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 T315I mutation....
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Taylor & Francis Group
2025-12-01
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| Series: | Hematology |
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| Online Access: | https://www.tandfonline.com/doi/10.1080/16078454.2025.2534196 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Timothy Devos |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-6ff7024da9b541cd8a416d6b99e18500 |
| institution | Kabale University |
| issn | 1607-8454 |
| language | English |
| publishDate | 2025-12-01 |
| publisher | Taylor & Francis Group |
| record_format | Article |
| series | Hematology |
| spelling | doaj-art-6ff7024da9b541cd8a416d6b99e185002025-08-20T04:01:03ZengTaylor & Francis GroupHematology1607-84542025-12-0130110.1080/16078454.2025.2534196Real-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 registryTimothy Devos0Dries Deeren1Koen Theunissen2Dominik Selleslag3Benjamin Bailly4Violaine Havelange5Philippe Lewalle6Stef Meers7Fleur Samantha Benghiat8Alain Gadisseur9Nikki Granacher10Gaëtan Vanstraelen11Hélène Vellemans12Ann De Becker13Mia Janssen14Inge Vrelust15Marie Lejeune16Ann Van de Velde17Agnès Triffet18Michael Beck19Hinde Sebti20Dominiek Mazure21Department of Hematology, University Hospitals Leuven and Laboratory of Molecular Immunology (Rega Institute), KU Leuven, Leuven, BelgiumAlgemeen Ziekenhuis Delta, Roeselare, BelgiumJessa Ziekenhuis, Hasselt, BelgiumAlgemeen Ziekenhuis Sint-Jan Brugge, Brugge, BelgiumHôpital de Jolimont, Haine-Saint-Paul, BelgiumUCL Saint-Luc, Woluwe-Saint-Lambert, BelgiumInstitut Jules Bordet, Université Libre de Bruxelles, Bruxelles, BelgiumAlgemeen Ziekenhuis Klina, Brasschaat, BelgiumHUB Hôpital Erasme, ULB, Brussels, BelgiumUniversitair Ziekenhuis Antwerpen, Edegem, BelgiumZiekenhuis Netwerk Antwerpen Stuivenberg, Antwerpen, BelgiumCHR Verviers, Verviers, BelgiumCHU UCL Namur, Site Godinne, Yvoir, BelgiumDepartment of Hematology, Vrije Universiteit Brussel (VUB), Universitair Ziekenhuis Brussel (UZ Brussel), Brussels, BelgiumZiekenhuis Oost-Limburg, Genk, BelgiumAlgemeen Ziekenhuis Turnhout, Turnhout, BelgiumCentre Hospitalier Universitaire de Liège (Sart Tilman), Liège, BelgiumUniversitair Ziekenhuis Antwerpen, Edegem, BelgiumCentre Hospitalier Universitaire Charleroi Vésale, Charleroi, BelgiumIncyte Biosciences Benelux BV, Amsterdam, The NetherlandsIncyte Biosciences Benelux BV, Amsterdam, The NetherlandsUniversitair Ziekenhuis Gent, Gent, BelgiumBackground: 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.https://www.tandfonline.com/doi/10.1080/16078454.2025.2534196Ponatinibreal-world evidenceregistryPhiladelphia chromosome-positive acute lymphoblastic leukemiachronic myeloid leukemia |
| spellingShingle | 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 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 Hematology Ponatinib real-world evidence registry Philadelphia chromosome-positive acute lymphoblastic leukemia chronic myeloid leukemia |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | Ponatinib real-world evidence registry Philadelphia chromosome-positive acute lymphoblastic leukemia chronic myeloid leukemia |
| url | https://www.tandfonline.com/doi/10.1080/16078454.2025.2534196 |
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