How Doctors Decide Which Treatment To Try for Immune Thrombocytopenia

In adults, the rare blood disorder immune thrombocytopenia (ITP) occasionally resolves on its own. But more often, it’s a chronic disease that may require treatment.

But there’s good news: These days, there are more options than ever for helping manage ITP.

“The treatment landscape for ITP has changed dramatically in the last 20 years,” says hematologist Keith McCrae, MD.

Dr. McCrae gives an overview of possible treatments and explains how your healthcare team weighs which ones to try.

What are the options for ITP?

ITP causes your immune system to attack and destroy your own platelets, so the goals of treatment are to raise your platelet count and reduce your risk of serious bleeding.

There’s no one-size-fits-all way to do that. But the initial go-to therapy for ITP typically consists of corticosteroids, in some cases, accompanied by intravenous immunoglobulin (IVIg).

Recent guidelines also suggest the addition of other medications early in the course of ITP, either rituximab or a type of medication called a thrombopoietin receptor agonist (TPO-RA).

“The goal of using combination of medications early in the course of ITP is to increase the likelihood of long-term ITP remission without ongoing treatment,” Dr. McCrae explains. “More intensive treatment may increase the likelihood that you’ll go into remission and no longer require treatment.”

If your first treatment plan doesn’t raise your platelet count as hoped, then your healthcare provider may consider other medications instead.

But how do they decide where to start? Let’s take a look.

How doctors decide which ITP treatment to try

To make decisions about the best way to treat you, your provider will consider a variety of personalized factors.

“We’ll typically present you with options and give you recommendations that weigh your needs and preferences with where you are in the course of the disease,” Dr. McCrae says.

He offers a more in-depth look at some of the factors that come into play.

The specifics of your condition

The specifics of ITP vary from person to person. In deciding how to treat your condition, your provider will consider things like:

• What caused it: Sometimes, ITP is primary, meaning it doesn’t have another underlying cause, or it may be secondary, meaning it’s associated with another condition.
• How long you’ve had it: Treatment may be different depending on how long you’ve been living with ITP. Persistent ITP lasts three to 12 months, while chronic ITP lasts a year or longer.
• The severity of your condition: For mild cases of ITP, providers may recommend monitoring your condition. But very low platelet counts and bleeding symptoms (like unexplained bruising, petechiae and nosebleeds) require treatment.

“Your provider will look at treatment options based on the course of your disease and how well the treatments are likely to work,” Dr. McCrae says.

Other aspects of your health

ITP doesn’t exist in a vacuum. Bodies are complex, and there’s a lot of nuance to every person’s health profile.

“Medication choice depends on each person’s unique situation,” Dr. McCrae acknowledges. “Certain aspects of your health may require special considerations and treatment paths.”

Your provider will account for your whole-body health when considering your treatment plan. The right treatment for you may depend on factors like:

• A history of blood clots
• Chronic infections
• Liver disease
• Other blood disorders or cancer
• Past organ transplant
• Pregnancy

Possible side effects

Every medication brings benefits as well as potential risks, and the medications used to treat ITP are no different. Your provider will weigh how each medication could affect you — beyond just its ability to help manage your ITP.

Corticosteroids are still recommended as the initial treatment of ITP (and often for acute ITP relapses). But in the past, people sometimes took them for long periods of time — and extended corticosteroid use can cause serious side effects, including glucose intolerance, psychiatric concerns, bone loss and more.

“For ITP, we now try to limit steroid courses to six weeks or fewer,” Dr. McCrae says. “And we have many more options for therapies that are effective but without as many risks.”

Newer therapies avoid some of the risks of prolonged steroid use, but they can still cause side effects of their own. Your provider will talk to you about the pros and cons of the medications they propose, but don’t hesitate to ask for more details if you have any concerns.

Your personal preferences

When it comes to treatment plans, what you think, feel and want all matter. For example, Dr. McCrae says many people prefer oral medications (pills) over medications that are given by IV infusion or injection.

Or you might have preferences about how often you need to take medication. Rituximab is typically given as a limited course of infusions, while TPO-RAs often involve regular pills or injections.

“If you take rituximab and go into remission, it might last three months, or it might last three years,” Dr. McCrae notes. “So, if you want to aim for long-term ITP remission, rituximab may be the best option.”

On the other hand, TPO-RAs may offer more predictable results — even though you need to continue taking medication.

“If you place a high value on achieving a lasting improvement in platelet counts but don’t mind staying on a medication, taking a TPO-RA may be the way to go,” he says.

What you’ve already tried

Sometimes, your first treatment plan doesn’t work as hoped. If you’ve already been on medication to treat ITP and your platelet count still isn’t where your provider wants it to be, they’ll look at other options:

• If you’ve only been on corticosteroids, they may add rituximab or a TPO-RA.
• If you were on rituximab, they may try a TPO-RA instead.
• If you were on a TPO-RA, they may try a different TPO-RA or rituximab instead.
• If neither rituximab nor a TPO-RA has worked for you (or if you can’t take them), they may try other medications, like BTK inhibitors, SYK inhibitors or mycophenolate mofetil (MMF).

Each of these medications works a little differently and may affect you differently, too. Your provider will explain what each one entails and why they’re recommending it for you.

Cost and availability

Some medications cost more than others, and your health insurance may not cover them all to the same extent.

“For example, the cost of corticosteroids and MMF is typically minimal,” Dr. McCrae says. “On the other hand, the cost of rituximab and TPO-RAs is moderate, and insurance coverage of them can be unpredictable.”

Your healthcare provider will work with you to try to find a treatment option that’s financially feasible for you.

Final thoughts

There used to be very limited treatment options for immune thrombocytopenia. But today, your healthcare provider has multiple avenues they can pursue to help manage ITP.

To decide which one is right for you, they’ll take into account a variety of factors that are unique to you and your health.

“Our goal is to help you achieve a treatment-free remission,” Dr. McCrae reassures. “And fortunately, these days, there are many possible options that we can turn to in order to help you get there.”