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Treatment of high or very high risk myelodysplastic syndromes

Elihu H Estey, MD
Stanley L Schrier, 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 development of acute myeloid leukemia (AML), which is often refractory to treatment. (See "Clinical manifestations and diagnosis of the myelodysplastic syndromes".)

Most patients with MDS die because of the consequences of bone marrow failure rather than development of 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".)

There is no consensus regarding a standard treatment approach for patients with symptomatic MDS, and patients should be encouraged to enroll on clinical trials whenever available. Our treatment approach incorporates knowledge of the patient’s performance status, the International Prognostic Scoring System (IPSS) (table 1) (calculator 1) and revised IPSS (IPSS-R) (table 2) (calculator 2) MDS risk categories, and other disease characteristics (ie, cytopenias present, serum erythropoietin level) to help guide management decisions.

This topic review will discuss the management of patients with MDS and a high (>4.5 to 6 points) or very high (>6 points) IPSS-R score. The treatment of patients with an intermediate (>3 to 4.5 points), low (>1.5 to 3 points), or very low (≤1.5 points) IPSS-R score, the management of the complications of MDS, details on the use of hematopoietic cell transplantation in MDS, and the prognosis of MDS are discussed separately. (See "Treatment of intermediate, low, or very low risk myelodysplastic syndromes" and "Hematopoietic cell transplantation in myelodysplastic syndromes" and "Prognosis of the myelodysplastic syndromes in adults".)

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Literature review current through: Oct 2017. | This topic last updated: Jul 07, 2017.
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