Ponatinib is a third generation kinase inhibitor designed to overcome the gatekeeper T315I
mutation. In different trials this drug showed inhibitory activity against native BCR-ABL1 kinase and
several ABL1 mutations. For this reason, ponatinib is currently indicated for the treatment of chronic
myeloid leukaemia (CML) in every phase of disease resistant and/or intolerant to dasatinib and
nilotinib and for whom imatinib is not indicated anymore or for patients with T315I mutation. The drug
is also indicated for Ph+ acute lymphoblastic leukaemia (ALL). Ponatinib was temporarily suspended
in 2013 for the occurrence of cardiovascular thrombotic events. Since then, different investigators analyzed
baseline characteristics of patient candidates for ponatinib, especially cardiovascular profile, in
order to describe general management recommendations in this setting.
In this review, clinical trials data about the use of ponatinib in CML and Ph+ ALL patients will be
discussed. It will be focused also about the safety and tolerability profile of the drug and future perspectives
Keywords: Chronic myeloid leukemia, Ph+ acute lymphoblastic leukemia, ponatinib, T315I mutation, cardiovascular events,
PACE trial, EPIC trial.
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