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Overview of the treatment of myelodysplastic syndromes

Stanley L Schrier, MD
Elihu H Estey, MD
Section Editor
Richard A Larson, MD
Deputy Editor
Alan G Rosmarin, MD


The myelodysplastic syndromes (MDS) encompass a series of hematologic conditions characterized by chronic cytopenias (anemia, neutropenia, thrombocytopenia) accompanied by abnormal cellular maturation. As a result, patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as progression to acute myeloid leukemia (AML), which is often refractory to treatment. (See "Clinical manifestations and diagnosis of the myelodysplastic syndromes".)

Most patients with MDS, particularly those with lower risk disease, die because of the consequences of bone marrow failure rather than transformation to AML. Thus, use of terms such as "pre-leukemia" or "smoldering leukemia" can be misleading if taken to imply that death or morbidity from MDS results only when AML develops. Indeed, the distinction between MDS and AML is itself arbitrary, as patients with 20 to 30 percent blasts are considered to have MDS by French-American-British (FAB) criteria, but AML by the World Health Organization (WHO) classification.

For many years, transfusion with packed red blood cells and platelets and the use of erythropoiesis stimulating agents were the only therapy available. More recently, chemotherapy agents directed at the underlying disorder have been developed and continue to be studied for patients with MDS (eg, azacitidine, decitabine, and lenalidomide). However, due to the advanced age of most patients, the chronicity of the disease, and its attendant morbidities, supportive care remains a central component of the management of all patients with MDS. Patients should be treated as needed with antibiotics for infection and platelet transfusions for bleeding in the setting of thrombocytopenia. (See "Management of the complications of the myelodysplastic syndromes".)

A general overview of the treatment of MDS will be reviewed here. More detailed discussions of the following issues are presented separately:

Treatment of very low, low or intermediate risk MDS (see "Treatment of intermediate, low, or very low risk myelodysplastic syndromes")

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Literature review current through: Nov 2017. | This topic last updated: Jun 07, 2017.
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