Frequently Asked Questions About PROMACTA

Many health care professionals follow the guidelines of the American Society of Hematology (ASH), the society for doctors who treat blood diseases. Here is an overview of the recommendations you can use when discussing treatment with your child’s doctor.

Newly diagnosed ITP
(sometimes called "acute ITP")

Corticosteroids (steroids)
Most patients with ITP start with a round of corticosteroids ("steroids") to stop the body from destroying platelets and quickly raise platelet counts. Unfortunately, steroids can also have side effects, such as insomnia, weight gain, and mood changes. That’s one of the reasons why ASH recommends the use of steroids for no more than 6 weeks for adults and no more than 7 days for children.

Intravenous immunoglobulin (IVIg)
IVIg is also an option to raise platelet counts quickly, primarily in adults. IVIg is an infusion given with a needle and requires sitting still for some time while the medicine goes into the veins.

Persistent or chronic ITP
(lasting ≥3 months despite treatment)

TPO-RAs (thrombopoietin receptor agonists)
Instead of trying to stop the body from destroying platelets, TPO-RAs are platelet boosters that encourage the body to make more. ASH recommends the use of TPO-RAs before other treatments for persistent or chronic ITP (see Monoclonal antibodies and Splenectomy). Currently, there are 2 TPO-RAs approved for use in pediatric patients:

• Nplate^® (romiplostim) is an injection that requires a weekly doctor visit to receive treatment
• PROMACTA^® (eltrombopag) is a once-daily oral treatment that can be taken wherever and whenever it works for you and your child. PROMACTA comes in both tablets and oral suspension for people who have difficulty swallowing a pill. When you first start taking PROMACTA, your doctor will monitor your platelet counts once a week to help find the appropriate dose. Complete blood counts with differentials, including platelet counts, will be obtained monthly thereafter.

Monoclonal antibodies
Rituxan^® (rituximab) is an IV infusion that suppresses a specific target in the immune system. While part of the ASH guidelines, it is not a US Food and Drug Administration (FDA)–approved treatment for ITP.

Splenectomy
Surgery to remove the spleen can help some people with persistent or chronic ITP, although it is associated with an increased risk of infection. There is no test to show whether or not a splenectomy will work, and there is always a risk with any surgical procedure.

Rescue therapy

Sometimes, treatment doesn’t work or it works for a while and then platelets drop again. If platelet levels get too low, your child may receive a different or additional treatment until they feel better again. Your child’s doctor may try to get their platelet count back up with:

• IVIg
• Platelet transfusions

Nplate is a registered trademark of Amgen Inc.

Rituxan is a registered trademark of Genentech USA, Inc.

The most common side effects of PROMACTA in children 1 year and older when used to treat persistent or chronic ITP are:

• upper respiratory tract infection (symptoms may include runny nose, stuffy nose, and sneezing)
• pain or swelling (inflammation) in your nose or throat (nasopharyngitis)

Less common but more serious side effects that may occur with PROMACTA include:

• Liver problems
• Worsening of a precancerous blood condition to a blood cancer called acute myelogenous leukemia (AML)
• High platelet counts and higher risk for blood clots
• New or worsened cataracts (a clouding of the lens in the eye)

Discuss potential side effects of PROMACTA with your health care provider.

PROMACTA is a prescription medication, so you will have to work with your doctor to see if it is the right treatment for your child’s persistent or chronic ITP. Questions about cost? Click here to learn about the PROMACTA Co-pay Card and other patient support.

Here are 3 important things to know:

1. PROMACTA can be taken without a meal or with a meal low in calcium (≤50 mg)
2. PROMACTA should be taken 2 hours before or 4 hours after taking medications like antacids, mineral supplements, or foods that are high in calcium
3. Weekly doctor visits aren't required for administration of PROMACTA

Click here to learn more tips about taking PROMACTA.

Tablets:

• Store PROMACTA tablets at room temperature between 68°F to 77°F (20°C to 25°C)
• Keep PROMACTA in the bottle given to you

For oral suspension:

• Store PROMACTA for oral suspension at room temperature between 68°F to 77°F (20°C to 25°C)
• After mixing, PROMACTA should be taken right away but may be stored for no more than 30 minutes between 68°F to 77°F (20°C to 25°C). Throw away (discard) the mixture if not used within 30 minutes

You and your doctor will need to work together to determine which activities are safe with your child’s platelet count. The good news is that most children with ITP can still do a number of different sports. Click here for a list and other tips about living with persistent or chronic ITP.

People may be curious about ITP and might ask questions to learn more about the disease. To help them understand, it could be useful to prepare answers to their questions. Click here to see example conversations about ITP.

First, check out our PROMACTA Patient Support Program by clicking here.

For information on financial support programs, click here.

Other useful resources include:

Platelet Disorder Support Association www.pdsa.org

• A helpful resource for ITP education, advocacy, research, and support. This site can help you find ITP experts or hematology centers of excellence near you

Foundation for Women & Girls with Blood Disorders (FWGBD) www.fwgbd.org/patient

• Helping to ensure that all women with blood disorders are correctly diagnosed and treated at every stage of life

ITP and Me www.itpandme.com

• Provides patients with ITP with medical support as well as emotional, lifestyle, and daily ITP guidance 

American Society of Hematology www.hematology.org/education/patients

• Furthering the understanding, diagnosis, treatment, and prevention of certain blood and bone disorders

HealthyChildren.org www.healthychildren.org

• A site run by the American Society of Pediatrics. The chronic condition section might be especially useful for parents of children with persistent or chronic ITP

• NHLBI promotes the prevention and treatment of heart diseases, lung diseases, and blood diseases such as ITP

• Providing a unified voice for those with rare diseases and their caregivers, seeking to help them so they won’t have to fight that battle alone

Do not flush unused or expired medications or pour down the drain. Follow federal, state, or local guidance to dispose of medications safely. Visit the FDA website for information about drug take-back programs and drop-off sites and how to properly dispose of medications in your home.

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