Ponatinib is the first generation of targeted drugs and its role in the treatment of chronic myelogenous leukemia

Ponatinib is a third-generation tyrosine kinase inhibitor (TKI) that mainly targets the BCR-ABL fusion protein, including resistance to the T315I mutation. This mutation is a common cause of drug resistance in patients with chronic myelogenous leukemia (CML) who receive first- or second-generation TKI treatment, resulting in a significant decrease in the efficacy of conventional drugs. As a third-generation TKI, ponatinib can inhibit this drug-resistant mutation and provide patients with a new treatment option.

In the treatment of chronic myelogenous leukemia, ponatinib is usually used for patients who are resistant or intolerant to first- and second-generation TKIs such as imatinib, dasatinib or nilotinib. It effectively inhibits BCR-ABL kinase activity and blocks abnormal signaling, thereby inhibiting the proliferation and survival of leukemia cells and helping patients achieve better molecular responses and remission rates. Clinical studies have shown that ponatinib is particularly effective for patients with T315I mutation-positive CML and can significantly improve their disease prognosis.

The use of ponatinib also requires strict monitoring. Although effective, it may cause serious adverse reactions such as thrombosis and cardiovascular events. Therefore, patients need to regularly monitor blood pressure, heart function and blood indicators while taking it, and assess the risk of thrombosis. Doctors will adjust the dosage and medication regimen according to the patient's specific conditions to ensure efficacy while minimizing side effects.

In general, ponatinib, as a third-generation TKI, provides an important treatment for CML patients, especially patients with drug resistance or T315I mutation. It effectively controls the progression of leukemia by precisely inhibiting BCR-ABL and its drug-resistant mutations, but it needs to be used under professional guidance and safety must be closely monitored to achieve the best therapeutic effect.

Keyword tags:

Ponatinib, Ponatinib, third generation TKI, chronic myelogenous leukemia, CML, T315I mutation, drug resistance, cardiovascular risk

References:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7172538/