Hypomethylating Agents in the Treatment of High-Risk MDS


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The treatment of patients with high-risk myelodysplastic syndrome (MDS) who do not appear to respond to hypomethylating agents is challenging, given a lack of effective treatment options. If a patient does not respond to treatment following 4 to 6 cycles of azacitidine or decitabine, evidence suggests it may be beneficial to continue treatment for 9 cycles, if the patient has stable disease, suggests Rami S. Komrokji, MD.

At this point, there are only a few approved treatment options for patients with high-risk MDS, notes Ruben A. Mesa, MD. As a result, progression or intolerance are the primary drivers for switching to an alternative therapy. For patients who progress on hypomethylating agents, the overall outcome is very poor with a median survival of around 4 months, adds Elias J. Jabbour, MD.

For patients with low-risk MDS, it may be appropriate to stop treatment with a hypomethylating agent if a response is not seen after 4 to 6 cycles. In this scenario, the goal is less focused on extending survival and more to control the number of transfusions, Komrokji states.

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