Myelodysplastic syndromes (MDS) are a mixed group of hematologic disorders with a highly variable clinical progression. As the disease develops, ineffective hematopoiesis results in peripheral blood cytopenias such as anemia or thrombocytopenia. Additional risks include transformation to acute myeloid leukemia (AML) and vary based on disease severity. As a result, the need for novel treatment options is high.1

In a review article published in Expert Review of Hematology, Ulrich Germing, MD, of the department of hematology at the University Hospital Düsseldorf in Germany, and colleagues summarized the current literature surrounding new treatment options for lower- and higher-risk patients with MDS. In addition, they reviewed several novel agents currently under investigation in clinical trials throughout the United States and European Union.

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Treatment Options for Lower-Risk MDS

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Because of a high degree of heterogeneity, prognostication for patients with MDS is challenging. Current risk stratification systems generally classify patients into lower- and higher-risk categories. Optimal treatment for patients with lower-risk disease involves correcting hematopoietic insufficiencies that often lead to symptoms secondary to peripheral blood cytopenias, including fatigue, infections, and bleeding abnormalities.

The mainstays of treatment for patients with lower-risk MDS are blood transfusions, lenalidomide, erythropoietin, and iron chelation therapy, which is used to remove excess iron attributable to chronic transfusions. Transfusion-dependent patients, in addition to those with an isolated deletion of chromosome 5q (del[5q]), often show particular benefit with lenalidomide. Some retrospective studies have also reported an overall survival benefit in lower-risk patients who respond to treatment. No evidence has indicated that amelioration of anemia alters the risk for transformation to AML or higher-risk disease.

Erythropoiesis stimulating agents (ESAs) and thrombopoietin (TPO) analogs have both shown clinical benefit for patients with lower-risk MDS. ESAs raise hemoglobin levels and correct symptoms of anemia, while TPO analogs raise platelet counts and, in some cases, reduce the need for platelet transfusions. TPO analogs such as eltrombopag and romiplostim are currently being studied for lower-risk patients with severe thrombocytopenia. The review authors recommended initiating eltrombopag in an attempt to raise platelets and prevent bleeding events when the patient’s counts are less than 20,000/μl. Under ideal circumstances, these patients should be enrolled in a clinical trial.