If you need treatment for immune thrombocytopenia (ITP), you've got more medication options than ever before to raise your low platelet count and ease symptoms like bruising, bleeding, and fatigue. Studies on promising experimental medicines are also underway.
"The last few years have been an exciting time for ITP treatment because there have been a handful of new treatments approved," says Anne Hubben, MD, a hematologist specializing in benign blood disorders at Cleveland Clinic.
She says the expected release of new professional guidelines for hematologists means your ITP doctor may offer you treatment earlier in the course of the disease. Here's a look at recent advances in treatment and possible future medicines in the pipeline.
Newer ITP Treatments
In 2025, the FDA approved two medicines for ITP. They offer promising new options for adults and kids in need of alternatives.
Recently approved medicines for ITP include:
Rilzabrutinib (Wayrilz). This medicine treats ITP in adults. It's a tablet you take by mouth twice a day. It's approved for people who haven't been helped by (or have stopped benefiting from) steroids, immune globulin, or other treatments, Hubben says.
In clinical trials, rilzabrutinib raised platelet counts and kept them in a safe range for longer in people who hadn't responded to many other treatments. It may start to work in as little as two weeks and continues to increase the platelet count over time, Hubben adds.
Rilzabrutinib works in three ways, she says: It blocks your immune system from making proteins called antibodies. (These usually fight germs, but in ITP, they mistakenly attack platelets.) The medication also helps prevent your immune system from destroying and removing platelets. And it lowers inflammation inside your body, which can be fueled by ITP.
"One of the advantages to rilzabrutinib and the newer treatments that are being studied is that the companies are studying quality of life and fatigue specifically in ITP," Hubben says. "Rilzabrutinib actually showed a significant improvement in fatigue and in other health-related quality-of-life measures in patients with ITP."
Because rilzabrutinib is a type of medicine that turns down your immune system, it can raise your risk of infections. But you can work with your doctor to prevent infections or get them treated as soon as possible.
Avatrombopag (Doptelet Sprinkle). Children with ITP often have mild symptoms and don't need treatment. But if the condition doesn't go away on its own, your child's doctor will likely recommend ITP medicine.
Doptelet Sprinkle is approved to treat persistent or chronic ITP in children 1 to 5 years old. It helps a child's bone marrow make more platelets. It comes in a form that you sprinkle onto your child's food. A higher risk of infections is one of the side effects.
A tablet version for people ages 6 and older – called Doptelet – earned the FDA's approval in 2019.
A Changing Approach to Treatment
If your platelet levels aren't dangerously low, your ITP doctor may suggest simply tracking your levels over time rather than starting treatment right away. This is a common tactic called "watchful waiting." But many doctors may be shifting their thinking in favor of treating ITP more aggressively earlier.
"One of the changes that we are expecting are new guidelines from the American Society of Hematology recommending using second-line treatments – medicines other than steroids or immune globulin (IVIG) – earlier in the course of ITP," Hubben says. That's because most adults with the condition who need treatment will see their platelet count drop repeatedly over time. Starting treatment sooner may help ITP medicine work even better, she says, and prevent drops in the platelet count.
"So rather than waiting, we're now thinking about adding additional treatments for ITP a little earlier in the disease course," often with the immune system-lowering drug rituximab (Rituxan, Ruxience, Truxima) or with drugs that help your bone marrow make more platelets, like avatrombopag (Doptelet), eltrombopag (Promacta), or romiplostim (Nplate), Hubben says. "You could think about using rilzabrutinib pretty early in the course of disease," too, she adds.
Hubben recommends working closely with your doctor to decide what treatment plan might be right for you, noting that each person with ITP is different. It depends on many things, including your overall health, other health conditions you have, your ITP symptoms, and your preferences about how often you'll need treatment and the side effects it might cause. Not everyone will respond to steroids, IVIG, or newer treatments the same way.
Possible Treatments on the Horizon
Several other promising medicines are being developed and studied for treating ITP. It's a good idea to ask your doctor about any new options and their pros and cons. New medicines and medication classes now under study for ITP include:
Ianalumab. This medication for adults could work by blocking B cells (which make antibodies that attack platelets in ITP) and tagging them for destruction, while recruiting other parts of the immune system for help, Hubben says. It's given by IV once a month for four months. In a clinical trial, ianalumab plus the drug eltrombopag kept the platelet count in a safe range and prevented bleeding or the need for other treatments for longer than eltrombopag alone, Hubben says. If the drug keeps showing promise in late-stage clinical trials, ianalumab may be presented for FDA review in 2027, she says.
Efgartigimod alfa (Vyvgart). While this medicine for adults with ITP is approved in Japan, it's not currently under FDA review in the U.S., Hubben says. That's because study results showing how well it works have so far been mixed. It's given by IV or a shot under the skin. It could work by lowering immune system proteins (antibodies), including those that attack healthy platelets by mistake.
Spleen tyrosine kinase (SYK) inhibitors. In this class of medication, a drug called fostamatinib (Tavalisse) is currently used to treat ITP. It prevents platelets from being destroyed by specialized immune cells called macrophages. Newer SYK inhibitors are being studied looking for better responses and fewer side effects, Hubben says.
Complement inhibitors. This class of medication is also going through early clinical trials for ITP, but it's not yet close to FDA review, Hubben says. Complement inhibitors aim to block overactive parts of the immune system that play a role in the destruction of platelets with ITP, she says. Medicines in this class are already approved to treat multiple other health conditions.
The Bottom Line
Thanks to advances in treatment, you've got lots of options that could help you take charge of ITP if needed. Before you start a treatment, ask your doctor about any side effects it might cause and how to manage them.
The right treatment plan can boost your platelet count into a safe range and help you manage ITP symptoms that can seem scary, like sudden bruising, a rash, blood blisters in your mouth, or bleeding gums. The key is to get medical care right away, rather than ignore these symptoms or hope they'll go away on their own, Hubben says.
"ITP can be effectively managed," she says. "It's important to work with a hematologist who's available and knowledgeable, and you feel comfortable talking with." Let your care team know if you have ITP symptoms beyond bleeding and bruising, like fatigue, which can take a toll on your quality of life.
"It really is a disorder that affects people much more than just their lab value and their platelet count," Hubben says.