Myelodysplastic syndromes (MDS) are a group of hematopoietic stem cell disorders characterized by ineffective hematopoeisis and an increased risk of transforming to acute myelogenous leukemia (AML). Determining the molecular basis of the disease has been hampered by its heterogeneity. Thrombocytopenia is often a manifestation of MDS and needs to be monitored and treated accordingly. Treating the underlying disorder with a variety of differentiation and immunosuppressive agents alleviates the problem in a small percentage of patients but more often complicates the issue. Several treatments used for primary immune thrombocytopenic purpura (ITP) have been tried in MDS patients, though with only modest success rates. Preliminary studies suggest that the use of a thrombopoietic growth factor may afford substantial increases in platelet levels without excessive deleterious side effects. Primary myelofibrosis (MF) is a chronic myeloproliferative disorder associated with hepatosplenomegaly and refractory cytopenias. Immunomodulatory agents have shown promise in treating the anemia associated with this MF. However, there are currently no standard therapies to treat the thrombocytopenia that is often found in patients with this disease.
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