title: Combination therapy with rhGM-CSF and rhEpo for two patients with refractory anemia and aplastic anemia.
abstract: Because GM-CSF possesses burst-promoting activity (BPA) and megakaryocyte colony-stimulating activity (Meg-CSF) as well as stimulating activity on granulocyte-macrophage progenitors, and erythropoietin (Epo) has thrombopoietin-like activity, the combination therapy of GM-CSF and Epo seems to be more effective for stimulating erythropoiesis and thrombocytopoiesis in patients with pancytopenia. For this reason, the combination therapy of recombinant human GM-CSF (rhGM-CSF) and rhEpo was performed in two patients with refractory anemia (RA) and aplastic anemia (AA). Epo-unresponsive anemia was remarkably improved by adding rhGM-CSF to Epo and the effect lasted for 1 1/2 months in a patient with RA, but severe anemia occurred again immediately after the discontinuation of Epo. The neutralizing antibodies against GM-CSF were not demonstrated at the phase when anemia re-progressed in this patient. In a patient with AA, anemia and thrombocytopenia, which were refractory to previous administration of rhGM-CSF, responded to the combined administration of GM-CSF and Epo. Although the effects were maintained for 3 1/2 months, the anemia and thrombocytopenia became worse again after the administration of rhGM-CSF was changed from daily to every other day. These findings suggest the usefulness of combination therapy of GM-CSF and Epo for patients with pancytopenia.
authors.affiliation: First Department of Internal Medicine, Niigata University School of Medicine, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Takahashi
authors.forename: A
authors.initials: A
authors.lastname: Aoki
authors.forename: M
authors.initials: M
authors.lastname: Mito
authors.forename: K
authors.initials: K
authors.lastname: Nikkuni
authors.forename: T
authors.initials: T
authors.lastname: Ohtsuka
authors.forename: H
authors.initials: H
authors.lastname: Saitoh
authors.forename: Y
authors.initials: Y
authors.lastname: Moriyama
authors.forename: A
authors.initials: A
authors.lastname: Shibata
mesh_terms: D000328:Adult; D000741:Anemia, Aplastic / Q000188:drug therapy*; D000753:Anemia, Refractory / Q000188:drug therapy*; D004359:Drug Therapy, Combination; D004921:Erythropoietin / Q000008:administration & dosage*; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000008:administration & dosage*; D006801:Humans; D008297:Male; D011994:Recombinant Proteins / Q000008:administration & dosage
chemical_list: D011994:Recombinant Proteins; D004921:Erythropoietin; D016178:Granulocyte-Macrophage Colony-Stimulating Factor