Ponatinib treatment for leukemia – expert recommendation consensus

This review aimed to look at ponatinib treatment for chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia with a focus on cardiovascular management.  

This review concluded that ponatinib can lead to increased cardiovascular events and other treatments should be used where available.  

Ponatinib (Iclusig) is a targeted therapy used as a treatment for chronic myeloid leukemia (CML) and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Ponatinib is a tyrosine kinase inhibitor (TKI). Most patients with CML achieve long-term survival similar to individuals without CML due to TKI treatment.  

Ponatinib has shown to be effective in patients with unresponsive CML and Ph+ ALL. In previous studies, ponatinib has shown a dose-dependent, increased risk of serious cardiovascular (CV) events. 

There is a need to evaluate the benefit-risk profile of TKIs such as ponatinib. There is also a need to evaluate safety measures to prevent treatment-associated CV events. 

This review summarized the current evidence regarding the safety and effectiveness of ponatinib. It also proposed CV management strategies for patients who are candidates for ponatinib.  

Studies have shown that ponatinib is a good therapeutic option in patients with CML or Ph+ ALL who do not respond to other TKIs. The benefit-risk balance should always be evaluated for each individual patient.  

Treatment with ponatinib and other TKIs leads to an increased CV risk. This seems to be dose-dependent and can be made less severe by changing doses and managing CV conditions. The European guidelines on CV disease prevention in clinical practice can be applied for CV risk assessment and management in patients with CML and Ph+ ALL.  

For high- and very high-risk patients, other treatment options should be considered before starting treatment with ponatinib. When other options are not available, treatment with ponatinib can be used but requires close monitoring. 

This review concluded that ponatinib can lead to increased cardiovascular events and other treatments should be used where available.