Five-year follow-up of OPTIC: long-term efficacy, safety, and mutation analyses of ponatinib in chronic-phase chronic myeloid leukemia from a randomized phase 2 trial.
LeukemiaJul 24, 2026 (epub)
Hagop Kantarjian, Michael Deininger, Jane F Apperley et al.
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In 30 seconds
This phase 2 randomized trial evaluated the long-term efficacy and safety of ponatinib in patients with chronic-phase chronic myeloid leukemia (CP-CML) over five years. Among 283 patients, 60% of those on 45 mg achieved ≤1% BCR::ABL1 IS, with overall survival rates exceeding 80%. The study highlights the benefits of response-based dosing.
Key findings
- By five years, 60% of patients on 45 mg achieved ≤1% BCR::ABL1 IS.
- Five-year progression-free survival rates were 63%, 57%, and 60% for the 45 mg, 30 mg, and 15 mg cohorts, respectively.
- Overall survival rates exceeded 80% across all cohorts.
- Exposure-adjusted rates of arterial occlusive events were 4.1, 3.8, and 2.0 per 100 patient-years by cohort.
Why it matters
The findings underscore the long-term benefits of response-based ponatinib dosing in managing CP-CML, particularly for patients with T315I mutations, which may influence treatment strategies in this population.