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Myelodysplastic syndrome (MDS) has evolved from an entity for which no treatment options were available 10 years ago to one where several treatment options are now beneficial. These include:
• growth factors—erythropoietin (EPO) ± granulocyte colony-stimulating factor (G-CSF);
• lenalidomide—US Food and Drug Administration (FDA)-approved for low-risk MDS transfusion dependence and 5q abnormality;
• immunotherapy with anti-thymocyte globulins (ATG), cyclosporine A, and steroids;
• decitabine and azacitidine—FDA-approved for MDS and chronic myelomonocytic leukemia (CMML);
• intensive chemotherapy with topotecan and cytarabine or other antiacute myeloid leukemia (AML) regimens;
• allogeneic stem cell transplant (SCT);
• oral chelation therapy (to prevent iron overload); and
• imatinib for translocations involving 5q33.
Investigational approaches of significant potential include clofarabine, homoharringtonine, AMG531 (a thrombomimetic agent), and others. Treatment of MDS depends on patient age, prognostic risk, and comorbid conditions. The availability of these options has resulted in an increase in the documented incidence of MDS from 12,000 cases per year in the US to more than 20,000 cases. This is because more bone marrow studies are now performed in elderly patients with mild cytopenias to obtain a precise diagnosis, which may document MDS and allow early interventions.