CLINICAL USE OF HEMATOPOIETIC GROWTH-FACTORS IN THE MYELODYSPLASTIC SYNDROMES

Recent advances in the molecular genetics of myelodysplastic syndromes (MDS) have shed new light on the pathogenesis of MDS allowing a bette r understanding of the defects of differentiation of the transformed c lone and suppression of normal hematopoiesis. The clinical hematologis t, however, continues to be challenged with the treatment of patients with MDS. Pancytopenia and defective function of neutrophils and plate lets lead to a high risk of infectious and hemorrhagic complications. The progression to acute myeloid leukemia adds to morbidity and mortal ity. Supportive care including red blood cell and platelet transfusion s are still the cornerstone of therapeutic management. While prophylac tic administration of G-CSF or GM-CSF cannot be recommended, treatment of febrile neutropenia might benefit from administration of G-CSF in addition to antibiotics. Administration of high-dose erythropoietin wi ll improve erythropoiesis in around 20% of the patients, mainly in tho se with rather preserved erythroid function and no or low transfusion need. Coadministration of erythropoietin with either G-CSF or GM-CSF c ould increase the response rate. Allogeneic stem cell transplantation still is the only curative treatment and prolongs survival. Intensive chemotherapy for advanced MDS is possible with an acceptably low rate of early death and a complete remission rate between 45% to 60%, while initial results of autologous transplantation are promising.