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Clinical outcomes in patients in any phase of CML treated with ponatinib in France—Data from the TOPASE observational study.

Authors :
Huguet, F.
Guerci‐Bresler, A.
Roth‐Guepin, G.
Cayssials, E.
Slama, B.
Santagostino, A.
Penot, A.
Quittet, P.
Cony‐Makhoul, P.
Saad, A.
Bastie, J. N.
Hacini, M.
Coiteux, V.
Uzunov, M.
Roy, L.
Le Clech, L.
Berger, M.
Agneray, A. M.
Messas, E.
Etienne, G.
Source :
British Journal of Haematology; Dec2024, Vol. 205 Issue 6, p2295-2304, 10p
Publication Year :
2024

Abstract

Summary: 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. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
00071048
Volume :
205
Issue :
6
Database :
Complementary Index
Journal :
British Journal of Haematology
Publication Type :
Academic Journal
Accession number :
181803339
Full Text :
https://doi.org/10.1111/bjh.19819