Hagop Kantarjian, MD, University of Texas MD Anderson Cancer Center, Houston, TX, discusses the findings of an analysis of safety and efficacy of ponatinib use in chronic-phase chronic myeloid leukemia (CML) patients who have previously failed on second-generation tyrosine kinase inhibitors (TKIs). Switching to alternative second-generation TKIs after failure has not been associated with durable outcomes, but ponatinib therapy resulted in deep and durable responses in this setting in the PACE trial (NCT01207440) of CML patients. This analysis combined patients previously treated with second-generation TKIs from the PACE trial, with CML patients from the Phase II OPTIC trial (NCT02467270), which aimed to characterize efficacy and safety of ponatinib dosing regimens. The results of 350 chronic-phase CML patients showed that response rates to ponatinib were high, with robust long-term survival outcomes. The occurrence of arterial occlusive events and serious adverse events were lower in the OPTIC cohort, where a response-adjusted dosing scheme was employed. The OPTIC trial is ongoing and lower starting doses are being investigated. The data produced from this large-scale analysis suggests ponatinib is highly effective compared to other second-generation TKIs for this patient population. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.

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