Omacetaxine is a protein synthesis inhibitor administered subcutaneously. Elias Jabbour, MD, states that in patients who failed multiple tyrosine kinase inhibitors (TKIs), approximately 10% will experience a complete cytogenetic response with omacetaxine. The drug originally required an office visit for administration for the 14 day, twice-a-day induction schedule and eventual maintenance therapy. This made the treatment cumbersome, Jabbour states, but as of August 2014, the drug can be taken at home.

In the era of the numerous available TKIs, Jabbour sees only a minimal role for omacetaxine. He explains that he prescribes omacetaxine to patients in whom all TKIs have failed and who are in blast phase of their disease where chemotherapy may not be appropriate. Stuart L. Goldberg, MD, agrees with using the agent in patients with blast crisis who have failed TKIs. However, he cautions that it is fairly myelosuppressive and the 14-day regimen can lower the patient’s blood counts dramatically. As a result, he continues to restrict its use and views the agent as more of an end-stage approach.