Purpose of Review
Myelodysplastic syndrome (MDS) is a clinically and molecularly heterogeneous disease, which primarily occurs in older adults. Although hypomethylating agents have survival benefit and are the current standard of care, many MDS patients will not garner a response from therapy. For those who do respond, most responses are not durable, and the only hope for a cure is allogeneic stem cell transplant. New therapies to improve outcomes are urgently needed.
Clinical trials combining standard hypomethylating agents with novel experimental agents are underway in an effort to improve clinical outcomes in MDS patients. Several of these small molecules have demonstrated the ability to augment the response rates of hypomethylating agents alone, including complete remission rates, in both the front line and refractory settings.
Combination approaches utilizing hypomethylating agents and novel-targeted therapies have demonstrated the ability to improve response rates in MDS patients in both the front line and salvage settings, and thus may change the standard of care.
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Conflict of Interest
The authors declare that they have no conflict of interest.
Human and Animal Rights and Informed Consent
This article does not contain any studies with human or animal subjects performed by any of the authors.
This article is part of the Topical Collection on Myelodysplastic Syndromes
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Bradley, T.J., Watts, J.M. & Swords, R.T. Leveraging Hypomethylating Agents for Better MDS Therapy. Curr Hematol Malig Rep 13, 507–515 (2018). https://doi.org/10.1007/s11899-018-0477-3
- Myelodysplastic syndrome
- Acute myeloid leukemia
- Hypomethylating agents
- Isocitrate dehydrogenase