Effects of Long-Term Treatment with Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor in Patients with Myelodysplastic Syndrome

Abstract

We examined the effects of long-term treatment with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) in 61 patients with myelodysplastic syndrome (MDS). Patients were randomly assigned to receive daily subcutaneous injection of 60 μg/m², 125 μg/m² or 250 μg/m² for 8 weeks. A significant increase in granulocyte counts including neutrophils and eosinophils was shown from one week after the start of the treatment in all three dose groups. The increase in granulocyte counts reached a plateau at the 4th week and was sustained during the treatment period. However, no consistent change in other cell lineages including monocytes, lymphocytes, reticulocytes and platelets were observed. Nevertheless peak-levels of these cells were significantly higher than the pre-treatment levels. In higher dose groups, the number of patients developing infections was reduced. There was no significant difference in the incidence of adverse events among the 3 dose groups, and the toxicity was generally well-tolerated. These observations indicate that treatment with rhCM-CSF can be of potential therapeutic benefit to patients with MDS.

Key Words:

• rhGM-CSF
• long-term treatment
• myelodysplastic syndrome