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
{"title":"法国使用泊纳替尼治疗任何阶段CML患者的临床结果--来自TOPASE观察性研究的数据。","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","doi":"10.1111/bjh.19819","DOIUrl":null,"url":null,"abstract":"<p>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.</p><p><b>NCT number</b>: NCT04048564.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"205 6","pages":"2295-2304"},"PeriodicalIF":5.1000,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11637719/pdf/","citationCount":"0","resultStr":"{\"title\":\"Clinical outcomes in patients in any phase of CML treated with ponatinib in France—Data from the TOPASE observational study\",\"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\",\"doi\":\"10.1111/bjh.19819\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>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.</p><p><b>NCT number</b>: NCT04048564.</p>\",\"PeriodicalId\":135,\"journal\":{\"name\":\"British Journal of Haematology\",\"volume\":\"205 6\",\"pages\":\"2295-2304\"},\"PeriodicalIF\":5.1000,\"publicationDate\":\"2024-11-06\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11637719/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"British Journal of Haematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1111/bjh.19819\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"British Journal of Haematology","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1111/bjh.19819","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Clinical outcomes in patients in any phase of CML treated with ponatinib in France—Data from the TOPASE 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.
期刊介绍：
The British Journal of Haematology publishes original research papers in clinical, laboratory and experimental haematology. The Journal also features annotations, reviews, short reports, images in haematology and Letters to the Editor.
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