title: Phase II study of recombinant human granulocyte-macrophage colony-stimulating factor in myelodysplastic syndrome and aplastic anemia.
abstract: As phase II study, we treated 18 patients with myelodysplastic syndrome (MDS) and 37 patients with aplastic anemia (AA) with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) for 14-28 days. Administration of rhGM-CSF resulted in a dose-dependent increase in circulating granulocyte counts, which was statistically significant in patients with AA. There were no consistent changes in monocyte and lymphocyte counts. Although no increase in both thrombocyte and erythrocyte counts was detected in the majority of the patients, a response of both lineages to rhGM-CSF, in addition to granulocyte lineage, was observed in 3 patients. Drug-associated adverse events developed in 28 patients (51%). The most frequent adverse event was fever. In general, the treatment with rhGM-CSF was well tolerated. The results suggest that rhGM-CSF is effective for patients with MDS and AA.
authors.affiliation: First Department of Internal Medicine, Niigata University School of Medicine, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Takahashi
authors.forename: Y
authors.initials: Y
authors.lastname: Yoshida
authors.forename: K
authors.initials: K
authors.lastname: Kaku
authors.forename: T
authors.initials: T
authors.lastname: Masaoka
authors.forename: Y
authors.initials: Y
authors.lastname: Moriyama
authors.forename: S
authors.initials: S
authors.lastname: Nakanishi
authors.forename: T
authors.initials: T
authors.lastname: Kaneko
authors.forename: A
authors.initials: A
authors.lastname: Shibata
authors.forename: S
authors.initials: S
authors.lastname: Miwa
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000097:blood / Q000188:drug therapy*; D004804:Eosinophils / Q000473:pathology; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000009:adverse effects / Q000627:therapeutic use*; D006098:Granulocytes / Q000473:pathology; D006801:Humans; D007958:Leukocyte Count; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000188:drug therapy*; D009504:Neutrophils / Q000473:pathology; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use
chemical_list: D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor