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Prognosis of the myelodysplastic syndromes in adults

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.

Most patients with MDS die because of the consequences of bone marrow failure, rather than transformation to AML. Thus, use of such terms 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 in the marrow were considered to have MDS by French-American-British (FAB) criteria, but AML by the World Health Organization (WHO). (See "Clinical manifestations and diagnosis of the myelodysplastic syndromes", section on 'Acute myeloid leukemia'.)

The natural history of patients with MDS is varied. This heterogeneity reflects both known and unknown differences in the pathobiology of specific subtypes within MDS and patient related characteristics, such as age and prior exposure to chemotherapy. While the pathologic classification systems (eg, French American British classification system and WHO classification system) have been valuable as benchmarks for diagnosis, they have been less useful for the determination of prognosis. As a result, further dissection of these classification schemes has been performed, taking into account variables such as age and sex, morphologic features, blast percentage, clinical characteristics, the presence of cytopenias, transfusion requirements, and cytogenetic abnormalities.

There are three main prognostic systems that have been developed and validated in patients with MDS:

International prognostic scoring system (IPSS) and a revised IPSS (IPSS-R)

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Literature review current through: Dec 2017. | This topic last updated: Jul 12, 2017.
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