Clinical outcomes in patients in any phase of CML treated with ponatinib in France—Data from the TOPASE observational study
Year: 2024
Type: article
Study design: Observational Study
Abstract: 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.
Source: British Journal of Haematology
DOI: 10.1111/bjh.19819
Authors: F. Huguet, A. Guerci‐Bresler, G. Roth‐Guepin, E. Cayssials, B. Slama, A. Santagostino, A. Penot, P. Quittet, P. Cony‐Makhoul, A. Saad, J. N. Bastie, M. Hacini, V. Coiteux, M. Uzunov, L. Roy, L. Le Clech, M. Berger, A. M. Agneray, E. Messas, G. Etienne, A. Turhan, F. E. Nicolini, P. Rousselot
Institutions: Institut universitaire du cancer de Toulouse Oncopole, Centre Léon Bérard, Centre Hospitalier Régional et Universitaire de Nancy, Inserm, Centre Hospitalier Universitaire de Poitiers, Centre Hospitalier de Troyes, Centre Hospitalier Universitaire de Limoges, Centre Hospitalier Universitaire de Montpellier, Centre Hospitalier Annecy Genevois, Centre Hospitalier de Béziers, Université de Bourgogne, Centre Hospitalier Métropole Savoie, Hôpital Claude Huriez, Sorbonne Université, Pitié-Salpêtrière Hospital, Université Paris-Est Créteil, Hôpitaux Universitaires Henri-Mondor, Centre Hospitalier de Cornouaille, Université Clermont Auvergne, Centre Hospitalier Universitaire de Clermont-Ferrand, Clonal Heterogeneity, Leukemic environment, ThErapy Resistance of chronic leukemias, Hôpital Européen Georges-Pompidou, Paris Cardiovascular Research Center, Institut Bergonié, Genopole (France), Bicêtre Hospital, Centre de Recherche en Cancérologie de Lyon, Université Paris-Saclay, Centre Hospitalier de Versailles
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,940
OpenAlex ID: W4404151128