Living with a long-term condition like chronic immune thrombocytopenia (ITP) can feel like a constant uphill battle. Fortunately, doctors and researchers are always looking for new and better ways to treat ITP, helping you to manage your symptoms and improve your quality of life.

In this article, we’ll discuss a few of the mainstay treatments doctors and hematologists use to initially treat and control chronic ITP, along with some new advances. These new therapies use unique ways to boost your platelet count and prevent severe bleeding events.

First-Line Treatments for Chronic ITP

Chronic ITP is a long-term autoimmune disease that’s caused by your immune system attacking your platelets. These cells are responsible for helping your blood clot. Specifically, your body creates IgG autoantibodies or immune system proteins that attach themselves to your platelets, targeting them for destruction.

When your platelet count is too low, you’re at a higher risk of bleeding. For people with chronic ITP, it can be hard to stop this bleeding, which can become dangerous. Initial or first-line treatment of ITP focuses on controlling severe bleeding as quickly as possible.

Corticosteroids are one of the most common first-line treatments. They work by lowering inflammation levels in your body and preventing your immune system from destroying your platelets. Examples include dexamethasone, prednisone, and deflazacort. You may also receive an infusion with intravenous immunoglobulin (IVIG) — a concentrated dose of healthy donor antibodies to help control inflammation.

Second-Line Treatments for Chronic ITP

Chronic ITP is diagnosed when you’ve had ITP symptoms for six months or longer. This means that you’ll need long-term treatment to help prevent other bleeding events. These second-line treatments can offer longer-lasting results and help stabilize your platelet counts.

Rituximab

B cells are specialized immune cells that make antibodies. Normally, antibodies help your body fight off foreign invaders to prevent infection and keep you healthy. In people with chronic ITP, B cells make autoantibodies that target your body’s platelets. Researchers believe that blocking B cells from doing this may help prevent platelet destruction.

Rituximab (Rituxan) is a monoclonal antibody drug, or a laboratory-engineered protein, designed to target B cells. In studies, around 60 percent of people with chronic ITP have experienced a short-term increase in platelet levels in response to taking rituximab. For one-quarter of those people, those effects lasted five years or more.

Thrombopoietin Receptor Agonists

Your platelets are produced by specialized cells known as megakaryocytes, which are found in your bone marrow (the spongy tissue inside your bones). In chronic ITP, your bone marrow can’t make enough platelets to replace those that are destroyed. As a result, your platelet count drops, and your risk of bleeding increases.

Thrombopoietin receptor agonists (TPO-RAs) are a newer type of medication that trigger megakaryocytes to make more platelets. They act similarly to the TPO hormone found naturally in our bodies. The U.S. Food and Drug Administration (FDA) has approved four TPO-RAs:

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

Studies show that TPO-RAs tend to have better results compared to other second-line therapies. However, you may have to take them longer than other treatments, such as rituximab.

Splenectomy

Although your spleen is just a small organ, it can play a big role in chronic ITP. Located in your abdomen next to your stomach and kidneys, your spleen acts like a filter for your immune system and bloodstream. In people with chronic ITP, their spleens destroy platelets that are covered in autoantibodies. This process contributes to a low platelet count and ITP symptoms.

Another second-line treatment option for chronic ITP is a splenectomy, or surgery to remove your spleen. This procedure can help prevent your platelets from being destroyed, which helps raise your overall count. Your hematologist will likely recommend this procedure if you have chronic ITP with severe symptoms or if you haven’t responded to other treatments.

4 New Treatments for Chronic ITP

As doctors and researchers have learned more about chronic ITP, they’ve found several new ways to treat it. Some therapies target B cells in a different way than current treatments, while others prevent immune cells from destroying platelets.

In order for these new treatment options to become widely available to people living with chronic ITP, they first have to go through clinical trials. These large studies help doctors and researchers learn about a new drug’s long-term safety, side effects, and effectiveness.

Here’s a list of new treatments for chronic ITP that have been recently approved or are still in clinical trials. If you’re interested in learning more about one of these therapies, talk to your hematology care provider.

1. Fostamatinib

Macrophages are like your body’s cleaning crew — they “eat” and break down waste and old cells to help keep your body healthy. They can be found throughout your body, especially in the spleen. There, macrophages are responsible for breaking down platelets in people with chronic ITP.

While other available treatments work to boost platelet production, fostamatinib (Tavalisse) works to prevent their destruction. The FDA approved Tavalisse in 2018 as a second- or third-line treatment for chronic ITP. Specifically, it stops macrophages from “eating” platelets, which helps increase your platelet count.

2. Rilzabrutinib

Bruton’s tyrosine kinase (BTK) is a protein used by B cells to help them grow and divide. When you’re sick, your B cells need to be able to grow quickly, make antibodies, and fight infection. However, in chronic ITP, your B cells often do more harm than good.

Rilzabrutinib is a new BTK inhibitor being studied for treating chronic ITP. A small research study found that 40 percent of participants who tried the medication experienced a rapid, clinically significant platelet response. Because of these positive results, scientists are now examining rilzabrutinib in a larger clinical study.

3. Efgartigimod

Your body has many systems in place to make sure everything is balanced. For example, your immune system regulates your antibody levels using a protein known as the neonatal Fc receptor (FcRn). Doctors and researchers have discovered that blocking FcRn causes your body to clear out extra autoantibodies. This means your platelets are less likely to be destroyed, which can increase your count.

Scientists are currently studying efgartigimod (Vyvgart) as a treatment for chronic ITP. A 2022 study found that efgartigimod is a safe medication that significantly improves platelet counts.

4. New Monoclonal Antibodies

Doctors and researchers have also developed new monoclonal antibodies that target B cells in different ways. Two treatments that show promise for chronic ITP are daratumumab (Darzalex) and mezagitamab. They work like rituximab to block B cells and stop them from making autoantibodies that destroy platelets.

The FDA has already approved Darzalex for treating multiple myeloma, a type of cancer that affects B cells. However, doctors and researchers need to make sure it’s also safe and effective in people with chronic ITP. A research study is currently underway in Europe and is expected to be completed in late 2024.

Mezagitamab is a newer therapeutic approach scientists are studying to treat several conditions caused by abnormal antibodies. A study is underway comparing the drug to a placebo (inactive treatment) in people with chronic ITP. Researchers estimate it will be complete in August of 2024.

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.

Have you tried a new treatment for your chronic ITP? How has it improved your symptoms or your quality of life? Share your experience in the comments below.