Ponatinib Dose-Ranging Study Pinpoints Optimal Dose for Treating Chronic Myeloid Leukemia

Phase 2 Trial Identifies Dosage Strategy to Maximize Efficacy and Reduce Risk of Arterial Occlusions

Investigators with Hackensack University Medical Center recently participated in the first study to evaluate a novel, response-based, dose-reduction strategy for tyrosine kinase inhibitor (TKI) treatment of chronic-phase chronic multiple myeloma (CP-CML).

Published in Blood, the study assessed the benefit/risk ratio for three ponatinib starting doses, 45 mg, 30 mg and 15 mg, with the goal of helping physicians better manage the potential significant adverse effects of arterial occlusion that can occur with TKIs. All three starting doses resulted in clinical benefit for individuals with resistant CP-CML. The study showed the best outcome balancing benefit and adverse event risk, though, with a starting dose of 45 mg, which was decreased to 15 mg upon response.

While most patients with CP-CML experience good long-term outcomes of TKI treatment, a significant percentage experience ineffectiveness or adverse effects. In analysis of previous research, investigators saw potential for ponatinib’s effectiveness in patients whose CP-CML was resistant to prior generation TKIs.

They observed, however, that the benefit came with risk of a serious side effect, arterial occlusion events. By conducting this most recent study, they demonstrated the proper dose strategy to manage this risk while preserving the treatment’s strong, durable efficacy.

Learn more about cancer care breakthroughs at Hackensack University Medical Center.