Immune Thrombocytopenia (ITP)

Immune thrombocytopenia (ITP) is an autoimmune disorder in which the body attacks its own platelets, blood cells that help control bleeding. Formerly called immune thrombocytopenic purpura or idiopathic thrombocytopenic purpura, ITP affects at least 3,000 children under the age of 16 each year in the United States.

When a child has ITP, the immune system “sees” healthy platelets as foreign, like an infection, and starts to destroy them. The end result is a low platelet count, which prevents the blood from forming clots and increases the risk of bleeding.

Common symptoms of ITP include:

• Sudden appearance of bruises in an otherwise healthy child
• Pinpoint-sized red dots on the skin (petechiae)
• Bloody noses
• Bleeding from the gums
• Heavy menstrual bleeding
• Blood in the urine or stool
• Fatigue

In most children, ITP goes away on its own within six to 12 months of diagnosis. ITP is more likely to resolve in children who are diagnosed at a young age with a very low platelet count.

Immune thrombocytopenia in children sometimes occurs after a viral infection. However, for the majority of children the exact cause is unknown. In most children, ITP is not associated with any other medical problems. However, sometimes ITP can occur as a complication of other conditions.

The first step in treating your child is forming an accurate and complete diagnosis. ITP can usually be identified by:

• A careful medical history
• Physical examination
• A complete blood count (CBC), including a hematologist’s examination of the blood under the microscope

After all tests are completed, your care team will be able to outline the best treatment options.
 

There are a number of treatments for ITP that can help increase platelet levels to decrease or prevent bleeding in children with ITP. While every child responds differently, the majority of cases of ITP in children will resolve within six to twelve months after diagnosis, with or without treatment.
 
We customize treatments individually for each patient. The available medications work in different ways to prevent the immune system from attacking platelets.

When medication is needed to quickly raise the platelet count, the most common choices are steroids and intravenous immunoglobulin (IVIG). These medications are not effective in every child with ITP and may work for only a brief amount of time, after which symptoms can return. 

Fortunately, many additional options have become available, including those listed below. Your hematologist can discuss these with you in detail. 

Treatment options for ITP

• Steroids: Steroids temporarily calm the immune system to slow the rate at which platelets are destroyed. They can be taken by mouth. They do have side effects, including irritability, trouble sleeping, upset stomach, weight gain, acne, high blood pressure, and high blood sugar.
• Intravenous immunoglobulin (IVIG), also known as intravenous gamma globulin: IVIG contains antibodies that help slow the rate at which platelets are destroyed. It is given intravenously, through a needle inserted into a vein, over the course of three to six hours. It has temporary side effects, including fever, chills, headache, muscle and joint pain, hives, rash, and allergic reactions.
• Thrombopoietin (TPO) receptor agonists: This family of medications increases platelet production. They include eltrombopag, a daily oral medication; romiplostim, a weekly injection under the skin; and avatrombopag, a daily oral medication. These, too, can have side effects, including headaches, an elevated platelet count, and a risk of blood clots.
• Rituximab: Rituximab calms the immune attack on platelets by suppressing B cells, the white blood cells that produce antibodies. It is given as an IV infusion, typically weekly for four weeks, and works for about six to 12 months. It has temporary side effects, including immune reactions to the infusion, joint pain, rash, fever, and an increased risk of infections.
• Mycophenolate and sirolimus: These oral medications help prevent attacks on platelets by inhibiting activity of white blood cells called lymphocytes (B and T cells). Each has different side effects that your care team can discuss with you.
• Hydroxychloroquine: Hydroxychloroquine is sometimes prescribed if ITP is accompanied by other autoimmune disorders.
• Other treatments: As needed, our team may offer hormone therapy if patients have heavy menstrual bleeding and anti-fibrinolytic medications (aminocaproic acid, tranexamic acid) to reduce nosebleeds, gum bleeding, or heavy menstrual bleeding.

More than 80 percent of children with ITP recover on their own in six to twelve months regardless of whether they receive medication. After ITP resolves, it can occasionally occur again up to several years after the first episode, sometimes triggered by a viral infection.

The sports and activities your child or teen can participate in will depend on their platelet count and risk for bleeding. Your child’s health care provider can make specific recommendations on the types of activities they can participate in safely.

Children, teens, and young adults with immune thrombocytopenia are treated through the Blood Disorders Center at Dana-Farber/Boston Children’s, within the Immune Hematology Program. Our expert hematologists will evaluate you or your child to get a precise diagnosis and determine the best course of treatment.

As leaders of the Pediatric ITP Consortium of North America (ICON), a consortium of centers in the U.S. and Canada, we can provide opportunities to participate in clinical trials of new treatment approaches. We are currently conducting a number of studies to improve the diagnosis and treatment of ITP and better understand how it impacts children and their families.

The Platelet Disorder Support Association was founded by patients to educate and empower those with immune thrombocytopenia and other platelet disorders. It provides extensive information on ITP and support resources for families.