Treatment of MDS Refractory to Hypomethylating Agent

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Few treatment options exist for patients with myelodysplastic syndromes (MDS) who progress on hypomethylating agents, such as azacitidine and decitabine. In this setting, for patients with high-risk MDS, the median survival is 4 to 6 months, states Elias J. Jabbour, MD. The best option at this point is a clinical trial.

For patients with low-risk MDS, the median survival for patients following progression on hypomethylating agents is nearly 17 months, Jabbour states. Moreover, the rate of transformation to acute myeloid leukemia is low. In this setting, the most effective options appear to be bone marrow transplant or a clinical trial, adds Rami S. Komrokji, MD.

Numerous clinical studies have examined commercially available agents and combinations following progression, notes Ruben A. Mesa, MD. However, none of these data have transformed the standard of care, since it is unclear if one combination is superior to another. Moreover, none of the trials have resulted in FDA approval.

A clinical trial following progression on hypomethylating agents is the clear favorite of the panel. Unfortunately, access to trials is not always feasible, particularly in the community setting, adds Harry P. Erba, MD, PhD. This fact places more importance on rapidly discovering an optimal and widely available approach.