Patient Handout: Ponatinib (Iclusig®)

What is Ponatinib?

Ponatinib (brand name: Iclusig®) is a medication used to treat certain types of leukemia (blood cancer) and other conditions where there are abnormal white blood cells. It works by targeting and blocking proteins that drive the growth of cancer cells, specifically the BCR-ABL fusion protein, which is present in chronic myelogenous leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

Ponatinib is typically used when other treatments have not worked or are no longer effective. It can also be prescribed in cases where certain mutations (like the T315I mutation) make other treatments ineffective.

How Does Ponatinib Work?

Ponatinib is a tyrosine kinase inhibitor (TKI). It blocks signals that tell cancer cells to grow and divide. By interfering with these signals, Ponatinib helps to slow or stop the growth of abnormal cells in the body.

How Should I Take Ponatinib?

• Dosage: Your doctor will determine the exact dose based on your condition and response to treatment.
• Administration: Ponatinib is taken by mouth in the form of a tablet. It is usually taken once a day, with or without food.
• Swallow the tablet whole: Do not break, crush, or chew the tablet.
• Missed dose: If you miss a dose, take it as soon as you remember. However, if it is almost time for your next dose, skip the missed dose. Do not take two doses at once.

Possible Side Effects

Ponatinib can cause side effects, and some of them can be serious. It is important to report any new or unusual symptoms to your doctor. Common side effects include:

• Common side effects:
  • Fatigue
  • Headache
  • Nausea
  • Diarrhea
  • Skin rash
• Serious side effects (seek medical attention immediately):
  • Blood clots: Ponatinib increases the risk of blood clots, which can lead to heart attack, stroke, or other serious conditions. Symptoms of a blood clot include chest pain, shortness of breath, and swelling in your legs.
  • Heart problems: Ponatinib can cause heart issues, such as heart failure or changes in heart rhythm. Notify your doctor if you experience chest pain, shortness of breath, or swelling in the ankles or legs.
  • Liver problems: Your liver function may be affected. Symptoms include yellowing of the skin or eyes (jaundice), dark urine, and abdominal pain.
  • High blood pressure: Ponatinib can cause high blood pressure. Your doctor will monitor this regularly.
  • Pancreatitis: This is inflammation of the pancreas and can cause severe stomach pain, nausea, and vomiting.

Precautions

Before starting treatment with Ponatinib, inform your doctor if you have any of the following:

• Heart disease or problems (e.g., history of heart attack, heart failure, or arrhythmia)
• Blood clotting disorders or history of stroke
• Liver problems (e.g., hepatitis or cirrhosis)
• High blood pressure
• Pancreatitis
• Pregnancy or breastfeeding: Ponatinib may harm an unborn baby and should not be used during pregnancy unless the potential benefits outweigh the risks. You should also avoid breastfeeding during treatment.

Regular Monitoring and Tests

While you are taking Ponatinib, your doctor will perform regular tests to monitor for side effects, including:

• Blood tests to check for liver function, blood counts, and other important indicators
• Monitoring of blood pressure
• Regular heart function assessments (e.g., echocardiogram or ECG)

Drug Interactions

Ponatinib can interact with other medications, which may increase the risk of side effects or affect how well Ponatinib works. Be sure to inform your doctor about all medications you are taking, including:

• Prescription drugs
• Over-the-counter medications
• Herbal supplements
• Vitamins

What to Do in Case of an Emergency

If you experience any of the following symptoms, get emergency medical help right away:

• Chest pain, shortness of breath, or difficulty breathing
• Sudden weakness or numbness on one side of the body
• Severe headache or vision changes
• Unusual bruising or bleeding
• Severe stomach pain, nausea, or vomiting

Pregnancy and Breastfeeding

• Pregnancy: Ponatinib may harm an unborn baby. You should not become pregnant while taking Ponatinib. If you are planning to become pregnant or suspect that you are pregnant, talk to your doctor immediately.
• Breastfeeding: It is not known whether Ponatinib passes into breast milk. You should not breastfeed while taking Ponatinib.

Lifestyle Tips

• Avoid certain activities: Because Ponatinib may increase the risk of blood clots and other heart-related issues, avoid activities that could result in injury, such as contact sports, without consulting your doctor.
• Stay hydrated: Drink plenty of fluids to stay hydrated, especially if you experience diarrhea or other gastrointestinal side effects.
• Follow up appointments: Attend all scheduled appointments with your doctor for regular monitoring of your condition and side effects.

Conclusion

Ponatinib is a powerful treatment option for certain types of leukemia, but it comes with potential side effects and risks. It is essential to follow your doctor’s instructions carefully and report any new or unusual symptoms promptly. If you have any questions or concerns about your treatment plan or Ponatinib, do not hesitate to contact your healthcare provider.

Disclaimer: This handout is for educational purposes only and is not intended as medical advice. Always consult your healthcare provider for specific medical guidance regarding your treatment.