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

- Ponatinib has shown efficacy in CML patients who have received prior TKIs.
- Disease state, mutational status, and line of treatment are key to TKI selection.
- Patient-specific comorbidities and TKI safety profiles should also be considered.
- The risk-benefit balance should always be evaluated for each individual patient.
- Results from ongoing clinical trials are expected to provide further information.

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.