# Real-life observational cohort “TOPASE” subgroup analysis highlights efficacy of ponatinib to maintain or induce a deep molecular response in CP-CML patients

Agnes Guerci-Bresler, Ali Turhan, Philippe Rousselot, Gabriel Etienne, Marc Berger, Emilie Cayssials, Valerie Coiteux, Françoise Huguet

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## To cite this version:

Agnes Guerci-Bresler, Ali Turhan, Philippe Rousselot, Gabriel Etienne, Marc Berger, et al.. Real-life observational cohort “TOPASE” subgroup analysis highlights efficacy of ponatinib to maintain or induce a deep molecular response in CP-CML patients. European Shool of Haematology ESH, 2021, Paris, France. **hal-03462869**

HAL Id: hal-03462869

https://uca.hal.science/hal-03462869

Submitted on 2 Dec 2021

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Agnes Guerci-Bresler¹, Ali Turhan², Philippe Rousselot³, Gabriel Etienne⁴, Marc Berger⁵, Emilie Cayssials⁶, Valerie Coiteux⁷, Françoise Huguet⁸

¹Hematology, CHRU Nancy, ²APHP University Paris Saclay, Villejuif, ³Hematology, CH Versailles, ⁴Hematology, Institut Bergonie, Bordeaux,

⁵Hematology, CHU Estaing, Clermont- Ferrand, ⁶Hematology, CHU Poitiers, ⁷Hematology, CHRU Lille, ⁸Hematology, Oncopole Toulouse

## INTRODUCTION

Ponatinib is a 3rd generation tyrosine kinase inhibitor (TKI) indicated in chronic phase (CP), accelerated phase (AP) and blast phase (BP) chronic myeloid leukemia (CML) as well as in patients with the gatekeeper T315I mutation. TOPASE is an observational cohort, currently carried out in 40 CML French centers, designed to evaluate, over a two-year follow-up period, the efficacy and safety of ponatinib in routine clinical practice. The present interim analysis focused on a subgroup of 24 patients (out of 110 included) at least in major molecular response (MMR) for whom ponatinib was prescribed for intolerance to previous TKIs or for increasing the depth of MR. Data cut-off for analysis was December 2020.

## RESULTS

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## CONCLUSION

Results from this subgroup analysis highlight the high efficiency of ponatinib to maintain or induce a deep MR in CP-CML patients, with an acceptable safety profile. Further analysis on a larger population would be necessary to confirm this result.

**Keywords:** BCR-ABL, Chronic myeloid leukemia, Targeted therapy, Tyrosine kinase inhibitor