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Management of the complications of the 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 progression to acute myeloid leukemia (AML), which is often refractory to standard treatment. (See "Therapy-related myeloid neoplasms: Acute myeloid leukemia and myelodysplastic syndrome".)

Most patients with MDS 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 the World Health Organization (WHO) classification describes patients with <20 percent bone marrow blasts as having MDS, while those with ≥20 percent blasts are considered to have AML.

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.

This topic will review the major complications of MDS and the role of supportive care in its management. The diagnosis of MDS, the prognosis of MDS, and the treatment of the underlying disorder are presented separately. (See "Treatment of intermediate, low, or very low risk myelodysplastic syndromes" and "Prognosis of the myelodysplastic syndromes in adults", section on 'Other prognostic features' and "Hematopoietic cell transplantation in myelodysplastic syndromes" and "Cytogenetics and molecular genetics of myelodysplastic syndromes".)


Patients with MDS typically present with a normocytic or macrocytic anemia that can vary in severity. At least 80 percent of patients are anemic at the time of diagnosis, while about 50 percent have a hemoglobin level less than 10 g/dL [1]. Anemia can manifest as fatigue, weakness, exercise intolerance, angina, dizziness, cognitive impairment, or an altered sense of wellbeing. The degree of anemia is also a prognostic marker in patients with MDS. (See "Prognosis of the myelodysplastic syndromes in adults", section on 'WHO prognostic scoring system (WPSS)'.)

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