Long-term administration of pegylated recombinant human megakaryocyte growth and development factor dramatically improved cytopenias in a patient with myelodysplastic syndrome.

To date, there are no curative therapeutic options for patients with myelodysplastic syndromes (MDS) other than allogeneic stem cell transplantation. We treated an MDS patient with 10 microg/kg pegylated recombinant human megakaryocyte growth and development factor (rHuMGDF) for more than 450 d. The patient's platelet counts increased from <10 x 10(9)/l to 50 x 10(9)/l. Interestingly, haemoglobin levels increased dramatically and reached over 13 g/dl without additional transfusion. Adverse events and neutralizing antibodies were not observed during treatment, suggesting that the long-term administration of rHuMGDF might be of clinical benefit to patients with MDS.