EFFECTS OF LONG-TERM TREATMENT WITH RECOMBINANT HUMAN GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR IN PATIENTS WITH MYELODYSPLASTIC SYNDROME

We examined the effects of long-term treatment with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) in 61 pati ents with myelodysplastic syndrome (MDS). Patients were randomly assig ned to receive daily subcutaneous injection of 60 mu g/m(2), 125 mu g/ m(2) or 250 mu g/m(2) for 8 weeks. A significant increase in granulocy te counts including neutrophils and eosinophils was shown from one wee k after the start of the treatment in all three dose groups. The incre ase in granulocyte counts reached a plateau at the 4th week and was su stained during the treatment period. However, no consistent change in other cell lineages including monocytes, lymphocytes, reticulocytes an d platelets were observed. Nevertheless peak-levels of these cells wer e significantly higher than the pre-treatment levels. In higher dose g roups, the number of patients developing infections was reduced, There was no significant difference in the incidence of adverse events amon g the 3 dose groups, and the toxicity was generally well-tolerated. Th ese observations indicate that treatment with rhGM-CSF can be of poten tial therapeutic benefit to patients with MDS.