Treatment Options for Immune Thrombocytopenia (ITP)

Immune thrombocytopenia (ITP) is a condition where the immune system targets and destroys platelets, small red blood cells that help the blood clot when there is an injury. ITP causes platelet counts to become too low, which can result in numerous bleeding symptoms, such as bleeding excessively from injuries, bruising easily, or bleeding for no noticeable reason.

ITP is different for everyone. In some cases, the only treatment required is monitoring. Patients who experience a bleed or injury may require hospitalization and platelet transfusions. For others, low platelet counts from ITP can persist for years, and require ongoing therapy. The appropriate treatment for ITP will depend on a number of factors, including a person’s age, platelet count, symptoms, medical history, career, and lifestyle.

The following slides look at different treatment options for adults who have been diagnosed with ITP.

Corticosteroids are anti-inflammatory drugs used to treat a wide variety of conditions—asthma, allergies, arthritis, MS (to name a few). While they are a variety of steroid, corticosteroids are an altogether different drug than the steroids used to build muscle and boost athletic performance. Corticosteroids reduce the activity of the immune system, and in people with ITP this slows the autoimmune response that destroys blood platelets, which allows platelet counts to increase. The corticosteroids that are prescribed for ITP are taken as pills. They are a first-line therapy prescribed for ITP, but long-term or repeated use can cause numerous side effects and is not recommended.

Immunoglobulin is another first-line therapy. Immunoglobulin is a blood product that contains a concentration of antibodies and is taken as an intravenous infusion. Antibodies are proteins that are made by the immune system that fight off infectious agents and other foreign invaders. Infusions of immunoglobulin are used to treat immunodeficiency and conditions where the immune system attacks healthy cells (like ITP). Immunoglobulin is believed to regulate immune responses. There are numerous types of immunoglobulin products available. IVIG and anti-D immunoglobulin are the types prescribed for short-term treatment of ITP.

Immunosuppressant drugs are drugs that suppress the immune system. More specifically, they are drugs that disrupt certain functions of the immune system. They are prescribed for a number of different health conditions, including autoimmune diseases like psoriasis, lupus, Crohn’s disease, and multiple sclerosis. Though not approved for treating ITP, certain immunosuppressant drugs have nonetheless been prescribed to treat ITP, with some promising results. Immunosuppressant drugs increase the risk of infections and some types of cancers, and cannot be taken by pregnant women.

There are a number of other medications that are used to treat ITP. These are given as second or third line treatments when other treatments have not worked:

• Chemotherapy drugs, which are a staple of cancer treatment, but can be useful in treating ITP due to their ability to regulate cell production.
• Spleen tyrosine kinase (SYK) inhibitors, drugs that work by blocking a protein that signals antibodies to destroy platelets.
• Thrombopoietin receptor agonist drugs, which boost platelet production in the bone marrow.

There are also several therapies that are being studied as treatments for ITP, including combination therapy, the use of two or more separate treatments taken simultaneously. Some of these are being tested in clinical trials.

Surgery to remove the spleen—called splenectomy—is another treatment option for ITP. Why the spleen? The spleen is where the antibodies that attack platelets are produced. In previous decades, this was a go-to treatment if ITP did not respond to corticosteroids. It has proven an effective treatment option for many—but not all—patients. Today, with more treatment options available, and more treatment options being developed, it is a third line treatment, used when ITP has not responded to other treatments. There are a number of risks associated with splenectomy. Living without a spleen puts a person at a higher risk of serious infections and increases the risk of blood clots. Surgery is also risky when platelet counts are low.