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

Background Autologous haemopoietic stem-cell transplantation (AHSCT) had be en described as a possible treatment for severe autoimmune disease refracto ry 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 poly articular JCA. Unprimed bane marrow was taken 1 month before AHSCT. T-cell depletion of the graft was done with CD2 and CD3 antibodies. We used a prep arative regimen of antithymocyte globulin (20 mg/kg), cyclophosphamide (200 mg/kg) and low-dose total body irradiation (4 Gy). Methotrexate and cyclos porin 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 ma rked decrease in joint swelling, pain, and morning stiffness. Erythocyte se dimentation rate, C-reactive protein, and haemoglobin returned to almost no rmal values within 6 weeks. Despite T-cell depletion there was a rapid immu ne reconstitution in three out of four children. Two patients developed a l imited varicella tester virus eruption, which was treated by aciclovir. Interpretation AHSCT for severe JCA was well tolerated and induced a remiss ion of disease in four children with JCA that was resistant to conventional treatment. Prolonged prednisane-free growth catch-up and general well-bein g 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 cure d of their disease.