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

Lancet. 1999 Feb 13;353(9152):550-3. doi: 10.1016/S0140-6736(98)05399-9.


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. Erythrocyte 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.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Arthritis, Juvenile / therapy*
  • Child
  • Female
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Lymphocyte Depletion
  • Male
  • T-Lymphocytes / immunology
  • Time Factors
  • Transplantation Conditioning*
  • Transplantation, Autologous
  • Treatment Outcome