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What is new for treating patients with low-risk MDS?

By Pierre Fenaux

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Feb 8, 2021


During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, the MDS Hub spoke to Pierre Fenaux, Université de Paris, Paris, FR, about the new advances for treating patients with low-risk MDS.

What is new for treating patients with low-risk MDS?

Low-risk MDS is characterized predominantly by anemia or thrombocytopenia, fatigue, cardiovascular complications, and the need for regular transfusions. In this podcast, Fenaux discusses the new treatment options to prevent anemia. He starts with erythropoietin and its derivatives, which yield responses in about 60% of patients. He also mentions lenalidomide for patients with 5q deletion, and luspatercept, another promising drug that will soon be available in Europe. Fenaux continues with roxadustat, a hypoxia-inducible factor prolyl hydroxylase inhibitor, which showed promising results in a phase II study. He also discusses the use of hypomethylating agents, with response rates of up to 25% for patients who have mainly anemia.

For the minority of patients with predominant thrombocytopenia, the recent use of thrombopoietin receptor agonists yielded response in around 50% of cases, with limited side effects. Finally, for young, low-risk patients that do not respond to treatment and have severe thrombocytopenia, Fenaux highlights allogeneic stem cell transplantation as a treatment option.