution. Two patients developed a limited varicella zoster virus (VZV) eruption which was treated by acyclovir.