How To Treat ITP: 10 Treatment Options and How They Work

Immune thrombocytopenia (ITP) is an autoimmune disorder that results in low platelet counts, reducing the number of cells in blood that help with blood clotting. Although there’s no cure, certain treatments can 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 might improve without therapy.

People with chronic (long-lasting) ITP may need medications to control their condition. First-line drugs, 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 treatments for ITP and how they work.

First-Line Treatments

First-line treatments are therapies that healthcare providers try first to address a condition. With ITP, the goal of first-line treatment is to calm the immune system and preserve the production of platelets. Treatments that do so are corticosteroids, intravenous immunoglobulin (IVIG), and intravenous Rho(D) immunoglobulin.

1. Corticosteroids

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

The most common first-line treatment for ITP is corticosteroids. These medications work to decrease inflammation and help prevent the immune system from targeting the body’s platelets. Oral corticosteroids (taken by mouth) help increase platelet count within two to four weeks of starting treatment. IV methylprednisolone can raise platelet counts in a few days, but doctors usually use it only in emergencies when there is heavy bleeding.

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

Although these medications for ITP are very 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 diabetes risk
• Eye problems that lead to blurry vision and, in some cases, vision loss
• High blood pressure
• Osteoporosis (weak or brittle bones)
• Buildup of body fluid, which can lead to swollen 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. This liquid medication contains concentrated antibodies collected from donors. Scientists believe IVIG helps block the immune system from targeting platelets.

IVIG may be added to treatment when corticosteroids alone don’t help enough. It’s also used in emergencies to treat uncontrollable bleeding. IVIG is given as an infusion into a vein 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 last only 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 (discoloration and warming of skin) in your face, arms, or legs
• Achy joints and muscles

3. Intravenous Rho(D) Immunoglobulin

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

Doctors and researchers aren’t sure exactly how RhIG treatment works, but they believe it stops the spleen from destroying platelets. Like IVIG, RhIG is given as an infusion, 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.

Second-Line and Additional Treatments

If first-line treatments aren’t effective or stop working, your doctor may recommend other therapies to help manage ITP. These second-line and additional treatments are often considered when ITP becomes chronic or harder to control. The options below may be used alone or in combination, depending on your individual needs and health history.

4. Immunosuppressants

If corticosteroids and IVIG 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:

• Azathioprine
• Cyclosporine
• Dapsone
• Mycophenolate mofetil

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

• Headache
• 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, so they’re prescribed after trying other treatments.

5. Rituximab

Rituximab 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. The U.S. Food and Drug Administration (FDA) has approved rituximab for many conditions, but it’s still used off-label for ITP. Platelet counts improve for nearly 60 percent of people who take rituximab — for some, these effects can last at least five years.

Rituximab is given as an IV infusion once a 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 drug class used as second-line therapy for ITP. These medications mimic the function of the TPO hormone and help activate the production of platelets. Examples of TPO receptor agonists include:

• Avatrombopag (Doptelet, Doptelet Sprinkle)
• Eltrombopag (Promacta)
• Romiplostim (Nplate)

Most TPO receptor agonists are taken as tablets once daily. Most side effects are mild, such as:

• Nausea
• Fatigue
• Headache
• Achy joints
• Common cold

In rare cases, TPO receptor agonists can cause blood clotting or liver function issues. 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 that can be a second- or third-line therapy for ITP. Fostamatinib blocks an enzyme called spleen tyrosine kinase.

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

8. 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. Removing the spleen helps keep those platelets in circulation, improving platelet counts.

A splenectomy can be laparoscopic (removing the spleen through several small holes in the abdomen) or open (requiring a large incision and longer recovery). Both methods improve platelet levels, helping 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 impairs your immune system and makes it more difficult for your body to fight off certain infections.

9. Platelet Transfusion

A platelet transfusion may be done in an emergency to stop excessive bleeding, but it’s not usually a long-term solution to ITP symptoms. The platelets are collected from donors and checked for any bacteria or viruses. During the procedure, you’ll have a cannula (a thin, flexible tube) inserted into a vein in your arm. The platelets are transfused for 30 to 60 minutes.

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

New Therapies

Researchers are continually working to develop new treatments for people living with ITP. Recent advances have led to options that may help those who haven’t responded to other approaches.

10. Rilzabrutinib

Rilzabrutinib (Wayrilz) is a newly approved oral medication used to treat adults with persistent or chronic ITP who haven’t responded to other treatments. It belongs to a class of drugs known as Bruton’s tyrosine kinase (BTK) inhibitors. These medications work by targeting immune cells that mistakenly destroy platelets, helping to reduce inflammation and prevent further platelet loss.

Rilzabrutinib is taken by mouth as a tablet. In clinical trials, it has shown promising results in maintaining healthy platelet counts over time. Common side effects may include:

• Diarrhea
• Nausea
• Stomach pain
• Headache
• COVID-19

This BTK inhibitor offers a new treatment option for people with ITP who haven’t seen results with corticosteroids, IVIG, or other therapies.

What Is the Best Treatment for ITP?

The best treatment for ITP is one that your doctor or team of healthcare professionals recommends after considering your input. When determining the right treatment for you, your healthcare provider will consider your specific case of ITP, including your age, symptoms, condition severity, and other health conditions you may have. Work closely with your doctor to provide feedback about your condition and treatment plan so they can make adjustments as needed.

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