title: Treatment of refractory aplastic anemia with recombinant human granulocyte-macrophage-colony-stimulating factor.
abstract: Fifteen patients with refractory aplastic anemia or agranulocytosis received treatment with recombinant human granulocyte-macrophage-colony-stimulating factor (rhGM-CSF) in doses from 4 to 64 micrograms/kg/d by continuous intravenous (IV) infusion. Ten of 11 evaluable patients with aplastic anemia had substantial increments in granulocytes, monocytes, and eosinophils associated with myeloid and eosinophilic hyperplasia in the bone marrow. Patients with pretreatment granulocytes greater than 0.3 x 10(9)/L had greater increments in circulating myeloid cells than patients with more severe granulocytopenia. Only one patient had improvement in erythrocytes and platelets. Blood counts fell to baseline after rhGM-CSF treatment was discontinued. Doses up to 16 micrograms/kg/d were relatively well tolerated in the absence of extreme leukocytosis. Fatigue and myalgia were common. Three patients developed pulmonary infiltrates that resolved with discontinuation of treatment. Patients tended to have recurrent inflammation in previously diseased tissues. These data indicate that rhGM-CSF will increase circulating granulocytes, monocytes, and eosinophils in patients with refractory aplastic anemia. Further studies are necessary to determine if rhGM-CSF treatment will reduce morbidity or improve survival.
authors.affiliation: UCLA Center for Health Sciences 90024-1678.
authors.forename: R E
authors.initials: RE
authors.lastname: Champlin
authors.forename: S D
authors.initials: SD
authors.lastname: Nimer
authors.forename: P
authors.initials: P
authors.lastname: Ireland
authors.forename: D H
authors.initials: DH
authors.lastname: Oette
authors.forename: D W
authors.initials: DW
authors.lastname: Golde
mesh_terms: D000753:Anemia, Refractory / Q000188:drug therapy* / Q000473:pathology; D001853:Bone Marrow / Q000473:pathology; D003115:Colony-Stimulating Factors / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D006133:Growth Substances / Q000627:therapeutic use*; D006410:Hematopoiesis; D006801:Humans; D007958:Leukocyte Count; D011994:Recombinant Proteins; D013997:Time Factors
chemical_list: D003115:Colony-Stimulating Factors; D006133:Growth Substances; D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor