[Clinical study of GM-CSF in patients with aplastic anemia and myelodysplastic syndrome. (CSF39-300 Study Group)].

We administered granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with aplastic anemia and myelodysplastic syndrome (MDS), as a phase III trial. The GM-CSF was given by 3 hrs intravenous drip infusion daily for at least fourteen days. Twenty-five patients with aplastic anemia and nineteen patients with MDS were evaluable for efficacy. Peripheral blood granulocyte counts, especially neutrophil counts and eosinophil counts, increased markedly by the administration of GM-CSF in each disease. Fifteen patients with MDS and nineteen patients with aplastic anemia responded to the GM-CSF. Dose-related increase of granulocytes were seen in patients with MDS, but no relation was seen in patients with aplastic anemia. Adverse effects were observed in some patients and flu-like syndrome including fever, general fatigue and anorexia were seen most commonly but were transient. Our results showed that GM-CSF is a potent stimulator of hematopoiesis in patients with aplastic anemia and MDS.