PHASE-III RANDOMIZED, DOUBLE-BLIND PLACEBO-CONTROLLED TRIAL OF RHGM-CSF FOLLOWING ALLOGENEIC BONE-MARROW TRANSPLANTATION

Preliminary studies in allogeneic BMT suggest that recombinant human g ranulocyte-macrophage colony-stimulating factor (rhGM-CSF) is well tol erated. This is a prospective, multicenter, randomized, double-blind, placebo-controlled trial. Yeast-derived rhGM-CSF 250 mu g/m(2)/day or placebo was administered by 4-hour i.v. infusion starting on the day o f marrow infusion (day 0) to day 20. All patients received HLA-identic al sibling marrow and cyclosporine and prednisone for GVHD prophylaxis . Fifty three patients received rhGM-CSF and 56 received placebo. Comp arison of demographics revealed no differences. The time to achieve an absolute neutrophil count of > 0.5 x 10(9) cells/l was shortened in r hGM-CSF treated patients (day 13 vs. 17, P = 0.0001). The incidences o f grade III-IV mucositis and infection were significantly reduced (P = 0.005, P = 0.001, respectively) and duration of hospitalization was m odestly shortened by 1 day (P = 0.02) in rhGM-CSF treated patients. No differences in platelet recovery, erythrocyte recovery, incidence of veno-occlusive disease, GVHD severity, relapse or survival were observ ed. In conclusion, rhGM-CSF is well tolerated and reduces post-transpl ant morbidity in patients undergoing HLA-identical allogeneic BMT.