Thrombotic Thrombocytopenic Purpura (TTP)

What is TTP?

Thrombotic thrombocytopenic purpura (TTP) is a rare and serious blood disorder. It causes blood clots to form in small blood vessels throughout the body. 

Blood clots can block blood flow and decrease the amount of oxygen that can reach important organs such as the brain and kidneys. This can cause serious medical issues, such as organ damage or stroke. 

Because TTP causes more clotting than usual, the body uses more platelets. This lowers the number of platelets in the bloodstream. Platelets are blood cells that help stop bleeding. When your child’s platelet count is low, it can cause your child to bruise and bleed more easily. Your child might bleed more than normal when hurt or bleed for no reason.

The clots float in the blood and damage red blood cells, causing them to break down. This leads to a rare type of anemia called hemolytic anemia.

TTP can develop suddenly. It usually lasts for days or weeks but recovery can take months.

TTP can be life-threatening. It is important to get treatment for TTP right away.

Recovery can take several weeks or months. TTP flare-ups or relapses can happen. Ongoing medical care is important.

Symptoms of TPP

Symptoms of TTP can be caused by blood clots, low platelet counts, or damage to red blood cells.

Signs and symptoms of TTP include:

• Petechiae: Tiny red or purple “pinpoint” dots on the skin that may look like a rash
• Purpura: Red or purple spots on the skin caused by bleeding
• Jaundice: A yellowish color of the skin or whites of the eyes
• Unexplained bleeding or bruising
• Paleness
• Feeling tired or weak
• Fever
• Headache
• Nausea or vomiting
• Fast heart rate
• Shortness of breath
• Confusion, changes in speech, blurry vision, or seizure
• A low amount of urine (pee) or protein or blood in your urine

If your child has any of these symptoms, contact your care team right away.

Causes of TTP

TTP happens when there is a problem with an enzyme called ADAMTS13. This enzyme breaks down von Willebrand factor (VWF) into smaller pieces. VWF is a protein that helps blood to clot. When ADAMTS13 does not work properly, platelets can stick together and make too many blood clots. 

TTP is more common in females and in people of Black or African backgrounds.

Types of TTP

There are 2 types of TTP: acquired and inherited.

Acquired TTP

Acquired means the person is not born with the disease. The body makes antibodies (proteins) that stop the ADAMTS13 enzyme from working properly. Acquired TTP is the most common type of TTP. It does not have a clear cause. There is no way to prevent it.

Inherited TTP

Inherited or hereditary TTP occurs when there is a problem with the gene that controls ADAMTS13. Parents can pass the gene change (mutation) for inherited TTP on to their children. This is a rare cause of TTP. Symptoms often develop at an early age but in some cases may not appear until adulthood.

Diagnosis of TTP

Your child’s doctor may refer them to a hematologist. A hematologist diagnoses and treats blood disorders. TTP is diagnosed based on your child’s medical history, physical exam, and lab tests. The doctor may ask about any changes in bleeding or bruising, recent illnesses, or medicines your child is taking.

Lab tests may include:

• ADAMTS13 test: This measures how active the ADAMTS13 enzyme is. If the activity is low, your child may have TTP.
• Blood smear test: Doctors place a small amount of your child’s blood on a slide. They look at the blood under a microscope. If your child has TTP, the red blood cells appear broken or torn.
• Bilirubin test: When red blood cells are damaged, they release hemoglobin into the blood. Hemoglobin breaks down into bilirubin. High levels of bilirubin can indicate TTP.
• Complete blood count (CBC): This test measures the number of red blood cells, white blood cells, and platelets in your child’s blood. People with TTP have low numbers of red blood cells and platelets in their blood.
• Creatinine test: Creatinine is a blood waste product that is usually removed by the kidneys. Those with TTP may have high levels of creatinine in the blood.
• Lactate dehydrogenase (LDH) test: This is an enzyme found in most cells. When cells break, the enzyme releases into the blood. If your child has TTP, their LDH levels will be higher than normal.
• Urine and kidney function tests: These tests can tell if your child’s kidneys work as they should. If your child has TTP, they may have protein or blood cells in their urine.
  

Treatment of TTP

TTP can be life-threatening if it is not treated right away. Your child needs treatment to prevent a stroke, seizure, bleeding, or further damage to their organs. Most children do well when TTP treatment begins soon after diagnosis.

Plasma treatments are the most common ways to treat TTP. It can also be treated using medicine or surgery.

Plasma treatments

There are 2 kinds of plasma therapy: plasma exchange and fresh frozen plasma infusion.

Therapeutic plasma exchange (plasmapheresis) is used to treat acquired TTP. Plasma exchange removes unhealthy plasma, the liquid part of the blood, and replaces it with healthy plasma from a donor.

Learn more about therapeutic plasma exchange.

Fresh frozen plasma is used to treat inherited TTP. Your child receives healthy plasma from a donor through an IV in a vein. The plasma infusion replaces the missing or faulty ADAMTS13 enzyme.

Your child repeats plasma therapy treatments until symptoms get better. This can take some time. Talk to your care team about how long it may take for your child.

Medicines

For Immune TTP, your child’s doctor may prescribe medicines to help the immune system stop attacking ADAMTS13. These medicines include steroids and rituximab.

Your health care provider might prescribe other medicines for TTP. This includes caplacizumab, a medicine which acts to help prevent platelets from sticking together so that blood clots don’t form.

Surgery

Rarely, surgeons must remove a child’s spleen (splenectomy) if other treatments do not work. Antibodies that attack the ADAMTS13 enzyme are created in the spleen. Removing the spleen prevents the body from creating these antibodies.

Living with TTP

With proper care, your child can live without symptoms. If symptoms return, contact your hematology care team as soon as possible to restart treatment.

To help keep your child safe, talk to your child’s care team before:

• Starting or taking any new medicines that could thin the blood
• Doing intense physical activity or activities that could put your child at risk for falls or injury

TTP is a serious condition. If symptoms appear, they must be treated right away.

Be sure your child gets all tests and medical care as recommended. Always follow the instructions given by your care team.

Questions to ask your care team

• What are our treatment options?
• How will TTP affect my child’s life?
  
• Will TTP come back after treatment?
  
• What is the long-term outlook for children with TTP?
  
• Will my child need to see other doctors?
  
• What warning signs should we watch for?
  

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