{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T18:50:39Z","timestamp":1790880639864,"version":"4.1.0"},"reference-count":30,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,11,6]],"date-time":"2024-11-06T00:00:00Z","timestamp":1730851200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2024,11,6]],"date-time":"2024-11-06T00:00:00Z","timestamp":1730851200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>The TOPASE study was set up to evaluate the outcomes of chronic myeloid leukaemia [CML] patients treated with ponatinib (PON) in a real\u2010world 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\u2010one (49%) of the CP\u2010CML patients were in third line of treatment. The trigger for PON initiation in CP\u2010CML was \u2018poor response\u2019 in 67 patients, \u2018poor tolerance\u2019 in 28 patients and \u2018response enhancement\u2019 in seven patients. The median dose at initiation was 30\u2009mg\/day [Q1; Q3\u2009=\u200915; 30] in CP\u2010CML and 45\u2009mg\/day [Q1; Q3\u2009=\u200930; 45] in AP\/BP\u2010CML. Of 98 CP\u2010CML 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\u2010six 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\u2010life data confirm the potency of ponatinib in resistant or intolerant patients with an acceptable safety profile in non\u2010selected patients.<\/jats:p>\n                  <jats:p>\n                    <jats:bold>NCT number<\/jats:bold>\n                    : NCT04048564.\n                  <\/jats:p>","DOI":"10.1111\/bjh.19819","type":"journal-article","created":{"date-parts":[[2024,11,7]],"date-time":"2024-11-07T02:56:39Z","timestamp":1730948199000},"page":"2295-2304","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Clinical outcomes in patients in any phase of\n                    <scp>CML<\/scp>\n                    treated with ponatinib in France\u2014Data from the\n                    <scp>TOPASE<\/scp>\n                    observational study"],"prefix":"10.1111","volume":"205","author":[{"given":"F.","family":"Huguet","sequence":"first","affiliation":[{"name":"Haematology Department, Cancer University Institute University Hospital  Toulouse France"},{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Guerci\u2010Bresler","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Haematology Department Regional University Hospital Brabois  Vandoeuvre\u2010les\u2010Nancy France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G.","family":"Roth\u2010Guepin","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Haematology Department Regional University Hospital Brabois  Vandoeuvre\u2010les\u2010Nancy France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1654-5948","authenticated-orcid":false,"given":"E.","family":"Cayssials","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Haematoloy and Cellular Therapy Department University Hospital  Poitiers France"},{"name":"CIC INSERM 1402  Poitiers France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B.","family":"Slama","sequence":"additional","affiliation":[{"name":"Hospital Centre  Avignon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Santagostino","sequence":"additional","affiliation":[{"name":"Haematology Department Hospital Centre  Troyes France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Penot","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Department of Clinical Haematology and Cellular Therapy University Hospital  Limoges France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P.","family":"Quittet","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Haematology Clinical Department University Hospital  Montpellier France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P.","family":"Cony\u2010Makhoul","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Hospital Centre  Annecy Genevois France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Saad","sequence":"additional","affiliation":[{"name":"Haematology Department Hospital Centre  B\u00e9ziers France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. N.","family":"Bastie","sequence":"additional","affiliation":[{"name":"Department of Clinical Haematology, University Hospital and INSERM, UMR 1231 University of Burgundy &amp; Franche\u2010Comt\u00e9  Dijon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Hacini","sequence":"additional","affiliation":[{"name":"Savoie Metropol Hospital Centre  Chamb\u00e9ry France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V.","family":"Coiteux","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Blood Diseases Allograft and Cell Therapy Unit, Huriez Hospital  Lille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Uzunov","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Clinical Haematology Department, Piti\u00e9\u2010Salp\u00eatri\u00e8re Hospital, APHP Sorbonne University  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1833-9673","authenticated-orcid":false,"given":"L.","family":"Roy","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Clinical Haematology Department, University Hospital Henri Mondor, AP\u2010HP &amp; Health Faculty Paris Est Cr\u00e9teil University (UPEC)  Cr\u00e9teil France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L.","family":"Le Clech","sequence":"additional","affiliation":[{"name":"Haematology Department Hospital Centre of Cornouaille  Quimper France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1858-0587","authenticated-orcid":false,"given":"M.","family":"Berger","sequence":"additional","affiliation":[{"name":"Fi LMC Centre L\u00e9on B\u00e9rard  Lyon France"},{"name":"Biological Haematology University Hospital Centre  Clermont Ferrand France"},{"name":"Host Team EA7453 CHELTER Clermont Auvergne University  Clermont\u2010Ferrand France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. 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