5 New Treatments for Chronic ITP

Living with chronic immune thrombocytopenia (ITP) means dealing with more than just low platelets (cell fragments that help blood clot). It often brings bruises that appear out of nowhere, fatigue, and the constant worry of serious complications.

The good news is that, in recent years, researchers have made big strides in finding new treatments for ITP.

These options go beyond common treatments, like corticosteroids or intravenous immunoglobulin (IVIG). They’re designed to raise platelet counts in new ways, improve safety, and offer longer-lasting results. Some are already approved by the U.S. Food and Drug Administration (FDA). Others are being tested in ongoing clinical trials.

In this article, we’ll discuss the latest advances in the treatment of chronic ITP. These new therapies boost low platelet counts in unique ways and prevent severe bleeding events.

Here, we’ll cover five of the most recent approaches.

One of the biggest breakthroughs in the treatment of chronic ITP has been the use of thrombopoietin receptor agonists (TPO-RAs). These medicines work with your body’s own system for making platelets.

In healthy people, the hormone thrombopoietin (TPO) tells megakaryocytes (special bone marrow cells) to make platelets. In chronic ITP, platelet destruction happens faster than new ones can be made. TPO-RAs step in to boost platelet production and restore balance.

Three TPO-RAs are FDA-approved for persistent or chronic ITP:

• Eltrombopag (Promacta) — This oral (pill) option was approved in 2008 for adults with chronic ITP who haven’t responded well to steroids, immunoglobulins, or surgery. In 2015, it gained approval for children aged 1 year or older who haven’t responded to other treatments.
• Romiplostim (Nplate) — This once-weekly injection was first approved for adults with chronic ITP in 2008. It is now also approved for children aged 1 year or older who haven’t responded to other treatments.
• Avatrombopag (Doptelet) — This oral option is now approved for adults and children over the age of 1 with persistent or chronic ITP who haven’t responded to other treatments. This treatment increases platelet levels quickly and reduces the risk of bleeding events.

Studies show that many adults respond well to these newer treatments. Many people reach safe platelet counts and reduce their risk of bleeding events. Treatment is usually long term.

While not FDA-approved for chronic ITP specifically, lusutrombopag (Mulpleta) has also been studied for thrombocytopenia related to liver disease.

Side Effects

The overall safety profile of these drugs is good, and serious side effects are rare.

Spleen tyrosine kinase (SYK) inhibitors help protect the platelets you already have. In ITP, autoantibodies can attach to platelets, marking them for destruction by immune cells called macrophages. SYK inhibitors block a signal these macrophages use to recognize and destroy antibody-coated platelets. This can help raise platelet counts and reduce the risk of bleeding.

Fostamatinib (Tavalisse) is currently the only SYK inhibitor approved for ITP. The FDA approved it in 2018 for adults with chronic ITP who haven’t had an adequate response to a previous treatment. It may be considered as a later treatment option when initial therapies haven’t worked well enough.

Side Effects

Most people tolerate fostamatinib well, but some may experience side effects. The most common side effects include diarrhea, elevated liver enzymes, neutropenia (a decrease in the cells that fight infections), high blood pressure, tiredness, and abdominal pain. Your healthcare provider usually checks blood pressure and liver function during treatment.

A newer type of treatment for persistent or chronic ITP is called a BTK inhibitor. BTK stands for Bruton’s tyrosine kinase — an enzyme that helps B cells grow and make antibodies. In chronic ITP, B cells make harmful autoantibodies that mistakenly tag platelets for destruction.

These medicines block BTK, which reduces B-cell activity. BTK inhibitors also help protect your platelets by reducing immune-cell processes that lead to platelet destruction.

Rilzabrutinib

Rilzabrutinib (Wayrilz) is the first BTK inhibitor approved by the FDA for ITP. This medicine is taken by mouth. It’s approved for adults with persistent or chronic ITP who did not respond well enough to a previous treatment.

One study found that about 23 percent of adults with ITP who took rilzabrutinib showed a strong platelet response, while nobody in the control group (people in the study who did not receive the treatment) had a platelet response. This means their platelet counts rose to safer levels.

Side Effects

So far, rilzabrutinib has shown a good safety profile. Mild side effects may include nausea, diarrhea, or headache. Serious adverse events have been rare.

Your immune system regulates your antibody levels using a protein known as the neonatal Fc receptor (FcRn). The idea behind this is 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.

Efgartigimod

There are currently no FDA-approved FcRn blockers to treat ITP. However, scientific research is underway. One drug under review as a treatment for chronic ITP, efgartigimod, has shown mixed results in clinical trials.

Rozanolixizumab

Rozanolixizumab has also been studied in adults with persistent or chronic ITP. Late-stage research trials were started but not completed, and the drug is not FDA-approved for ITP.

Rozanolixizumab is approved for another autoimmune disease, so more evidence and regulatory review would be needed before it could be used as an approved ITP treatment.

Monoclonal antibodies are lab-made proteins that target specific parts of your immune system. For chronic ITP, scientists are studying new ones that may work better and last longer.

Ianalumab

Ianalumab works by blocking a signal called B-cell activating factor (BAFF). The drug helps reduce harmful antibodies and allows platelet counts to rise. Studies have shown that ianalumab may increase platelet counts in people whose ITP didn’t improve with other therapies.

Late-stage research results from 2025 on ianalumab plus eltrombopag showed promising results, significantly prolonging time to treatment. This may become an important treatment if it’s approved for ITP by the FDA.

Daratumumab and Mezagitamab

Two treatments that show promise for chronic ITP are daratumumab and mezagitamab. They target CD38-positive plasma cells or plasmablasts to block cells and stop them from making autoantibodies that destroy platelets.

These new monoclonal antibodies aren’t yet approved for ITP, but the data looks promising so far. There may be more treatment options in the future for people who haven’t responded to other therapies.

The Future of ITP Treatment: New and Upcoming Studies

Research into chronic ITP has been moving quickly, with the recent FDA approval of rilzabrutinib and pediatric expansion of avatrombopag. Newer types of drugs are being studied as future treatment possibilities.

One promising area is complement inhibition. A drug called sutimlimab targets the complement system — a part of the immune system that can destroy platelets. In an early trial, some people had a rise in platelet counts in just 24 hours.

Another study is testing bortezomib — a medicine already used for multiple myeloma. Scientists are studying whether it can help reduce the long-lived plasma cells that make harmful antibodies in ITP.

Finally, researchers are also studying sirolimus. This drug has shown success in other autoimmune blood disorders and could help people with hard-to-treat ITP.

Join the Conversation

On myITPteam, people share their experiences with chronic immune thrombocytopenia, get advice, and find support from others who understand.

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