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.

What was studied

This study reports 5-year results of the OPTIC trial. OPTIC was a randomized phase 2 trial of ponatinib. People had chronic-phase chronic myeloid leukemia. Their disease was resistant to other drugs or had the T315I mutation. In all, 283 people were randomized. Starting doses were 45 mg, 30 mg or 15 mg daily. In the first 2 groups, the dose was cut to 15 mg after response. At data cutoff, 61 people remained on trial.

What they found

Median follow-up was 75 to 78 months. By 5 years, response was highest in the 45-mg group. Progression-free survival was similar across groups. Overall survival was high in all groups. People with the T315I mutation had the highest response in the 45-mg group. Arterial occlusive events came to 4.1, 3.8 and 2.0 by group. Rates were per 100 patient-years. Results were similar in people with T315I.

What it means for patients

The authors say these findings support long-term benefit of response-based ponatinib dosing. They say this holds especially in people with the T315I mutation. This is in people who had earlier treatments.

Why it is not certain yet

Only 61 people remained on trial at cutoff. The abstract gives no tests of significance between the groups. The 30-mg and 15-mg groups had lower response than the 45-mg group. Arterial occlusive events still occurred in all groups. The abstract does not name a comparison drug.

Source

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.

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