Jorge Cortes, MD
Ponatinib (Iclusig) maintained deep, durable responses in heavily pretreated patients with chronic-phase chronic myeloid leukemia (CP-CML), according to 5-year results from the pivotal PACE trial published in the journal Blood
Overall, the PACE trial included 449 patients who received ponatinib at a starting dose of 45 mg daily. Patients were heavily pretreated, with an estimated 93% and 56% of patients having previously received ≥2 TKIs and ≥3 TKIs, respectively.
At a minimum of 48 months’ follow-up (data cutoff: August 3, 2015), 110 of 270 CP-CML patients continued to receive ponatinib. The median durations of MCyR (range 2.7 to >50 months) and MMR (range 1.7 to >50 months) had not yet been reached.
In the overall 449-patient population, 150 patients had experienced AOEs at 4 years. Some patients had more than 1 type of AOE, with cardiac vascular, peripheral vascular, and cerebrovascular arterial occlusive events occurring in 21%, 12%, and 9% of patients, respectively. Arterial occlusive serious adverse reactions were reported for 22% of patients, including 12% cardiac vascular, 8% peripheral vascular, and 7% cerebrovascular. Venous thromboembolic events were reported in 6% of patients.
Ponatinib initially received an accelerated approval from the FDA in 2012 based on earlier findings from the PACE trial.
- Cortes JE, Kim DW, Pinilla-Ibarz J, et al. Ponatinib efficacy and safety in Philadelphia chromosome-positive leukemia: final 5-year results of the phase 2 PACE trial [published online March 22, 2018]. Blood. doi: https://doi.org/10.1182/blood-2016-09-739086.
- Cortes JE, Pinilla-Ibarz J, Le Coutre PD, et al. 4-year results of the ponatinib phase II PACE trial in patients (pts) with heavily pretreated leukemia. J Clin Oncol 34, 2016 (suppl; abstr 7013)..