10 Common Treatments for ITP: How Do They Work?

Medically reviewed by Richard LoCicero, M.D.
Written by Emily Wagner, M.S.
Posted on February 14, 2023

Immune thrombocytopenia (ITP) is an autoimmune disorder that results in low platelet counts, reducing the number of cells in your blood that help with blood clotting. While there is no cure, there are treatments that help improve platelet counts and manage ITP symptoms. Some ITP treatments encourage platelet production. Others focus on blocking platelet destruction caused by the immune system.

Most cases of mild ITP can be managed without medication, although your doctor will likely schedule follow-up monitoring appointments and regular blood tests. For children with ITP, the condition usually goes away on its own.

People with chronic (long-lasting) ITP may need more complex treatment plans. First-line or initial treatments are usually started after a diagnosis of ITP or a severe bleeding event. If these treatments don’t improve platelet counts, second- and third-line therapies may be considered.

Read on to learn more about 10 common treatments for ITP.

1. Corticosteroids

The immune system makes antibodies that recognize and destroy harmful bacteria and viruses. In some cases, it makes autoantibodies that recognize your body’s own cells and tissues as invaders. For people with ITP, these autoantibodies target platelets.

The most common first-line treatment used for ITP is corticosteroids. These medications work to decrease inflammation and help prevent the immune system from targeting your own platelets. Corticosteroids help increase your platelet count within five to 10 days of starting treatment.

Your hematologist may prescribe corticosteroids like prednisone or dexamethasone. They are taken daily as pills until your platelet counts are back to normal. Your doctor will then slowly lower your dose until you no longer need the medication.

While these medications are extremely effective at increasing platelet levels, taking them for a long time can cause side effects. These include:

  • Increased risk of infections
  • High blood sugar levels, which may increase your risk of diabetes
  • Eye problems that lead to blurry vision and, in some cases, vision loss
  • High blood pressure
  • Bone problems, such as weak or brittle bones (osteoporosis)
  • Buildup of fluid in your body, which can lead to swelling of your feet, legs, hands, or arms

For this reason, corticosteroids are usually prescribed as a short-term treatment.

2. Intravenous Immunoglobulin

Another common first-line treatment for ITP is intravenous immunoglobulin (IVIG). This is a liquid medication that contains concentrated antibodies collected from donors. Doctors believe IVIG helps block your immune system from targeting your platelets.

IVIG is recommended for people who don’t see improvements with corticosteroids. It’s also used in emergencies to treat uncontrollable bleeding. IVIG is given as an infusion into a vein intravenously over the course of a few hours in one or two large doses.

IVIG should begin raising your platelet count within a few days. However, these effects may only last for a few weeks. You may need another infusion to boost your platelet levels again.

You may experience some side effects (known as an infusion reaction) from IVIG, such as:

  • Headache
  • Fatigue
  • Chills or fever
  • Nausea or vomiting
  • Flushing (redness and warming of your skin) in your face, arms, or legs
  • Achy joints and muscles

Your doctor may give you a pain reliever before your infusion to help prevent these side effects. Some people react to IVIG if it’s infused very quickly in one large dose, but this can be eased by taking smaller doses over a long period of time.

3. Intravenous Rho(D) Immunoglobulin

A treatment option similar to IVIG is intravenous Rho(D) immunoglobulin, or RhIG. It only works in people with Rh-positive blood, which your health care provider can determine with a blood test.

Doctors and researchers aren’t sure exactly how RhIG treatment works, but they believe it stops your spleen from destroying platelets. RhIG is given as an infusion like IVIG, and it has similar side effects. Your doctor may monitor you for several hours after the infusion to make sure you don’t have a serious reaction.

4. Immunosuppressants

If corticosteroids and IVIG or RhIG don’t help increase your platelet levels, your doctor may prescribe immunosuppressants. These medications help slow activity of the immune system and prevent it from destroying your platelets. Your doctor may prescribe an immunosuppressant and corticosteroid together to make them more effective.

Examples of immunosuppressants in the treatment of ITP include:

  • Cyclosporine (Sandimmune)
  • Azathioprine (Imuran)
  • Mycophenolate mofetil (CellCept)

Because these medications dampen your immune system, they make it harder for your body to fight infections. Other side effects you may experience include:

  • Headaches
  • Trouble sleeping
  • Pale skin
  • Fever or chills
  • Shortness of breath
  • Rapid heartbeat

Immunosuppressants tend to take longer than corticosteroids or IVIG to raise platelet levels. As a result, these medications are prescribed as a second- or third-line option after trying other treatments.

5. Rituximab

Rituximab (Rituxan) is a medication used to prevent the immune system from attacking platelets in people with chronic ITP. Sometimes, it’s prescribed as a second-line treatment alongside dexamethasone (a corticosteroid) to treat people newly diagnosed with ITP. Nearly 60 percent of people who take rituximab see their platelet count improve — for some, these effects can last at least five years.

Rituximab is given as an IV infusion once per week for four weeks. You may experience symptoms of an infusion reaction. Since the medication blocks all antibody production, your immune system may have a harder time fighting infections.

6. Thrombopoietin Receptor Agonists

Thrombopoietin (TPO) receptor agonists are a newer class of medication used as second-line therapy for ITP. They mimic the function of the TPO hormone and help activate the production of platelets. Examples of TPO receptor agonists include:

  • Romiplostim (Nplate)
  • Eltrombopag (Promacta)
  • Avatrombopag (Doptelet)
  • Lusutrombopag (Mulpleta)

TPO receptor agonists are taken as tablets once daily. Most side effects are mild. These include:

  • Abdominal pain
  • Nausea
  • Headache
  • Dizziness
  • Fatigue
  • Indigestion
  • Muscle aches and pains
  • Achy joints
  • Trouble sleeping

In rare cases, TPO receptor agonists can cause blood clotting or issues with liver function. While you’re being treated for ITP, your doctor will periodically order blood tests to be sure your liver levels and platelet levels are within a healthy range.

7. Fostamatinib

Fostamatinib (Tavalisse) is a tyrosine kinase inhibitor medication. It can be a second- or third-line therapy for ITP. Fostamatinib blocks the activity of the spleen tyrosine kinase enzyme.

Fostamatinib is administered by mouth. Side effects include diarrhea, high blood pressure, a decrease in white blood cells, and elevated liver function. Your doctor will periodically monitor your blood and liver levels to make sure they are healthy.

8. Dapsone

Dapsone (Aczone) is a medication typically used to treat skin infections. It also has anti-inflammatory effects and can be used as a second- or third-line treatment for ITP. Researchers believe this medication prevents your spleen from trapping and destroying platelets.

Dapsone is taken as a tablet by mouth. The most common side effects are nausea and vomiting.

9. Platelet Transfusion

A platelet transfusion may be done in an emergency to stop excessive bleeding, although this is not usually a long-term solution to ITP symptoms. These platelets are collected from donors and checked for any bacteria or viruses. During the procedure, you’ll have a thin, flexible tube known as a cannula inserted into a vein in your arm. The platelets are transfused for 15 to 30 minutes.

Some people have a reaction to a platelet transfusion. If you notice a fever or you develop an itchy rash during your transfusion, be sure to let the nurse or doctor know.

10. Splenectomy

A splenectomy is a surgical procedure to remove the spleen — an organ that aids the immune system and helps clean the blood. In ITP, the spleen filters out platelets that have antibodies attached to them. Therefore, when the spleen is removed, it helps those platelets to stay in circulation and thus improves platelet counts.

A splenectomy can be laparoscopic, when the spleen is removed through several small holes in the abdomen, or open, which requires a large incision in the abdomen and longer recovery time. Both methods help improve platelet levels, and help people with ITP achieve remission in 70 percent to 80 percent of cases.

Splenectomy is reserved for cases of ITP that are severe, chronic (lasting longer than one year), and symptomatic. It’s not typically recommended for children with ITP since they tend to recover without treatment. Having your spleen removed will impair your immune system and make it more difficult for your body to fight off certain infections.

If you have been diagnosed with ITP, ask your doctor which treatment — if any — is appropriate for you. The answer will depend on the severity of your ITP, which treatments you have already tried, your overall health, and any other health conditions you have. You and your doctor can work together to find the best treatment plan for you.

Talk With Others Who Understand

On myITPcenter, the site for people with immune thrombocytopenia and their loved ones, people come together to gain a new understanding of ITP and share their stories with others who understand life with ITP.

What treatments have you tried for ITP? Which ones have worked best for you? Share your experience in the comments below.

References
  1. Immune Thrombocytopenia (ITP) — National Heart, Lung, and Blood Institute
  2. Immune Thrombocytopenia (ITP) — Mayo Clinic
  3. Platelets — Cleveland Clinic
  4. Immune Thrombocytopenia — NORD
  5. Immune Thrombocytopenia: Standard Therapies — NORD
  6. Pediatric Idiopathic Thrombocytopenia Purpura (ITP) — Children’s National
  7. Immune Thrombocytopenia: Antiplatelet Autoantibodies Inhibit Proplatelet Formation by Megakaryocytes and Impair Platelet Production In Vitro — Haematologica
  8. Corticosteroids — Cleveland Clinic
  9. Immune Thrombocytopenia Treatments — UCSF Health
  10. Prednisone and Other Corticosteroids — Mayo Clinic
  11. Patient Education: Intravenous Immune Globulin (IVIG) (Beyond the Basics) — UpToDate
  12. Efficacy and Mechanism of Intravenous Immunoglobulin Treatment for Immune Thrombocytopenia in Adults — Annals of Blood
  13. Rho(D) Immune Globulin — StatPearls
  14. Rho(D) Immune Globulin (Injection Route, Intramuscular Route, Intravenous Route) — Mayo Clinic
  15. Splenectomy — Platelet Disorder Support Association
  16. Immune Thrombocytopenia (ITP) Therapies — Rare Disease Advisor
  17. B-Cell Lymphocyte Depletion Therapy — Platelet Disorder Support Association
  18. T Cells, B Cells and the Immune System — The University of Texas MD Anderson Cancer Center
  19. Thrombopoietin Receptor Agonists in Primary ITP — Seminars in Hematology
  20. Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists — Drugs
  21. Immunosuppressants — Platelet Disorder Support Association
  22. How Does Dapsone Work in Immune Thrombocytopenia? Implications for Dosing — Blood
  23. Dapsone — MedlinePlus
  24. Platelet Transfusions — Macmillan Cancer Support
    Richard LoCicero, M.D. has a private practice specializing in hematology and medical oncology at the Longstreet Clinic Cancer Center, in Gainesville, Georgia. Review provided by VeriMed Healthcare Network. Learn more about him here.
    Emily Wagner, M.S. holds a Master of Science in biomedical sciences with a focus in pharmacology. She is passionate about immunology, cancer biology, and molecular biology. Learn more about her here.

    John4

    Please include more peer-reviewed postdoctoral theses and research synopses. Thank you.

    November 25, 2023
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