title: Phase III randomized, double-blind placebo-controlled trial of rhGM-CSF following allogeneic bone marrow transplantation.
abstract: Preliminary studies in allogeneic BMT suggest that recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) is well tolerated. This is a prospective, multicenter, randomized, double-blind, placebo-controlled trial. Yeast-derived rhGM-CSF 250 micrograms/m2/day or placebo was administered by 4-hour i.v. infusion starting on the day of marrow infusion (day 0) to day 20. All patients received HLA-identical sibling marrow and cyclosporine and prednisone for GVHD prophylaxis. Fifty three patients received rhGM-CSF and 56 received placebo. Comparison of demographics revealed no differences. The time to achieve an absolute neutrophil count of > 0.5 x 10(9) cells/l was shortened in rhGM-CSF treated patients (day 13 vs. 17, P = 0.0001). The incidences of grade III-IV mucositis and infection were significantly reduced (P = 0.005, P = 0.001, respectively) and duration of hospitalization was modestly 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 observed. In conclusion, rhGM-CSF is well tolerated and reduces post-transplant morbidity in patients undergoing HLA-identical allogeneic BMT.
authors.affiliation: Texas Oncology, PA/Sammons Baylor, Dallas 75204, USA.
authors.forename: J
authors.initials: J
authors.lastname: Nemunaitis
authors.forename: C S
authors.initials: CS
authors.lastname: Rosenfeld
authors.forename: R
authors.initials: R
authors.lastname: Ash
authors.forename: M H
authors.initials: MH
authors.lastname: Freedman
authors.forename: H J
authors.initials: HJ
authors.lastname: Deeg
authors.forename: F
authors.initials: F
authors.lastname: Appelbaum
authors.forename: J W
authors.initials: JW
authors.lastname: Singer
authors.forename: N
authors.initials: N
authors.lastname: Flomenberg
authors.forename: W
authors.initials: W
authors.lastname: Dalton
authors.forename: G J
authors.initials: GJ
authors.lastname: Elfenbein
mesh_terms: D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000401:mortality; D004311:Double-Blind Method; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000209:etiology; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000009:adverse effects / Q000627:therapeutic use*; D006680:HLA Antigens / Q000276:immunology; D016298:Hematopoietic Cell Growth Factors / Q000627:therapeutic use*; D006504:Hepatic Veno-Occlusive Disease / Q000453:epidemiology / Q000209:etiology; D006801:Humans; D007239:Infections / Q000453:epidemiology; D007938:Leukemia / Q000401:mortality / Q000628:therapy; D007958:Leukocyte Count / Q000187:drug effects; D008223:Lymphoma / Q000401:mortality / Q000628:therapy; D008297:Male; D009101:Multiple Myeloma / Q000401:mortality / Q000628:therapy; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000628:therapy; D009679:Nuclear Family; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens; D016298:Hematopoietic Cell Growth Factors; D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor