A Phase III Trial of Subcutaneous Administration of rhG-CSF in the Myelodysplastic Syndromes

Abstract

To evaluate the safety and efficacy of subcutaneous administration of recombinant human granulocyte colony-stimulating factor in the myelodysplastic syndromes (MDS), 20 patients were given a daily dose of 50μg/m² of KRN8601 for 4 weeks. When the blood neutrophil conut did not reach 2,000/μl within 2 weeks, the dose was increased to 100μg/m². A marked neutrophilic response was obtained in 17 of the 18 evaluable patients (94.4%), irrespective of the MDS disease type. Five patients showed a platelet increase, 3 of which also showed an erythroid improvement. To maintain neutrophil levels greater than 1,000/μl, 12 patients were treated with KRN8601 for 4 weeks. A dose of 25 to 50μg/m² 3∼4 times a week served to this end in 8 patients and 100μg/m² three times a week or daily in the remaining 4 patients. One patient with RAEB progressed to acute myeloid leukemia 8 weeks after KRN8601. The treatment was well-tolerated in the majority of patients with no severe toxicities. These results suggest that subcutaneous administration of KRN8601 is safe and useful in the treatment of cytopenias in MDS.