Ponatinib in chronic myeloid leukemia (CML): Consensus on patient treatment and management from a European expert panel

Crit Rev Oncol Hematol. 2017 Dec;120:52-59. doi: 10.1016/j.critrevonc.2017.10.002. Epub 2017 Oct 8.


Five tyrosine kinase inhibitors (TKIs) are currently approved in the European Union for treatment of chronic myeloid leukemia (CML) and all have considerable overlap in their indications. While disease-specific factors such as CML phase, mutational status, and line of treatment are key to TKI selection, other important features must be considered, such as patient-specific comorbidities and TKI safety profiles. Ponatinib, the TKI most recently approved, has demonstrated efficacy in patients with refractory CML, but is associated with an increased risk of arterial hypertension, sometimes severe, and serious arterial occlusive and venous thromboembolic events. A panel of European experts convened to discuss their clinical experience in managing patients with CML. Based on the panel discussions, scenarios in which a CML patient may be an appropriate candidate for ponatinib therapy are described, including presence of the T315I mutation, resistance to other TKIs without the T315I mutation, and intolerance to other TKIs.

Keywords: BCR-ABL1; Chronic myeloid leukemia; Philadelphia chromosome; Ponatinib; Tyrosine kinase inhibitor.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / therapeutic use
  • Consensus
  • Drug Resistance, Neoplasm
  • Female
  • Humans
  • Imidazoles / therapeutic use*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / enzymology
  • Male
  • Middle Aged
  • Protein Kinase Inhibitors / therapeutic use
  • Pyridazines / therapeutic use*


  • Antineoplastic Agents
  • Imidazoles
  • Protein Kinase Inhibitors
  • Pyridazines
  • ponatinib