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
Year: 2025
Type: article
Study design: Observational Study
Abstract: 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 T315I 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 T315I mutation.Trial registration: ClinicalTrials.gov identifier: NCT03678454; September 19, 2018.
Source: Hematology
Authors: Timothy Devos, Dries Deeren, Koen Theunissen, Dominik Selleslag, Benjamin Bailly, Violaine Havelange, Philippe Lewalle, Stef Meers, Fleur Samantha Benghiat, Alain Gadisseur, Nikki Granacher, Gaëtan Vanstraelen, Hélène Vellemans, Ann De Becker, Mia Janssen, Inge Vrelust, Marie Lejeune, Ann Van de Velde, Agnès Triffet, Michael Beck, Hinde Sebti, Dominiek Mazure
Institutions: KU Leuven, AZ Delta, Jessa Hospital, AZ Sint-Jan, Hôpital de Jolimont, University College London, Université Libre de Bruxelles, Institut Jules Bordet, AZ Klina, Erasmus Hospital, Antwerp University Hospital, Ziekenhuisnetwerk Antwerpen Stuivenberg, CHR Verviers, CHU Dinant Godinne UCL Namur, Vrije Universiteit Brussel, Universitair Ziekenhuis Brussel, Ziekenhuis Oost-Limburg, Centre Hospitalier Universitaire de Liège, Algemeen Ziekenhuis Sint-Maria Halle, Ghent University Hospital
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,557
Funder: Incyte
OpenAlex ID: W4413137391