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Observational Study
. 2024 Dec;205(6):2295-2304.
doi: 10.1111/bjh.19819. Epub 2024 Nov 6.

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

F Huguet  1   2 , A Guerci-Bresler  2   3 , G Roth-Guepin  2   3 , E Cayssials  2   4   5 , B Slama  6 , A Santagostino  7 , A Penot  2   8 , P Quittet  2   9 , P Cony-Makhoul  2   10 , A Saad  11 , J N Bastie  12 , M Hacini  13 , V Coiteux  2   14 , M Uzunov  2   15 , L Roy  2   16 , L Le Clech  17 , M Berger  2   18   19 , A M Agneray  20 , E Messas  21 , G Etienne  2   22 , A Turhan  23   24   25   26 , F E Nicolini  2   27 , P Rousselot  2   28   29
Affiliations
Observational Study

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

F Huguet et al. Br J Haematol. 2024 Dec.

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.

Keywords: CML; TKI; ponatinib; real‐life setting.

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Conflict of interest statement

FH is a consultant and speaker for Incyte Biosciences, Novartis, Pfizer. EC received honoraria from Novartis and Incyte. PCM is a speaker for Incyte. MH received grant from Incyte. VC is a board member and consultant for Incyte, Novartis and Pfizer. AMA is an employee of INCYTE France. AT is a consultant for Novartis, Bristol Myers Squibb and Abbvie. AT is also member of advisory Board for Novartis and is a speaker for Abbvie. FEN is an advisory board member for Novartis Pharma, Incyte Biosciences, Speaker for Incyte Biosciences, Novartis, Consultant for Novartis, Sun Pharma Ltd. FEN also received Institutional grants from Incyte Biosciences and Novartis. PR received research grant from Incyte. EM is a consultant for Incyte Biosciences, Novartis, Bayer and Pfizer. AGB, MGB, PR, VC, GE, AT and EC received financial support from Incyte to participate in the study's scientific committee. FH was specially commissioned to provide a critical review of the trial results. All other authors had no competing interest to disclose.

Figures

FIGURE 1
FIGURE 1
(A) Best response achieved in all patients in CP phase according to ponatinib treatment line. *The level of response was reported quantitatively by the investigators according to the following list: ‘no response’, ‘CHR’, ‘no MMR’, ‘MMR’, ‘MR4.0’ or ‘MR4.5 and beyond’ and according to their usual practice (approximately every 3 months the first year, and then every 6 months until at least 2 years of follow‐up). (B) 12‐month probability of achieving MMR in CP‐CML patients not in MMR at baseline according to ponatinib treatment line.
FIGURE 2
FIGURE 2
(A) Best response achieved in all CP‐CML patients according to mutational status at ponatinib treatment initiation. (B) 12‐month probability of achieving MMR in CP‐CML patients not in MMR at baseline according to mutational status at ponatinib treatment initiation.
See this image and copyright information in PMC

References

    1. Hochhaus A, Baccarani M, Silver RT, Schiffer C, Apperley JF, Cervantes F, et al. European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia. Leukemia. 2020;34(4):966–984. 10.1038/s41375-020-0776-2 - DOI - PMC - PubMed
    1. Hochhaus A, Saussele S, Rosti G, Mahon FX, Janssen JJWM, Hjorth‐Hansen H, et al. Chronic myeloid leukaemia: ESMO clinical practice guidelines for diagnosis, treatment and follow‐up. Ann Oncol. 2017;28(suppl_4):iv41–iv51. 10.1093/annonc/mdx219 - DOI - PubMed
    1. Asciminib EMA authorisation and product characteristics information. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/scemblix
    1. Bixby D, Talpaz M. Mechanisms of resistance to tyrosine kinase inhibitors in chronic myeloid leukemia and recent therapeutic strategies to overcome resistance. Hematology Am Soc Hematol Educ Program. 2009;2009:461–476. 10.1182/asheducation-2009.1.461 - DOI - PubMed
    1. Kantarjian HM, Giles FJ, Bhalla KN, Pinilla‐Ibarz J, Larson RA, Gattermann N, et al. Nilotinib is effective in patients with chronic myeloid leukemia in chronic phase after imatinib resistance or intolerance: 24‐month follow‐up results. Blood. 2011;117(4):1141–1145. 10.1182/blood-2010-03-277152 - DOI - PMC - PubMed

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