Evaluating new treatment options for MDS.

Myelodysplastic syndromes (MDS) comprise a collection of hematologic disorders characterized by deficiencies in peripheral blood cells, particularly those of the granulocyte, erythrocyte, and megakaryocyte lineages. A number of chromosomal abnormalities are associated with the development of MDS, including mutations involving chromosomes 7q, 5q, 20q, 6q, 11q, and 13q. Until recently, supportive care or allogeneic stem cell transplantation (ASCT) were the only available treatment options for this disease. ASCT is potentially curative but poses high mortality risk in the predominantly elderly MDS population. Supportive care options traditionally included blood transfusions and antibiotics, with the goal of reducing morbidity from ineffective hematopoiesis and improving quality of life. This approach has been enhanced by treatment with various growth factors aimed at stimulating blood cell production, including erythropoietin and granulocyte colony-stimulating factor. These growth factors, along with the use of iron chelation therapy to counteract the iron overload that can develop after frequent transfusions, have recently been shown to have the potential to prolong survival in MDS patients. In addition to these advances in supportive care, three new agents have been approved for the treatment of MDS in the past 3 years. Lenalidomide, azacitidine, and decitabine all target molecular processes underlying the pathophysiology of MDS and have shown considerable activity and, more recently, potential survival benefits in various subgroups of patients. Considerable changes in the treatment of MDS are expected in coming years as these and other novel agents are tested alone and in combination with one another to further refine the treatment paradigm for MDS.