Note: Some citations in the text of this section are followed by a level of evidence. The PDQ editorial boards use a formal ranking system to help the reader judge the strength of evidence linked to the reported results of a therapeutic strategy. (Refer to the PDQ summary on Levels of Evidence for more information.)

The mainstay of treatment of the myelodysplastic syndromes (MDS) has traditionally been supportive care.[1,2] Prophylactic platelet transfusion should be avoided to forestall alloimmunization, which will make platelet transfusion for bleeding difficult. Anemia should be treated with red cell transfusions regularly, and patients receiving chronic red cell transfusions should be considered for iron chelation therapy with subcutaneously administered desferrioxamine and vitamin C or oral deferasirox.[1,3] (For information on anemia, refer to the Fatigue summary.) Desferrioxamine may improve granulocyte and platelet counts in some patients, and it may reduce red cell transfusion requirements.[4] The use of erythropoietin may improve anemia. The likelihood of response to exogenous erythropoietin administration is clearly dependent on the pretreatment serum erythropoietin level and on baseline transfusion needs. In a meta-analysis summarizing the data on erythropoietin in 205 patients with MDS from 17 studies, responses were most likely in those patients who were anemic but who did not yet require a transfusion, patients who did not have ringed sideroblasts, and patients who had a serum erythropoietin level of less than 200 u/L.[5] Effective treatment requires substantially higher doses of erythropoietin than are used for other indications (150-300 µg/kg/day).

One decision model found that the likelihood of responding to growth factors was higher in patients with a low serum erythropoietin level (defined as a level <500/µL) and low transfusion needs (defined as <2 units of packed red blood cells every month), but ineffective in patients with a high erythropoietin level and high transfusion needs.[6] Some patients with poor response to erythropoietin alone may have improved response with the addition of low doses of granulocyte colony-stimulating factor (GCSF) (0.5-1.0 µg/kg/day).[7,8,9] Rates of response to the combination treatment vary with the French-American-British (FAB) classification, with responses more likely in those with refractory anemia and ringed sideroblasts RARS, and less likely for those with excess blasts.[10] Patients with RARS are unlikely to respond to erythropoietin alone.[5]

A pooled analysis of published MDS trials from 1985 to 2005 examined 1,587 patients from 83 studies of growth factors. The growth factors included recombinant human erythropoietin and GCSF. With the exclusion from analysis of patients with more advanced MDS subtypes and with standardized response criteria, an approximate 40% overall response rate to growth factors was found.[11] The use of high-dose darbepoietin (300 µg/dose weekly) has been reported to produce a major erythroid response rate of almost 50% in patients whose endogenous erythropoietin level was less than 500 u/mL.[12]