A phase 1 study to address the safety and efficacy of granulocyte colony-stimulating factor for the mobilization of hematopoietic progenitor cells in active rheumatoid arthritis

Abstract

Objective. To examine the safety and efficacy of granulocyte colony- stimulating factor (G-CSF) alone for the mobilization of peripheral blood progenitor cells in patients with resistant active rheumatoid arthritis (RA). Methods. Five patients with resistant active RA were studied. A dose of 5 μg/kg of G-CSF (Filgrastim) was given subcutaneously each day for 5 days, and the number of stem cells mobilized into the peripheral blood was assessed by daily CD34 counts. RA disease activity was assessed by standard clinical methods. Results. The absolute numbers of peripheral blood CD34+ cells peaked on day 4, with a mean value of 0.025 x 109/liter (range 0.013-0.048 x 109/liter). There was no significant change in disease activity during the study or in the month following therapy. Conclusion. Using G-CSF alone, CD34+ progenitor peripheral blood cells were mobilized in numbers suitable for leukopheresis. G-CSF therapy was well-tolerated in patients with active RA, and was not associated with a flare during treatment or in the month following treatment.