Patients with newly diagnosed chronic myeloid leukemia (CML) in chronic phase respond well to treatment with ponatinib, but due to the risk of vascular thrombotic events and the availability of alternative options, other drugs should be considered before ponatinib in the frontline setting, a recent study published online ahead of print in the journal The Lancet Haematology has shown.

For the phase 2 study, researchers sought to assess the activity and safety of ponatinib, a multi-targeted tyrosine kinase inhibitor, as first-line treatment for patients with chronic phase CML.

Researchers enrolled 51 patients with early chronic phase CML. Of those, 43 patients received ponatinib 45 mg daily and eight patients were started on ponatinib 30 mg daily.

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After a warning by the U.S. Food and Drug Administration (FDA) for vascular complications with ponatinib, all patients received aspirin 81 mg daily and were dose reduced to ponatinib 30 or 15 mg daily.

Results showed that 94% of evaluable patients achieved complete cytogenetic response at 6 months.

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In regard to safety, 69%, 63%, and 49% of patients experienced skin-related effects, elevated lipase, and cardiovascular events, respectively. Grade 3 to 4 myelosuppression was reported in 29% of patients.

Ultimately, the study was stopped early after the FDA warned of the increased risk for thromboembolism with ponatinib.


  1. Jain P, Kantarjian H, Jabbour E, et al. Ponatinib as first-line treatment for patients with chronic myeloid leukemia in chronic phase: a phase 2 study. Lancet Haematol. 2015. [epub ahead of print]. doi: 10.1016/S2352-3026(15)00127-1.