Scarce information is available on the use of ponatinib as second-line treatment in chronic phase chronic myeloid leukemia (CP-CML) patients resistant and/or intolerant to prior tyrosine kinase inhibitor (TKI) therapy. We collected data from 29 CML patients, with a median age of 54 years (range 32-72). Eleven patients had received dasatinib, 15 patients received nilotinib, and 3 patients received imatinib as first-line treatment. Forty-five percent of patients started ponatinib for secondary resistance, 38\% for primary resistance, 7\% for severe intolerance associated to a molecular warning, 7\% due to the presence of a T315I mutation, and 3\% for severe intolerance. Ponatinib was started at a dose of 45 mg in 60\% of patients, 30 mg in 38\%, and 15 mg in 2\% of patients. Overall, at a median follow-up of 12 months, 85\% of treated patients improved the level of response as compared to baseline, with 10 patients achieving a deep molecular response (MR4-4.5). No thrombotic events were recorded. The dose was reduced during treatment in 2 patients due to intolerance and in 8 patients in order to reduce the cardiovascular risk. Ponatinib seems a valid second-line treatment option for chronic phase CML, in particular for patients who failed a front-line second-generation TKI due to BCR-ABL-independent mechanisms of resistance.

Ponatinib as second-line treatment in chronic phase chronic myeloid leukemia patients in real-life practice / Breccia, Massimo; Abruzzese, Elisabetta; Castagnetti, Fausto; Bonifacio, Massimiliano; Gangemi, Domenica; Sorà, Federica; Iurlo, Alessandra; Luciano, Luigiana; Gozzini, Antonella; Gentile, Massimo; Bocchia, Monica; Luzi, Debora; Maggi, Alessandro; Sgherza, Nicola; Isidori, Alessandro; Crugnola, Monica; Pregno, Patrizia; Rita Scortechini, Anna; Capodanno, Isabella; Pizzuti, Michele; Robin, Foà. - In: ANNALS OF HEMATOLOGY. - ISSN 0939-5555. - 97:9(2018), pp. 1577-1580. [10.1007/s00277-018-3337-2]

Ponatinib as second-line treatment in chronic phase chronic myeloid leukemia patients in real-life practice

Massimo Breccia
Primo
;
Massimo Gentile;Robin Foà
2018

Abstract

Scarce information is available on the use of ponatinib as second-line treatment in chronic phase chronic myeloid leukemia (CP-CML) patients resistant and/or intolerant to prior tyrosine kinase inhibitor (TKI) therapy. We collected data from 29 CML patients, with a median age of 54 years (range 32-72). Eleven patients had received dasatinib, 15 patients received nilotinib, and 3 patients received imatinib as first-line treatment. Forty-five percent of patients started ponatinib for secondary resistance, 38\% for primary resistance, 7\% for severe intolerance associated to a molecular warning, 7\% due to the presence of a T315I mutation, and 3\% for severe intolerance. Ponatinib was started at a dose of 45 mg in 60\% of patients, 30 mg in 38\%, and 15 mg in 2\% of patients. Overall, at a median follow-up of 12 months, 85\% of treated patients improved the level of response as compared to baseline, with 10 patients achieving a deep molecular response (MR4-4.5). No thrombotic events were recorded. The dose was reduced during treatment in 2 patients due to intolerance and in 8 patients in order to reduce the cardiovascular risk. Ponatinib seems a valid second-line treatment option for chronic phase CML, in particular for patients who failed a front-line second-generation TKI due to BCR-ABL-independent mechanisms of resistance.
2018
Chronic myeloid leukemia; Ponatinib; Second line; Prognosis
01 Pubblicazione su rivista::01a Articolo in rivista
Ponatinib as second-line treatment in chronic phase chronic myeloid leukemia patients in real-life practice / Breccia, Massimo; Abruzzese, Elisabetta; Castagnetti, Fausto; Bonifacio, Massimiliano; Gangemi, Domenica; Sorà, Federica; Iurlo, Alessandra; Luciano, Luigiana; Gozzini, Antonella; Gentile, Massimo; Bocchia, Monica; Luzi, Debora; Maggi, Alessandro; Sgherza, Nicola; Isidori, Alessandro; Crugnola, Monica; Pregno, Patrizia; Rita Scortechini, Anna; Capodanno, Isabella; Pizzuti, Michele; Robin, Foà. - In: ANNALS OF HEMATOLOGY. - ISSN 0939-5555. - 97:9(2018), pp. 1577-1580. [10.1007/s00277-018-3337-2]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1685272
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