Ponatinib (Iclusig®)

Ponatinib is a tyrosine kinase inhibitor (TKI) that blocks the action of the BCR-ABL protein. It is only used in patients with CML if the other TKIs haven’t worked or if there is a T315I mutation (gene change) in the BCR-ABL protein. This mutation can occur in some CML patients and the mutation can prevent other TKIs from working. This drug can be used to treat: Chronic, Accelerated or Blast Phase, or T315I+ CML.

Side Effects

Common Side Effects:

These side effects are common when patients start therapy but are usually mild to moderate. Most of these side effects improve over time but it is important to tell your doctor about any side effects you may be experiencing. Supportive medications can help with symptoms from these side effects and often, the TKI doses do not need to be modified.

  • Diarrhea
  • Muscle pain/aches
  • Edema (fluid buildup) around the eyes, feet, or abdomen
  • Pruritus (itchy skin) and skin rashes
  • Fatigue
  • Nausea
  • Decreased white blood cell and platelet counts

In addition to the above common side effects observed in patients taking the other TKIs, patients taking ponatinib also sometimes experience high blood pressure.

Potential serious side effects:

  • Abnormal heart rhythm. Ponatinib can change your heart rhythm.
  • Rarely, patients taking ponatinib can develop liver irritation or hepatitis.

After FDA approval, ponatinib was briefly withdrawn from the market due to an increased risk of serious cardiovascular events (strokes, heart attacks, blood clots in your legs or lungs). FDA re-approved the use of ponatinib in more restricted settings and to ensure appropriate warnings were included with the medication information presented to patients and their physicians.

Tell your doctor if you have or have ever had a heart attack or stroke or abnormal blood clots in your lungs or legs. It is also important to discuss with your medical team any risk factors you have for cardiovascular disease such as high blood pressure; hyperlipidemia (high cholesterol) or cigarette smoking. 

Additional Information: https://medlineplus.gov/druginfo/meds/a613029.html

Richard T. Silver MD Myeloproliferative Neoplasms Center 525 East 70th St., Starr Pavillion, 3rd Floor New York, NY 10021 SilverMPNCenter@med.cornell.edu