Promacta (Eltrombopag) for Immune Thrombocytopenia

Promacta (eltrombopag) is a prescription medication approved by the U.S. Food and Drug Administration (FDA) to treat low platelet counts in children ages 1 year and older with persistent or chronic immune thrombocytopenia (ITP).

How Promacta Works and How It’s Taken

Promacta is a type of medicine called a thrombopoietin receptor agonist. It acts on the thrombopoietin receptor. This helps the body make more megakaryocytes, which are cells that make platelets. In ITP, this helps reduce low platelet counts that can raise the risk of bleeding.

Doctors prescribe Promacta for adults and children ages 1 year and older. It is used when other ITP treatments have not worked well enough. These treatments may include corticosteroids, immunoglobulins, or surgery to remove the spleen.

Promacta comes as a tablet or an oral suspension. Both are taken by mouth once daily.

Take Promacta tablets whole. Do not split, chew, or crush the tablets. Do not mix the tablets with food or liquids.

For Promacta oral suspension, use a new single-use oral dosing syringe to prepare each dose. Do not reuse the oral dosing syringe.

Your healthcare provider will prescribe the dose of Promacta that is right for you.

Take Promacta without a meal or with a meal low in calcium (50 milligrams of calcium or less). Take it at least two hours before or four hours after eating calcium-rich foods, such as dairy products, calcium-fortified juices, and certain fruits and vegetables.

Typical Dosing for ITP

For most adults and children ages 6 years and older with ITP, the recommended starting dose is 50 milligrams once daily. For children ages 1 to 5 years, the recommended starting dose is 25 milligrams once daily.

The dose may be lower for some adults and children ages 6 years and older who are of East Asian or Southeast Asian ancestry. The dose may also be lower for adults and children ages 6 years and older with liver problems. Doctors adjust the dose based on platelet counts.

The goal is to use the lowest dose needed to keep platelet counts at or above 50 x 109/L, as needed to lower the risk of bleeding. Promacta should not be used to make platelet counts normal.

Doctors may stop Promacta if platelet counts get too high. They may also stop it if platelet counts do not increase enough after four weeks at the maximum daily dose of 75 milligrams.

This information is based on the prescribing information, but your healthcare provider may tailor your treatment plan. Always follow their guidance.

Promacta Side Effects

In clinical studies of Promacta for persistent or chronic immune thrombocytopenia, side effects differed between adults and children.

In adults with ITP, between 3 percent and 10 percent of people experienced at least one common side effect, including:

Nausea
Diarrhea
Upper respiratory tract infection, such as cold-like symptoms
Vomiting
Urinary tract infection
Increased liver enzymes on blood tests
Muscle pain
Pain in the mouth or throat
Back pain
Flu
Tingling or numbness
Rash

In children ages 1 year and older with ITP, between 4 percent and 17 percent experienced at least one common side effect, including:

Upper respiratory tract infection, such as cold-like symptoms
Nasopharyngitis, such as a stuffy nose or sore throat
Cough
Diarrhea
Fever
Belly pain
Pain in the mouth or throat
Toothache
Increased liver enzymes on blood tests, such as ALT or AST
Rash
Runny nose

Serious Side Effects and Warnings

Promacta can cause serious side effects that may require immediate medical attention. These include:

Hepatotoxicity — Liver problems can be severe and life-threatening.
Thrombotic or thromboembolic complications — These include clots in the legs or lungs, heart attack, stroke, or clots in the liver vein.
Cataracts — New or worsening clouding of the eye’s lens can affect vision.
Increased risk of death and progression of myelodysplastic syndromes to acute myeloid leukemia — This risk is seen in people with myelodysplastic syndromes.

Get medical help right away if you think you are having a serious reaction.

How To Save on Promacta

Novartis, the manufacturer of Promacta, offers the Universal Co-pay Program. Eligible people with private insurance may pay as little as $0 per month for Promacta.

Novartis Patient Support can also help people learn about financial assistance options for Promacta.

To learn more, visit the Promacta website or call 877-577-7756.

What To Know Before Taking Promacta

Before starting Promacta, your doctor will order blood tests to check your liver function and a baseline eye exam to check for cataracts.

Before you take Promacta, tell your doctor about all of your medical conditions, including if you:

Have liver problems
Have a precancerous condition called MDS or a blood cancer
Currently have or have ever had a blood clot
Have a history of cataracts
Have had surgery to remove your spleen, called a splenectomy
Have bleeding problems
Are of East Asian or Southeast Asian ancestry, because you may need a lower dose of Promacta

Tell your doctor if you have any allergies to eltrombopag or any ingredients in Promacta. Do not take Promacta if you have had a serious allergic reaction to eltrombopag or any of its ingredients.

Tell your doctor about all medicines you take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements.

Especially tell your doctor if you take certain statins (medicines for high cholesterol) or a blood thinner.

Some medicines and supplements may keep Promacta from working correctly. Take Promacta at least two hours before or four hours after taking antacids, multivitamins, or products that contain iron, calcium, aluminum, magnesium, selenium, or zinc.

Promacta is also FDA approved to treat thrombocytopenia in people with chronic hepatitis C (to allow interferon-based therapy) and severe aplastic anemia.

If you miss a dose, wait and take your next dose at your regular scheduled time. Do not take more than one dose of Promacta in one day.

If you are pregnant, planning to become pregnant, or breastfeeding while taking Promacta, talk with your doctor about the risks and benefits. If you can become pregnant, use effective birth control during treatment and for at least seven days after stopping Promacta. Do not breastfeed during treatment with Promacta.

Community FAQs

These answers are fact-checked by our editorial staff.

How effective is Promacta for ITP?

In one study of adults with persistent or chronic ITP, 59 percent of people taking Promacta reached a platelet count of at least 50 x 109/L. This compared with 16 percent of people taking placebo (an inactive treatment).

In another study, adults taking Promacta were more likely to reach a platelet count of at least 50 x 109/L during treatment than people taking placebo. In that study, 18 percent of people taking Promacta needed rescue therapy, compared with 40 percent of people taking placebo.

In a long-term extension study, median platelet counts stayed between 64 and 119 x 109/L over seven years.

How long does Promacta take to work for ITP?

In clinical studies, platelet counts increased with daily Promacta treatment. Platelet counts reached their highest level about two weeks after starting treatment.

After the last dose, platelet counts returned to baseline in about two weeks.

What tests or monitoring are needed with Promacta for ITP?

For ITP, your doctor will check complete blood counts, also called CBCs. These tests include platelet counts. They should be checked weekly until your platelet count is stable. After that, they should be checked monthly.

Your doctor will also check liver tests. These include ALT, AST, and bilirubin. These tests should be done before starting Promacta, every two weeks while the dose is being adjusted, and then monthly after a stable dose is reached.

A baseline eye exam is also recommended before starting Promacta. Your doctor should also monitor you for signs and symptoms of cataracts during treatment.