ponatinibhas

Ponatinib is an oral multikinase inhibitor approved for the treatment of certain hematologic cancers. It primarily inhibits BCR-ABL, including forms with resistance to other therapies such as the T315I mutation, and also targets other tyrosine kinases such as PDGFR, VEGFR, FGFR, KIT, and Lck. By blocking these signaling pathways, ponatinib can reduce the growth and survival of malignant cells that rely on these kinases.

Clinically, ponatinib is used to treat chronic myeloid leukemia (CML) and Philadelphia chromosome–positive acute lymphoblastic leukemia (ALL) in adults and in selected pediatric patients, particularly those with resistance or intolerance to prior tyrosine kinase inhibitors or with specific mutations. It is generally considered when other treatments have failed or when a mutation susceptible to ponatinib is present.

Safety and monitoring are significant considerations with ponatinib. The drug carries a boxed warning for arterial occlusive events, including heart attack and stroke, as well as risks of hepatotoxicity, pancreatitis, and wound healing impairment. Patients require cardiovascular risk assessment at baseline and ongoing monitoring, with dose modifications or discontinuation in response to adverse effects or laboratory abnormalities. Hypertension, lipid abnormalities, and liver enzyme elevations are commonly monitored parameters during treatment.

Pharmacologically, ponatinib is metabolized by hepatic enzymes and is a substrate of CYP3A4. It can interact with strong CYP3A4 inhibitors or inducers, necessitating dose adjustments or avoidance of certain drug combinations. It is typically administered once daily, with or without food, as prescribed by a clinician. As with all cancer therapies, treatment decisions balance potential benefits against risks for each patient.