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Treatments for ITP
A number of treatment options are available for ITP:
Common initial medical treatments
If you have been diagnosed with ITP and are on traditional ITP therapy, then you may be taking one of several different medicines used to treat ITP.
Common treatments include:
- Intravenous immunoglobulin (IVIG)
All of these work by affecting the immune system in different ways. They maintain platelet countsA measure of how many platelets are in the blood, usually expressed in thousands per microliter (e.g., 50,000) or in 109 per liter (e.g., 50 x 109/L). A count of 50 x 109/L is equal to a count of 50,000 per microliter. by attempting to prevent the immune system from destroying plateletsSmall cells that are made in the bone marrow and circulate through the bloodstream. Whenever there is damage to a blood vessel, platelets stick to the area to stop or prevent bleeding. . Corticosteroids are often the first treatment a patient receives for ITP. They can reduce the risk of bleeding and bruising by preventing platelet counts from dropping. However, it is recommended that healthcare providers keep patients on corticosteroids only for short periods because patients often cannot tolerate the side effects.1,2
If corticosteroids and other drugs that suppress the immune system (called immunosuppressantsA drug that works by suppressing the immune system.) do not increase platelet counts enough to avoid symptoms, your healthcare provider may consider splenectomy (splen-ec-ta-me). A splenectomy is the surgical removal of the spleen, which is an organ that is part of the immune system. In ITP, patients produce antibodiesProteins made by the body's immune system to attack foreign cells. In ITP, antibodies attack the body's own platelets. against their platelets. Platelets with antibodies on them are removed from the blood by the spleen. After your spleen is removed, platelets marked with antibodies stay in the blood longer. Many adult patients may achieve sustained platelet counts with splenectomy but there may be an increased risk of having more serious infections.3-5
In recent years, it has become clear that there is more to ITP than platelet destruction. There is an additional process involved in ITP: the body doesn't produce enough platelets to make up for the healthy platelets that are destroyed.2 The diagram below shows this in more detail.
Platelet boosters are medicines that imitate thrombopoietin (TPO)The protein that stimulates the megakaryocytes in the bone marrow to produce platelets.. TPO is the protein that tells special cells in the bone marrowTissue inside the bones where blood cells are made. to produce platelets. By doing this, platelet boosters help increase platelet production. Unlike most treatments for ITP, platelet boosters do not work by interfering with the immune system.2
Nplate® is a platelet booster indicated to treat adult chronic ITP when other medicine to treat ITP is not the best choice or surgery to remove the spleen has not worked well enough.2
Nplate® is a man-made protein medicine used to treat low blood platelet counts in adults with chronic immune thrombocytopenia (ITP), when certain other medicines, or surgery to remove your spleen, have not worked well enough.
Nplate® is not for use in people with a precancerous condition called myelodysplastic syndrome (MDS) or low platelet count caused by any condition other than chronic (lasting a long time) immune thrombocytopenia (ITP). Nplate® is only used if your low platelet count and medical condition increase your risk of bleeding. Nplate® is used to try to keep your platelet count about 50,000 per microliter in order to lower the risk for bleeding. Nplate® is not used to make your platelet count normal. It is not known if Nplate® works or if it is safe in people under the age of 18.
Important Safety Information
What is the most important information I should know about Nplate®?
Nplate® can cause serious side effects:
Worsening of a precancerous blood condition to a blood cancer (leukemia): Nplate® is not for use in people with a precancerous condition called myelodysplastic syndromes (MDS) or for any condition other than chronic (lasting a long time) immune thrombocytopenia (ITP). If you have MDS and receive Nplate®, your MDS condition may worsen and become an acute leukemia. If MDS worsens to become acute leukemia you may die sooner from the acute leukemia.
Higher risk for blood clots:
- You may have a higher risk of getting a blood clot if your platelet count becomes high during treatment with Nplate®. You may have severe complications or die from some forms of blood clots, such as clots that spread to the lungs or that cause heart attacks or strokes. Your healthcare provider will check your blood platelet counts and change your dose or stop Nplate® if your platelet counts get high.
- If you have a chronic liver disease, you may get blood clots in the veins of your liver. This may affect your liver function.
Loss of response: If you do not experience results from Nplate®, your immune system may have created a response that is counteractive to Nplate®. Your healthcare provider will monitor your platelet counts and test your blood regularly to determine if this is an issue.
Blood test monitoring: Your healthcare provider will check your platelet count every week and change your dose of Nplate® as needed. This will continue until your healthcare provider decides that your dose of Nplate® can stay the same. After that, you will need to have blood tests every month. When you stop receiving Nplate®, you will need blood tests for at least 2 weeks to check if your platelet count drops too low.
What are the possible side effects of Nplate®?
- Nplate® may cause serious side effects. See "What is the most important information I should know about Nplate®?"
- The most common side effects of Nplate® are:
- Joint pain
- Trouble sleeping
- Muscle tenderness or weakness
- Pain in arms and legs
- Abdominal pain
- Shoulder pain
- Tingling or numbness in hands and feet
- People who take Nplate® may have an increased risk of developing new or worsening changes in the bone marrow called "increased reticulin". These changes may improve if you stop taking Nplate®. Your healthcare provider may need to check your bone marrow for this problem during treatment with Nplate®.
- These are not all the possible side effects of Nplate®. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. For more information, ask your healthcare provider or pharmacist.
- If you have any questions about this information, be sure to discuss them with your doctor. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see Prescribing Information and Medication Guide for more information about Nplate®.
- Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002;346:995-1008.
- Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115:168-186.
- Neunert C, Lim W, Crowther M, et al. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011;117:4190-4207.
- Kojouri K, Vesely SK, Terrell DR, George JN. Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess long-term platelet count responses, prediction of response, and surgical complications. Blood. 2004;104:2623-2634.
- Cines DB, McMillan R. Management of adult idiopathic thrombocytopenic purpura. Annu Rev Med. 2005;56:425-442.
- Emmons RV, Reld DM, Cohen RL, et al. Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction. Blood. 1996;87:4068-4071.
- Nichol JL. Endogenous TPO (eTPO) levels in health and disease: possible clues for therapeutic intervention. Stem Cells. 1998;16(suppl 2):165-175.