title: Recombinant human granulocyte-macrophage colony-stimulating factor plus recombinant human erythropoietin may improve anemia in selected patients with myelodysplastic syndromes.
abstract: The purpose of this study was to improve erythropoiesis in patients with anemia due to myelodysplastic syndromes (MDS). We treated 13 patients first with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) for 6 weeks, then with recombinant human erythropoietin (rhEpo) and rhGM-CSF for the next 12 weeks. Five patients had refractory anemia (RA), 3 refractory anemia with ringed sideroblasts (RAS), and 5 refractory anemia with excess of blasts (RAEB). Ten patients were transfusion-dependent at the time of inclusion. Eleven patients completed this phase II study. Five responded with an increase in hemoglobin level (3 patients) or a reduction in transfusion requirement (2 patients). We registered no response in the remaining 6 patients during treatment. Patients responding to combined treatment had relatively low concentrations of plasma Epo and plasma ferritin before treatment with rhEpo and a normal karyotype throughout the study. Long-term bone marrow cultures did not predict the response. Still, responders seemed to have a higher number of colony-forming progenitors than nonresponders. In conclusion, combined therapy with rhGM-CSF and rhEpo may stimulate hematopoiesis and correct or improve anemia in some patients with MDS.
authors.affiliation: Department of Internal Medicine and Haematology L, University of Copenhagen, Herlev Hospital, Denmark.
authors.forename: P B
authors.initials: PB
authors.lastname: Hansen
authors.forename: H E
authors.initials: HE
authors.lastname: Johnsen
authors.forename: E
authors.initials: E
authors.lastname: Hippe
authors.forename: E
authors.initials: E
authors.lastname: Hellström-Lindberg
authors.forename: E
authors.initials: E
authors.lastname: Ralfkiaer
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics; D001853:Bone Marrow / Q000473:pathology; D002478:Cells, Cultured; D004359:Drug Therapy, Combination; D004921:Erythropoietin / Q000097:blood / Q000627:therapeutic use*; D005260:Female; D005293:Ferritins / Q000097:blood; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000627:therapeutic use*; D006412:Hematopoietic Stem Cells / Q000187:drug effects* / Q000473:pathology; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007621:Karyotyping; D007958:Leukocyte Count / Q000187:drug effects; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood* / Q000235:genetics / Q000473:pathology; D009504:Neutrophils / Q000187:drug effects; D010976:Platelet Count / Q000187:drug effects; D011994:Recombinant Proteins / Q000627:therapeutic use
chemical_list: D006454:Hemoglobins; D011994:Recombinant Proteins; D004921:Erythropoietin; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D005293:Ferritins