Thrombotic Thrombocytopenic Purpura

Thrombotic thrombocytopenic purpura (TTP) is a rare blood disorder. With TTP, blood clots (thrombi) develop in small blood vessels throughout your body. These thrombi can restrict blood flow to your body’s vital organs, including your brain, kidneys and heart. As a result, serious medical problems can arise.

The name of this condition provides clues about what’s involved:

• Thrombotic refers to the thrombi — or blood clots — that develop.
• Thrombocytopenic means you have a reduced number of platelets in your blood. Platelets are necessary because they form blood clots that stop bleeding. But with TTP, they get used up making blood clots you don’t need.
• Purpura refers to purplish dots and bruises on your skin that may resemble a rash. This is a sign of bleeding beneath your skin, which is common in TTP.

TTP is a serious condition that comes on quickly and needs urgent medical care. It can come back after treatment, but when it’s active, the symptoms are usually clear. Sometimes it’s triggered by things like a viral infection, surgery or pregnancy. Without treatment, TTP can be life-threatening.

Symptoms of TTP

TTP usually comes on quickly, and while it can come back in some people, the symptoms don’t come and go. They tend to be steady and noticeable. It won’t improve on its own and always needs treatment.

TTP causes blood clots to form in small blood vessels throughout your body. These clots can affect any organ, but your brain is often the first place symptoms show up.

If clots form in your heart, they can cause dangerous heart rhythms or even death. TTP also leads to anemia and low platelet levels (called thrombocytopenia). Some people may have bleeding, but that’s less common.

Common thrombotic thrombocytopenic purpura symptoms include:

• Altered mental status
• Confusion
• Fatigue
• Headaches
• Nausea and vomiting
• Pale or yellowing skin (jaundice)
• Shortness of breath (dyspnea)
• Tiny red or purple dots beneath your skin (petechiae)
• Urine, or pee, with blood in it (hematuria)
• Vision changes, like blurred or double vision (diplopia)

Thrombotic thrombocytopenic purpura causes

The clots that cause problems with TTP form when you don’t have enough ADAMTS13 enzyme. This enzyme prevents platelets from forming blood clots when you don’t need them. When there’s a deficiency of ADAMTS13, clots form in small blood vessels throughout your body. Red blood cells can break apart when they collide with a clot.

Because so many platelets are used up to form unneeded clots, there aren’t many left over to help with clotting when you actually need it (like when you cut your skin). If your platelet count gets very low, you may bruise more easily or bleed more than usual.

TTP can be:

• Acquired (most common). Acquired, or immune-mediated, TTP happens when your body mistakenly makes antibodies that prevent ADAMTS13 enzyme from working correctly.
• Inherited (rare). This happens when both biological parents pass down a faulty gene for making the ADAMTS13 enzyme. Symptoms often start during pregnancy or when your body is under stress.

What triggers TTP?

Experts aren’t sure about what triggers symptoms of thrombotic thrombocytopenic purpura. But some people experience symptom flares associated with:

• HIV
• Lupus
• Pregnancy
• Certain medications, like chemotherapy, cyclosporine A and quinine

Complications of TTP

Thrombotic thrombocytopenic purpura can be life-threatening without treatment. The blood clots can block blood flow, leading to complications like:

• Brain damage
• Heart attack
• Kidney dysfunction
• Pregnancy loss
• Seizure
• Stroke

How doctors diagnose TTP

Your healthcare provider will perform a physical exam and ask about your symptoms and health history. If your provider suspects TTP, they might need to run tests like:

• ADAMTS13 assay. This test measures how active the ADAMTS13 enzyme is. Low levels confirm a TTP diagnosis.
• Complete blood count (CBC). A CBC counts your blood cells. People with TTP have a lower number of red blood cells and platelets.
• Peripheral blood smear (PBS). Your provider examines a blood sample under a microscope. If you have TTP, your red blood cells will be broken and torn (schistocytes).
• Bilirubin test. Bilirubin is a waste product of damaged red blood cells. High bilirubin levels may mean that you have TTP.
• Kidney function tests. These tests can tell your provider if your kidneys are functioning properly. If you have TTP, there may be blood cells or protein in your urine.
• Lactate dehydrogenase test. Lactate dehydrogenase (LDH) is an enzyme that tissues in your body release when they’re damaged. TTP raises the LDH levels in your blood.

How is thrombotic thrombocytopenic purpura treated?

In most cases, providers use plasma therapy to treat TTP. Other options include medication.

Plasma treatments

The most common treatments involve infusions:

• Plasma exchange replaces abnormal plasma with healthy donor plasma — a life-saving treatment for acquired TTP.
• Corticosteroids help treat acute TTP by reducing symptoms and inflammation.

Plasma therapy happens in a hospital. Your care team continues treatment until your symptoms improve. This may take days or weeks.

Medication

Certain medications can slow or stop anti-ADAMTS13 antibodies from forming. Medications healthcare providers use to treat TTP include:

• Glucocorticoids. This treatment suppresses your immune system and may be of benefit in TTP.
• Caplacizumab. This medication treats acute TTP by raising platelet counts faster than plasma exchange alone, leading to quicker remission.
• Rituximab. This treatment can prevent relapse after treatment with corticosteroids and plasma exchange.
• Recombinant ADAMTS13. This treatment replaces the ADAMTS13 enzyme. It’s U.S. Food and Drug Administration (FDA)-approved to treat the inherited form of TTP during emergencies. Sometimes, providers use it to prevent excess bleeding. Research is ongoing to test its uses for the immune type, too.

Surgery

In rare cases, your provider may recommend surgical spleen removal (splenectomy). You might need this if you have severe TTP that doesn’t respond to treatment or keeps coming back.

How long does it take to recover from thrombotic thrombocytopenic purpura treatment?

Recovery depends on several factors, including the severity of your condition and the treatment that you receive. Most people who have plasma therapy for TTP will need several days up to a few weeks. Those who have a splenectomy usually need about four to six weeks to recover.

When should I see my healthcare provider?

TTP symptoms are usually serious enough to require an emergency room visit. If TTP is suspected, don’t wait for a routine appointment — immediate treatment is critical. Managing acute TTP requires the expertise of hematologists and intensive care specialists.

What can I expect if I have thrombotic thrombocytopenic purpura?

TTP used to be fatal. But with fast treatment, the death rate is now around 10%. Because relapses can happen, it’s important to stay in close contact with your hematologist after treatment. Regular monitoring of your ADAMTS13 level helps catch changes early. If the level drops before symptoms return, preventive treatment with rituximab can help stop a relapse before it starts.

What is the survival rate of TTP?

Without treatment, only 1 out of 10 people with TTP survives. But with proper care, the survival rate goes up to 8 or 9 out of 10 people.

This is why it’s essential to seek treatment if you’re experiencing symptoms. And it’s important to follow your provider’s guidance about caring for yourself once you’re diagnosed.