MOORE TB, SHERRY D, DEODHAR A, TILFORD DL, JOHNSON FL, JONES GR, NICHOLSON HS, THOMAS GA, WOLFF LJ, BRADY K: Stem cell transplantation for severe juvenile rheumatoid arthritis. Blood 2000, 96:373b
Organism:Pediatric Hematology/Oncology, Oregon Health Sciences University, Portland, OR USA
Abstract: Autologous stem cell transplantation offers potential as a treatment for refractory juvenile rheumatoid arthritis (JRA). We report our experience in the treatment of a ten year old girl with an eight year history of severe systemic JRA using high dose cytotoxic/immunosuppressive therapy and autologous CD34+ selected stem cell support. Initially diagnosed at 2 years of age, her JRA was completely refractory to conventional therapy including corticosteroids, methotrexate and low dose cyclophosphamide. Pain reduction including narcotics was ineffective. She required biannual corticosteroid injections of as many as 24 joints at a time. Peripheral blood stem cells were collected after 5 days of 10 mcg/kg/d of G-CSF. There was no exacerbation of arthritis during the G-CSF administration. Four collections and CD34+ selection were performed yielding a final product of 2 X 10e6 CD34+ cells/kg and less than 10e5 CD3+ cells/kg. Conditioning prior to stem cell infusion consisted of ATG 20 mg/kg, cyclophosphamide 200 mg/kg and TBI 400 cGy. She achieved neutrophil engraftment by day 17 and platelet transfusion independence by day 18. Serologic markers of her JRA including C-reactive protein became normal. She was readily weaned off all pain medications by 4 months and tapered off prednisone rapidly over the first month to 5 mg/d, and by 7 months following transplant was off all immunosuppressive drugs. She is currently pain-free and off all medications. The affected joint count dropped from greater than 11 (pre-transplant) to zero. The Karnoffsky score is currently 100. By 6 months post-transplant, she had normal IgG, IgM, and IgA levels, and normal B cell number. There was a normal proliferative response to mitogens PHA, CON-A, and PWM. T cells show a persistent inversion of the helper-suppresser ratio with a follow up of 8 months42nd Annual Meeting of the American Society of Hematology
San Francisco, California, USA
December 01-05, 2000
American Society of Hematology_