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

Massimo Breccia, Elisabetta Abruzzese, Fausto Castagnetti, Massimiliano Bonifacio, Domenica Gangemi, Federica Sorà, Alessandra Iurlo, Luigiana Luciano, Antonella Gozzini, Massimo Gentile, Monica Bocchia, Debora Luzi, Alessandro Maggi, Nicola Sgherza, Alessandro Isidori, Monica Crugnola, Patrizia Pregno, Anna Rita Scortechini, Isabella Capodanno, Michele PizzutiRobin Foà

Research output: Contribution to journalArticle


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.

Original languageEnglish
Pages (from-to)1577-1580
Number of pages4
JournalAnnals of Hematology
Issue number9
Publication statusPublished - Sep 2018



  • Adult
  • Aged
  • Chemotherapy, Adjuvant
  • Dose-Response Relationship, Drug
  • Drug Resistance, Neoplasm/drug effects
  • Female
  • Humans
  • Imidazoles/therapeutic use
  • Leukemia, Myeloid, Chronic-Phase/drug therapy
  • Male
  • Middle Aged
  • Practice Patterns, Physicians'/statistics & numerical data
  • Pyridazines/therapeutic use
  • Retrospective Studies

Cite this

Breccia, M., Abruzzese, E., Castagnetti, F., Bonifacio, M., Gangemi, D., Sorà, F., Iurlo, A., Luciano, L., Gozzini, A., Gentile, M., Bocchia, M., Luzi, D., Maggi, A., Sgherza, N., Isidori, A., Crugnola, M., Pregno, P., Scortechini, A. R., Capodanno, I., ... Foà, R. (2018). Ponatinib as second-line treatment in chronic phase chronic myeloid leukemia patients in real-life practice. Annals of Hematology, 97(9), 1577-1580. https://doi.org/10.1007/s00277-018-3337-2