Autologous haemopoietic stem-cell transplantation in four patients with refractory juvenile chronic arthritis

Background Autologous haemopoietic stem-cell transplantation (AHSCT) had been described as a possible treatment for severe autoimmune disease refractory to conventional treatment. We report the first four children with severe forms of juvenile chronic arthritis (JCA) treated with AHSCT. Methods We studied three children with systemic JCA and one child with polyarticular JCA. Unprimed bone marrow was taken 1 month before AHSCT. T-cell depletion of the graft was done with CD2 and CD3 antibodies. We used a preparative regimen of antithymocyte globulin (20 mg/kg), cyclophosphamide (200 mg/kg) and low-dose total body irradiation (4 Gy). Methotrexate and cyclosporin were stopped before AHCST, prednisone was tapered after 2 months. Findings Our patients showed a drug-free follow-up of 6–18 months with a marked decrease in joint swelling, pain, and morning stiffness. Erythocyte sedimentation rate, C-reactive protein, and haemoglobin returned to almost normal values within 6 weeks. Despite T-cell depletion there was a rapid immune reconstitution in three out of four children. Two patients developed a limited varicella zoster virus eruption, which was treated by aciclovir. Interpretation AHSCT for severe JCA was well tolerated and induced a remission of disease in four children with JCA that was resistant to conventional treatment. Prolonged prednisone-free growth catch-up and general well-being is a major therapeutic gain in such children. The actual follow-up is too short, however, for us to conclude that these children are completely cured of their disease.