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Ponatinib for CML patients in routine clinical practice: the PONDEROSA study
Thomas Schenk1,2, Christian Fabisch3,4, Thomas Ernst3,4
1Abteilung Hämatologie und Internistische Onkologie, Klinik für Innere Medizin II, Universitätsklinikum Jena, Jena, Germany. thomas.schenk@med.uni-jena.de.
Annals of Hematology
|January 14, 2026
Summary
Ponatinib effectively treats chronic myeloid leukemia (CML), including resistant cases with the T315I mutation. The PONDEROSA study shows its real-world efficacy and safety, highlighting the need for individualized dosing to manage cardiovascular risks.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Ponatinib is a tyrosine kinase inhibitor for chronic myeloid leukemia (CML).
- Arterial occlusive events (AOEs) are a significant safety concern with ponatinib.
- The T315I BCR::ABL1 mutation presents a challenge in CML treatment.
Purpose of the Study:
- To evaluate ponatinib's effectiveness and safety in routine clinical practice.
- To assess outcomes in CML patients treated with ponatinib in Germany and the Czech Republic.
- To investigate the impact of individualized dosing on efficacy and safety.
Main Methods:
- Observational cohort study of 99 adult CML patients treated with ponatinib.
- Data collected between 2015 and 2022 across 31 centers.
- Median follow-up of 22 months, analyzing adverse events, response rates, and survival.
Main Results:
- 64.6% of patients experienced adverse events; 12.1% had severe cardiovascular/cerebrovascular events.
- 58.6% achieved major molecular response (MMR), a significant improvement from baseline (19.0%).
- Estimated 2-year progression-free survival was 84.4%, and overall survival was 85.7%.
Conclusions:
- Ponatinib demonstrates clinical effectiveness in real-world CML treatment.
- Individualized dosing is crucial for balancing ponatinib's efficacy with cardiovascular safety.
- Ponatinib serves as a valuable bridging therapy for patients awaiting allogeneic stem cell transplantation.
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