When Your ITP Treatment Doesn’t Work, What’s Next?

When you first started treatment for immune thrombocytopenia (ITP), you felt optimistic about how it might help raise your platelet count and manage the disease.

But it hasn’t been as successful as you and your healthcare provider hoped. So … now what?

Hematologist Keith McCrae, MD, explains what may be next if your initial ITP treatment isn’t doing the trick.

What if your first treatment doesn’t work?

The first treatment doctors try for ITP is typically a round of corticosteroids (for six weeks or fewer), plus one of the following:

• Rituximab, a monoclonal antibody that reduces the autoantibodies that are attacking your platelets
• A thrombopoietin receptor agonist (TPO-RA), a medication that increases your platelet count by mimicking the hormone thrombopoietin

But if this treatment doesn’t raise your platelet count to where it should be, your provider will want to revisit your treatment plan to see what needs to change. First, they may consider changing a medication.

“If you initially received corticosteroids and rituximab, your provider may consider a TPO-RA instead of rituximab,” Dr. McCrae explains. “Or if you first tried steroids plus a TPO-RA, your provider may consider switching you to a different TPO-RA or trying rituximab instead.”

He also notes that, until recently, corticosteroids alone were considered the go-to first treatment for ITP. If this is what you tried and it didn’t work, your provider will consider adding rituximab or a TPO-RA.

When doctors turn to other treatments

There are times when none of the initial treatments for ITP are the right fit — like if you can’t or don’t want to take these medications, or if they don’t work for you. Then what?

In these cases, your provider may consider other medications, such as:

• BTK inhibitors: “These medications help stop your immune system from destroying your platelets,” Dr. McCrae explains. One of them, rilzabrutinib (Wayrilz®), is approved for people with persistent or chronic ITP who haven’t had success with first-line therapy.
• SYK inhibitors: These medications, which are part of a class of drugs called tyrosine kinase inhibitors (TKIs), interfere with the process that destroys your platelets. Fostamatinib (Tavalisse™) is the first SYK inhibitor approved to treat chronic ITP if first-line therapies haven’t worked.
• Mycophenolate mofetil (MMF): This medication (CellCept®) is usually used to prevent organ rejection in transplant recipients. “In people with ITP, it can help suppress the overly reactive cells that are attacking your platelets,” Dr. McCrae says.

Right now, there’s not enough head-to-head data to rank these three treatments by order of effectiveness. So, which one will your provider try? That depends on a number of different factors that are all related to ... well, you.

“In deciding which option may be best for you, your provider will consider factors like possible side effects, disease progression and your personal preferences,” Dr. McCrae says. “They’ll also take into consideration things like medication availability and cost.”

What about spleen removal surgery?

You might’ve heard that a splenectomy (surgery to remove your spleen) can be an effective treatment for ITP.

“Splenectomy used to be a common treatment for ITP, but it’s rarely done now,” Dr. McCrae clarifies.

Still, if you’ve been trying medications for more than a year without success, it may be appropriate to discuss the possibility of surgery.

What about IVIG?

While you’re receiving treatment for ITP, you may also receive a therapy called intravenous immunoglobulin (IVIG). It’s an infusion of antibodies collected from donated blood plasma that can help temporarily raise your platelet count.

“The effects of IVIG are temporary, so it’s not typically used for long-term maintenance,” Dr. McCrae notes. “But it can be given alongside other treatments for ITP.”

Don’t forget about clinical trials

Your provider may also talk to you about the possibility of participating in a clinical trial, a research study looking into a potential treatment for ITP.

“When possible, we try to enter people into new clinical trials if they’re eligible and interested,” Dr. McCrae says.

Final thoughts

Today, there are more treatment possibilities than ever for people living with immune thrombocytopenia. And there may be more to come.

“New advances in ITP management have dramatically improved our treatment options, and researchers continue to study additional approaches,” Dr. McCrae says.

If your first treatment isn’t working as you’d hoped, don’t hesitate to talk to your healthcare provider about your options.

“No person is the same, and no person’s ITP is the same,” Dr. McCrae encourages. “There are many possible options that we can consider so that we can give you the best possible opportunity for treatment-free remission.”