Idiopathic thrombocytopenic purpura (ITP) Treatments and drugs

The goal of treating ITP is to ensure a safe platelet count and prevent bleeding complications while minimizing treatment side effects.
In children, idiopathic thrombocytopenic purpura usually runs its course without the need for treatment. About 80 percent of children with idiopathic thrombocytopenic purpura recover completely within six months. Even in children who develop chronic ITP, complete recovery may still occur, even years later.
Adults with mild cases of ITP may require nothing more than regular monitoring and platelet checks. But if your symptoms are troublesome and your platelet count remains low, you and your doctor may opt for treatment. Treatment usually consists of medications and sometimes surgery (splenectomy). Your doctor may also have you discontinue certain drugs that can further inhibit platelet function, such as aspirin, ibuprofen (Advil, Motrin, others) and the blood-thinning medication warfarin (Coumadin).
Medications
Common medications used to treat idiopathic thrombocytopenic purpura include:

• Corticosteroids. The first line of therapy for ITP is a corticosteroid, usually prednisone, which can help raise your platelet count by decreasing the activity of your immune system. Once your platelet count is back to a safe level, you can gradually discontinue taking the drug under the direction of your doctor. In general, this takes about two to six weeks.
  The problem is that many adults experience a relapse after discontinuing corticosteroids. A new course of corticosteroids may be pursued, but long-term use of these medications isn't recommended because of the risk of serious side effects, including cataracts, high blood sugar, increased risk of infections and loss of calcium from your bones (osteoporosis). You and your doctor will want to weigh the benefits of the medication against these risks. If you've taken corticosteroids for longer than three months, your doctor will likely recommend that you take calcium and vitamin D supplements to help maintain your bone density.
• Intravenous immune globulin (IVIG). If you have critical bleeding or need to quickly increase your blood count before surgery, you may receive medications, such as immune globulin, given intravenously. These medications are quick and effective, but the effect usually wears off in a couple of weeks. Possible side effects include headache, nausea and vomiting.
• Thrombopoietin receptor agonists. The newest medications approved to treat ITP are romiplostim (Nplate) and eltrombopag (Promacta). These drugs help your bone marrow produce more platelets, which helps prevent bruising and bleeding. Possible side effects include headache, joint or muscle pain, dizziness, nausea or vomiting, and an increased risk of blood clots.

Surgery
If you have severe ITP and an initial course of prednisone hasn't helped, surgical removal of your spleen (splenectomy) may be an option. This eliminates the main source of platelet destruction in your body and improves your platelet count within a few weeks. Splenectomy for ITP is not as routinely performed as it once was, however. Serious post-surgical complications sometimes occur, and not having a spleen permanently increases your susceptibility to infection. What's more, some people relapse even after splenectomy.
Splenectomy is rarely performed in children because of their high rate of spontaneous remission.
Emergency treatment
Although rare, severe bleeding can occur with ITP, regardless of age or platelet count. Severe or widespread bleeding is life-threatening and demands emergency care. This usually includes transfusions of platelet concentrates, intravenous methylprednisolone (a type of corticosteroid) and intravenous immune globulin.
Other treatments
If neither the initial round of corticosteroids nor a splenectomy has helped you achieve remission and your symptoms are severe, your doctor may recommend another course of corticosteroids, usually at the lowest effective dose.
Other possible treatments include:

• Immunosuppressant drugs. Medications that suppress the immune system, such as rituximab (Rituxan) — the most commonly used of this group — cyclophosphamide (Cytoxan) and azathioprine (Imuran, Azasan), have been used to treat ITP, but they can cause significant side effects, and their effectiveness has yet to be proved.
• Experimental drugs. New medications that increase platelet production, especially eltrombopag and AMG 531, are being studied in clinical trials. Although they appear to be well tolerated, questions about long-term side effects remain, and relapse is possible when the drugs are stopped.
• H. pylori treatment. Some people with ITP are also infected with Helicobacter pylori, the same bacteria that cause most peptic ulcers. Eliminating the bacteria has helped increase platelet count in some people, but the results for this therapy are inconsistent and need to be studied further.

Because of the potential complications of both the disease and its treatment, it's important for you and your doctor to carefully weigh the benefits and risks of treatment. For example, some people find that the side effects of treatment are more burdensome than the effects of the disease itself. Treatment decisions are usually based on:

• Severity of signs and symptoms (active bleeding is usually an indication for treatment)
• Platelet count — even relatively low counts (less than 30,000 platelets per microliter of blood) may not merit treatment, especially if you have no active bleeding and have a fairly sedentary lifestyle
• Your age and willingness to undergo treatment
• Risk of bleeding relative to lifestyle, such as participation in sports or other vigorous physical activities that may predispose you to injury
• Risk of bleeding based on other medical conditions (high blood pressure, infections, alcoholism, chronic liver disease, peptic ulcer) or needed medications, such as aspirin
• Potential side effects of ITP therapies

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