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


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.

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Author information




MB designed the study, analyzed the data, and wrote the manuscript; EA, AG, AI, PP, AI, DG, MG, MBo, FC, FS, LL, MBoc, DL, AM, NG, MC, IC, MP, and ARS collect data; RF critically revised the paper and approved the final version.

Corresponding author

Correspondence to Massimo Breccia.

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Conflict of interest

MB received honoraria for speaking by Incyte, Pfizer, Novartis, and BMS.

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Breccia, M., Abruzzese, E., Castagnetti, F. et al. Ponatinib as second-line treatment in chronic phase chronic myeloid leukemia patients in real-life practice. Ann Hematol 97, 1577–1580 (2018).

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  • Chronic myeloid leukemia
  • Ponatinib
  • Second line
  • Prognosis