Clinical outcomes in patients in any phase of <scp>CML</scp> treated with ponatinib in France—Data from the <scp>TOPASE</scp> observational study
The TOPASE study was set up to evaluate the outcomes of chronic myeloid leukaemia [CML] patients treated with ponatinib (PON) in a real-world setting in France. One hundred and twenty CML patients, 105 in chronic phase (CP), 8 in accelerated phase (AP) and 7 in blastic phase (BP) were included. Fifty-one (49%) of the CP-CML patients were in third line of treatment. The trigger for PON initiation in CP-CML was 'poor response' in 67 patients, 'poor tolerance' in 28 patients and 'response enhancement' in seven patients. The median dose at initiation was 30 mg/day [Q1; Q3 = 15; 30] in CP-CML and 45 mg/day [Q1; Q3 = 30; 45] in AP/BP-CML. Of 98 CP-CML evaluable patients, 72 (73.5%) were considered as responders (MMR) at one time point at least once, especially for those in second line of treatment and/or presenting a T315I mutation. Ninety-six of 120 (80%) patients reported at least one adverse event. An arterial occlusive event (AOE) was reported in 11 patients (9.2%). Thus, these real-life data confirm the potency of ponatinib in resistant or intolerant patients with an acceptable safety profile in non-selected patients. NCT number: NCT04048564.
Authors
- Madalina Uzunov (ORCID: https://orcid.org/0000-0002-5254-3483)
- Françoise Huguet (ORCID: https://orcid.org/0000-0002-9576-8578)
- Lydia Roy (ORCID: https://orcid.org/0000-0002-1833-9673)
- Amélie Penot
- Jean Bastié (ORCID: https://orcid.org/0000-0002-1851-1752)
- Philippe Quittet (ORCID: https://orcid.org/0000-0003-2324-5660)
- Ali G. Turhan (ORCID: https://orcid.org/0000-0002-4861-0137)
- Pascale Cony‐Makhoul (ORCID: https://orcid.org/0000-0002-1633-6479)
- Philippe Rousselot (ORCID: https://orcid.org/0000-0003-3238-4494)
- Valérie Coiteux
- Marc Berger (ORCID: https://orcid.org/0000-0003-1858-0587)
- Maya Hacini
- Gabrielle Roth-Guépin (ORCID: https://orcid.org/0009-0006-9270-9472)
- Gabriel Étienne (ORCID: https://orcid.org/0000-0001-7600-4954)
- Franck E. Nicolini (ORCID: https://orcid.org/0000-0002-3192-4199)
- Émilie Cayssials (ORCID: https://orcid.org/0000-0003-1654-5948)
- Lénaïg Le Clech (ORCID: https://orcid.org/0000-0001-5586-9772)
- Ayman Saad (ORCID: https://orcid.org/0000-0003-0003-0130)
- Bohrane Slama
- Emmanuel Messas (ORCID: https://orcid.org/0000-0003-3214-7501)
- Agnès Guerci‐Bresler
- A. Santagostino
- A. M. Agneray
Institutions
- Hôpital américain de paris (FR)
- Inserm (FR)
- Université de Bourgogne (FR)
- Université Paris-Est Créteil (FR)
- Université Clermont Auvergne (FR)
- Université Paris-Saclay (FR)
- Université de Poitiers (FR)
- Sorbonne Université (FR)
- Centre Hospitalier Universitaire de Montpellier (FR)
- Centre Hospitalier de Béziers (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
- Institut universitaire du cancer de Toulouse Oncopole (FR)
- Centre Hospitalier Régional et Universitaire de Nancy (FR)
- Hôpital Européen Georges-Pompidou (FR)
- University of Clermont Auvergne (FR)
- Centre Léon Bérard (FR)
- Hôpital Européen (FR)
- Centre Hospitalier Métropole Savoie (FR)
- Centre Hospitalier de Cornouaille (FR)
- Laboratoire d'études sur les monothéismes (FR)
- Laboratoire Informatique d'Avignon (FR)
- Pitié-Salpêtrière Hospital (FR)
- Centre Hospitalier de Troyes (FR)
- Centre de Recherche en Cancérologie de Lyon (FR)
- Centre Hospitalier Universitaire de Clermont-Ferrand (FR)
- Genopole (France) (FR)
- Centre Hospitalier Annecy Genevois (FR)
- Institut Bergonié (FR)
- Laboratoire Vision Action Cognition (FR)
- Services déconcentrés d'appui à la recherche Provence-Alpes-Côte d'Azur (FR)
- Hôpital Claude Huriez (FR)
- Centre Hospitalier de Versailles (FR)
- Bicêtre Hospital (FR)
- Université de Limoges (FR)
Publication Details
- Journal
- British Journal of Haematology
- Published
- 2024-11-06
- DOI
- https://doi.org/10.1111/bjh.19819
- Citations
- 2
- Primary Topic
- Chronic Myeloid Leukemia Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.91