Immune thrombocytopenia (ITP)

  • Immune thrombocytopenia (ITP), also called idiopathic thrombocytopenic purpura, is an autoimmune disorder that occurs when the body attacks its own platelets and destroys them too quickly. Platelets are a part of blood that helps control bleeding. ITP affects at least 3,000 children under the age of 16 each year in the United States.

    While ITP often arises after a viral infection, for the majority of cases the cause is unknown.

    Luckily, acute ITP, the most common form, usually goes away on its own over the course of weeks or months, sometimes without treatment. Chronic ITP, which appears most frequently in adults, occasionally is seen in children. This form of ITP is more serious, lasting for years and typically requiring specialized follow-up care.

    ITP Treatment at Dana-Farber/Boston Children's Cancer and Blood Disorders Center

    Children and young adults with immune thrombocytopenia are treated through the Platelet Function Disorders Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.


  • What is immune thrombocytopenia?

    Immune thrombocytopenia (ITP) is an autoimmune disorder (meaning the immune system attacks the body’s own tissues) that occurs when the body attacks its platelets, a part of the blood that helps control bleeding by forming blood clots.

    • In healthy children, the body produces proteins called antibodies that guard against infection.
    • ITP causes a child’s body to make abnormal antibodies that stick to platelets, which the spleen (the organ that helps filter infections in the blood) recognizes as signs of infection and destroys.
    • In a child with ITP, the body is producing platelets normally but also destroying them too quickly, with platelets surviving only a few hours instead of the normal seven to 10 days.
    • The end result is a low platelet count in the blood.

    There are two kinds of ITP. Acute thrombocytopenia is the most common form of ITP—accounting for more than 90 percent of cases—and occurring between the ages of 2 and 6.

    • The symptoms arise suddenly and usually disappear in less than six months, often within a few weeks.
    • Acute ITP usually does not recur after the child gets better.

    Chronic thrombocytopenia is more common in adults but can occur in children.

    • Symptoms of chronic ITP last a minimum of six months and can persist for many years.
    • Chronic ITP can require regular follow-up care with a hematologist (a doctor who specializes in blood disorders).

    Symptoms

    Each child may experience symptoms differently, but the symptoms of ITP include:

    • purpura (purple color of the skin after blood has leaked under it, forming a bruise, often from no known trauma)
    • petechiae (tiny red dots under the skin that are the result of very small bleeds into the skin)
    • nosebleeds
    • bleeding from the mouth
    • blood in vomit, urine or stool
    • in rare cases, life-threatening bleeding inside the brain, usually prompted by head trauma

    Causes

    What causes ITP?

    Often, a child may have had a viral infection a few weeks before developing symptoms of ITP. But in most cases, the underlying cause of ITP is not known.

    • Viral infections may trigger ITP, because the antibodies that the body develops to fight them can also attack platelets.
    • ITP is not contagious, meaning your child cannot catch it from playing with another child who has ITP. Nor can she spread it to other children.
  • 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
    • complete blood count (CBC), including a hematologist’s examination of the blood under the microscope

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

  • There are a number of treatments that can help increase platelet levels in children with immune thrombocytopenia (ITP), but there is no cure. The majority of children with ITP get better gradually on their own in a few days, weeks or sometimes months, with or without treatment.

    When treatment is necessary, the most common forms are:

    • Steroids – to temporarily reduce production of antibodies and increase platelet count by slowing the rate at which the spleen destroys them
    • Intravenous gamma globulin – a human blood product containing antibodies that help slow the rate at which abnormal platelets are destroyed by the spleen
    • Intravenous Rh immune globulin – a human plasma product that temporarily stops the spleen from destroying platelets; children must be blood type Rh positive and still have their spleen to receive this treatment

    Steroids:

    • temporarily reduce production of abnormal antibodies and increase platelet count by slowing the rate at which platelets are destroyed by the spleen
    • may be taken orally
    • side effects may include irritability, stomach irritation, weight gain, high blood pressure, acne or elevated levels of sugar in the urine

    Intravenous gamma globulin:

    • a human blood product containing many antibodies that help slow the rate at which abnormal platelets are destroyed by the spleen
    • given over three to six hours through a needle inserted into a vein
    • temporary side effects include fever, chills, headache, muscle and joint pain, hives, rash or allergic reactions

    Intravenous Rh immune globulin:

    • temporarily stops the spleen from destroying platelets
    • child must have Rh-positive blood and must still have their spleen in order for this medication to be effective
    • given intravenously
    • temporary side effects include: mild anemia, fever, chills, headache, blood pressure changes or allergic reactions
    • rarely, severe anemia from breakdown of blood cells can occur; this can possibly result in kidney damage

    Other treatments for ITP may include:

    • surgery to remove spleen (considered more often in older children with chronic ITP)
    • hormone therapy (for teenage girls, to stop their menstrual cycle if excessive bleeding occurs)

    Children with ITP also may  receive antibiotics to treat infections.

    Progressive or recurrent disease
    Recurrence of ITP is uncommon, but it can occur up to several years after the initial episode and may be associated with another viral infection.

    Long-term outlook
    More than 80 percent of children with treated ITP recover on their own in days, weeks or months. Fatal brain hemorrhages rarely occur with steroid, intravenous Rh immune globulin or intravenous gamma globulin therapy.

    Can my child participate in sports or other athletic activities?
    The sports and activities that your child can participate in will depend on her platelet count (the severity of the ITP). Your child’s physician can make specific recommendations on the types of activities that may be appropriate for her depending on her platelet levels.

    How Dana-Farber/Boston Children's approaches ITP
    Children and young adults with immune thrombocytopenia are treated through the Platelet Function Disorders Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

    Dana-Farber/Boston Children’s seamlessly integrates two internationally-recognized research and teaching institutions that have provided comprehensive care for pediatric cancer and blood disorder patients since 1947. Working together, we provide more specialists, more programs, and more clinical trials than almost any other pediatric center treating cancer and blood disorders in the world.

  • Dana-Farber/Boston Children's Cancer and Blood Disorders Center is a world leader in ITP research. We are currently conducting a number of studies to improve the diagnosis and treatment of ITP and other platelet disorders.

    • Rituximab: We are conducting a clinical trial to determine the safety of the medication rituximab and how well it works in children and adolescents with chronic, severe ITP.
    • North American ITP registry: Our Platelet Function Disorders Program has formed a North American database to help understand the long-term outcomes of children and adolescents with ITP. Once this information is analyzed, it may lead to a better understanding of chronic ITP in North America and improve the way specialists care for their young patients with this chronic disease.
    • International splenectomy registry: Researchers are collecting information internationally regarding splenectomy (surgical removal of the spleen) in young persons with ITP. This study may lead to the better understanding of the effects of splenectomy on children and young adults with ITP.
    • Intercontinental childhood ITP registry: We participate in a large, international study to help better define the long-term course in children newly diagnosed with ITP. Once this information is carefully analyzed, it may lead to the development of new treatment guidelines that will improve the care of children with ITP.
    • Study of treatment side effects: We also are participating in multicenter studies to evaluate the side effects of the various treatments of ITP.

    Clinical Trials

    For many children with rare or hard-to-treat conditions, clinical trials provide new options.

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