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Multicenter Study
. 2025 Dec;30(1):2534196.
doi: 10.1080/16078454.2025.2534196. Epub 2025 Aug 14.

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

Timothy Devos  1 , Dries Deeren  2 , Koen Theunissen  3 , Dominik Selleslag  4 , Benjamin Bailly  5 , Violaine Havelange  6 , Philippe Lewalle  7 , Stef Meers  8 , Fleur Samantha Benghiat  9 , Alain Gadisseur  10 , Nikki Granacher  11 , Gaëtan Vanstraelen  12 , Hélène Vellemans  13 , Ann De Becker  14 , Mia Janssen  15 , Inge Vrelust  16 , Marie Lejeune  17 , Ann Van de Velde  10   18 , Agnès Triffet  19 , Michael Beck  20 , Hinde Sebti  20 , Dominiek Mazure  21
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Free article
Multicenter Study

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

Timothy Devos et al. Hematology. 2025 Dec.
Free article

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.

Keywords: Philadelphia chromosome-positive acute lymphoblastic leukemia; Ponatinib; chronic myeloid leukemia; real-world evidence; registry.

Plain language summary

What is this study about? Chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) are types of blood cancer.Ponatinib belongs to a group of medicines called tyrosine kinase inhibitors (TKIs). People with CML or Ph+ ALL can use ponatinib if they no longer benefit from or cannot tolerate treatment with at least two previous TKIs, or if their cancer cells have a gene change (or mutation) called T315I.This study looked at people with CML and Ph+ ALL being given ponatinib at Belgian hospitals over 6 years and how they responded to ponatinib treatment.What were the results? Almost 50% of people with CML and 30% of people with Ph+ ALL received treatment with three or more TKIs before ponatinib.Many people started taking ponatinib because they could not tolerate their previous TKI.86% of people with CML were alive after 48 months of ponatinib treatment. The cancer did not get any worse in 84% of people during this time.83% of people with Ph+ ALL were alive after 30 months of ponatinib treatment. The cancer did not get any worse in 54% of people during this time.33% of people with CML and 24% of people with Ph+ ALL experienced cardiovascular side effectsWhat do the results mean? This real-world study shows that people with CML and Ph+ ALL who do not respond to or cannot tolerate another TKI, or who carry the T315I mutation, may benefit from ponatinib treatment and toxicity is generally acceptable.

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