Background and methods: In 38 patients with myelodysplastic syndromes (MDS) the values of serum erythropoietin were measured at diagnosis and compared with the haemoglobin level. A highly significant inverse relationship was found between these two parameters, suggesting that the physiologic mechanism of erythroid progenitor cell recruitment is preserved in MDS. Fourteen transfusion-dependent patients were treated with recombinant human erythropoietin at the dose of 150 U/Kg three times weekly for at least 2 months.
Results and conclusions: Under recombinant human erythropoietin, three patients became transfusion-independent and 5 had a transient decrease of their transfusion requirement. Two patients under prolonged treatment at the same dose of erythropoietin remain in complete remission after 12 and 15 months respectively. A direct relationship between response to erythropoietin treatment and degree of bone marrow erythroid hyperplasia, coupled to an inverse correlation between response to erythropoietin and baseline serum erythropoietin levels were noted. Based on these findings, recombinant human erythropoietin may represent an effective treatment modality for selected patients with MDS.