Treatment options for myelodysplastic syndromes.

Cancer Control 7 but this approach is unlikely to change the natural history of the disorder. Despite the recent approval of azacitidine, supportive care alone will remain an important treatment option for many patients with MDS. Supportive care consists of transfusions for symptomatic anemia or symptomatic thrombocytopenia, treatment of infections with antibiotics,and growth factors designed to improve one or more cytopenias.1 These approaches are summarized in Table 1. Recombinant erythropoietin alfa (EPO;Procrit®,OrthoBiotech; Bridgewater, NJ) is frequently used to support the anemia in patients with MDS.1 It is important to ensure that the patient is iron replete prior to starting erythropoietin therapy. This assessment is based on an iron stain performed on the bone marrow aspirate. If iron stores are absent, a source of bleeding should be suspected and repletion is indicated. If the endogenous EPO level is >500 mIU/mL, it is unlikely that the patient will respond. If the level is <100 mIU/mL, the chance of response is approximately 30%. A trial of recombinant EPO or darbepoetin alfa (Aranesp®, Amgen Inc;Thousand Oaks, Calif) should last for at least 6 weeks. One or two attempts at dose increases may be appropriate in the event of initial failure. However, some data indicate that the addition of granulocyte colony-stimulating factor (G-CSF; Neupogen®, Amgen Inc; Thousand Oaks,Calif) at low doses (0.3–3 μg/kg per day) may potentiate the response derived from erythropoietin alone.2-5 Myelodysplastic syndromes are a heterogeneous collection of bone marrow stem cell diseases. As a result, one should recognize that an appropriate treatment option for one patient may differ significantly from that for another. The spectrum of care for myelodysplastic syndrome (MDS) ranges from simple supportive management to highly aggressive therapies such as chemotherapy and/or bone marrow transplantation. In between these two disparate options are “intermediate” therapies, which include the newly approved azacitidine (Vidaza®,Pharmion Corp;Boulder,Colo) and a host of other agents that are currently in development. These newer modalities have been promulgated based on their ability to impact one or more of the pathophysiological features believed to characterize at least a subset of patients with MDS.