Key clinical point: Findings from routine clinical practice are in concordance with results from clinical trials and confirm the efficacy of ponatinib in patients with chronic myeloid leukemia (CML) who were resistant or intolerant to previous tyrosine kinase inhibitors (TKIs) or had T315I mutation. No new safety signals were identified.
Major finding: During a median follow-up of 15 months, major molecular response was achieved in 58% of patients with CML. Estimated 3-year overall survival and progression-free survival were 85.3% and 81.6%, respectively. Treatment termination because of adverse events occurred in 8 patients with CML.
Study details: Findings are from a prospective observational study including 33 patients with CML and 17 patients with Philadelphia chromosome-positive acute lymphoblastic leukemia. Patients were initiated on ponatinib following intolerance, relapse, or refractoriness to prior TKIs, disease progression, or T315I mutation.
Disclosures: This study was funded by Incyte Biosciences Benelux BV. Some investigators reported research funding, advisory board memberships, consultancy, honoraria, travel expenses, and membership of the board of directors for various pharmaceutical companies including Incyte Biosciences Benelux BV.
Source: Devos T et al. Ann Hematol. 2021 May 4. doi: 10.1007/s00277-021-04507-x .