ponatinib hydrochloride at a glance
Medicine class
BCR-ABL tyrosine kinase inhibitor
Usual role
Specialist treatment for selected adults with CML or Ph+ ALL
Administration
Swallow tablets whole; they may be taken with or without food
Important risk
Arterial blockage and other cardiovascular complications require careful assessment and monitoring
What is ponatinib hydrochloride used for?
UK-licensed uses in adults are:
Chronic-phase, accelerated-phase or blast-phase chronic myeloid leukaemia that is resistant to dasatinib or nilotinib; where these treatments are not tolerated and subsequent imatinib is not clinically appropriate; or where the T315I mutation is present.
Philadelphia-chromosome-positive acute lymphoblastic leukaemia that is resistant to dasatinib; where dasatinib is not tolerated and subsequent imatinib is not clinically appropriate; or where the T315I mutation is present.
How does ponatinib hydrochloride work?
Ponatinib blocks BCR-ABL, an abnormal tyrosine kinase that drives production of leukaemia cells in CML and Ph+ ALL. Its structure allows it to inhibit native BCR-ABL and several resistant forms, including T315I.
ponatinib hydrochloride preparations and strengths
How to take ponatinib hydrochloride
Take ponatinib exactly as directed by the specialist team. Do not change or stop treatment without their advice.
Swallow each film-coated tablet whole with water.
Do not crush or dissolve the tablet.
It may be taken with or without food.
Avoid grapefruit products, including grapefruit juice.
Do not swallow the desiccant canister in the bottle.
ponatinib hydrochloride side effects
Common or expected effects
- Reduced red cells, platelets or neutrophils
- High blood pressure
- Headache or dizziness
- Abdominal pain, nausea, vomiting, diarrhoea or constipation
- Rash, dry or itchy skin
- Muscle, joint, bone or back pain
- Fatigue, weakness, fever or peripheral swelling
- Reduced appetite or raised blood fats
- Cough or breathlessness
- Raised liver enzymes or lipase
Get urgent medical advice
- Symptoms of arterial blockage, heart attack, stroke or reduced limb circulation
- Blood clots, including deep-vein thrombosis or pulmonary embolism
- Heart failure or a serious abnormal heart rhythm
- Severe or uncontrolled high blood pressure
- Pancreatitis, particularly severe abdominal and back pain
- Severe bleeding or marked bone-marrow suppression
- Serious infection or hepatitis B reactivation
- Liver failure or jaundice
- Sudden neurological or visual symptoms suggesting PRES
- Blistering or peeling skin with mucosal or systemic symptoms
ponatinib hydrochloride in pregnancy
Ponatinib should generally not be used during pregnancy unless the woman's clinical condition requires it. Limited human evidence and animal findings indicate potential fetal harm. People who could conceive or father a child should discuss effective contraception with the specialist team; hormonal contraception may require an additional or alternative method.
ponatinib hydrochloride while breastfeeding
Breastfeeding should stop during ponatinib treatment because transfer into human milk is unknown and harm to the infant cannot be excluded.
ponatinib hydrochloride interactions
The specialist team and pharmacist should review prescribed, non-prescribed and herbal products before and during treatment.
Strong CYP3A inhibitors, including clarithromycin, itraconazole, voriconazole and ritonavir
May increase ponatinib exposure and adverse effects, requiring specialist review.
Grapefruit products
May increase ponatinib exposure and should be avoided.
Strong CYP3A inducers, including rifampicin, carbamazepine, phenytoin and St John's wort
May reduce ponatinib exposure and effectiveness; alternatives should usually be sought.
Common questions about ponatinib hydrochloride
Answers are fully visible for fast scanning and source review.
Why is ponatinib used after other tyrosine kinase inhibitors?
Its licensed role includes selected leukaemia that is resistant to or not tolerant of specified earlier inhibitors, or that carries the T315I mutation.
Does ponatinib work against the T315I mutation?
Yes. Ponatinib inhibits T315I-mutant BCR-ABL, and the mutation is specifically included in its licensed indications.
Why are cardiovascular checks needed with ponatinib?
Ponatinib can cause serious arterial blockage, blood clots, hypertension and heart failure, including in some people without recognised cardiovascular risk factors.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.