Real-world outcomes in patients with Philadelphia chromosome-positive acute lymphoblastic leukemia or chronic myeloid leukemia treated with ponatinib - final 6-year results from a Belgian registry.

[BACKGROUND] Ponatinib is a third-generation tyrosine kinase inhibitor (TKI) for treatment of chronic myeloid leukemia (CML) and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) in patients who fail or are intolerant to a second generation TKI or who carry the mutation.

[METHOD] This is the final analysis of the Belgian ponatinib registry evaluating use of ponatinib in clinical practice, with data available for up to 6 years after reimbursement.

[RESULT] Forty-eight percent of 54 CML and 28% of 29 Ph+ ALL patients had received ≥3 previous TKIs. Before ponatinib, most patients had already achieved a response, including at least a major molecular response (MMR), in 19% of CML and 17% of Ph+ ALL patients. Ponatinib was initiated due to intolerance to previous TKIs in 50% of CML and 41% of Ph+ ALL patients. Median follow-up was 545 and 258 days for CML and Ph+ ALL patients, respectively. Best response to ponatinib was at least an MMR in 65% of CML and 55% of Ph+ ALL patients. Overall and progression-free survival were 85.8% and 83.8% in CML patients after 48 months of treatment, and 82.5% and 54.2% in Ph+ ALL patients after 30 months of treatment. Adverse reactions were reported by 85% of CML and 76% of Ph+ ALL patients, with 33% of CML and 24% of Ph+ ALL patients experiencing cardiovascular events.

[CONCLUSION] In line with previously published trials, these real-world data support use of ponatinib in CML and Ph+ ALL patients with resistance or intolerance to previous TKIs or carrying the mutation. ClinicalTrials.gov identifier: NCT03678454; September 19, 2018.