What are the treatment options for Immune Thrombocytopenic Purpura (ITP)?

From the Guidelines

Treatment for immune thrombocytopenia (ITP) should prioritize thrombopoietin receptor agonists (TPO-RAs) as second-line therapy due to their high efficacy and favorable safety profile, as evidenced by recent studies 1. The management of ITP involves a stepwise approach, starting with first-line treatments such as corticosteroids, which can rapidly increase platelet counts but may have significant side effects with prolonged use 1.

First-line Treatment Options

• Corticosteroids like prednisone (1 mg/kg/day for 2-4 weeks, followed by a gradual taper) or dexamethasone (40 mg daily for 4 days) are commonly used as first-line therapy 1.
• Intravenous immunoglobulin (IVIG) at 1 g/kg for 1-2 days may be given for severe bleeding or very low platelet counts (<10,000/μL) 1.

Second-line Treatment Options

• TPO-RAs, such as eltrombopag (25-75 mg daily) or romiplostim (1-10 μg/kg weekly), are recommended as second-line therapy due to their high response rates (70-80%) and favorable safety profile 1.
• Rituximab (375 mg/m² weekly for 4 weeks) may be considered for patients who are refractory to first-line therapy or have a short history of ITP 1.
• Splenectomy may be considered for patients who are refractory to medical therapy, but it is associated with a higher risk of complications and relapse 1.

Refractory Cases

• Immunossuppressants like azathioprine, mycophenolate mofetil, or cyclosporine may be used in refractory cases, but their use should be carefully considered due to their potential side effects 1. The goal of treatment is to increase platelet production, decrease platelet destruction, and minimize bleeding events while maintaining an acceptable quality of life 1. Patients should avoid activities with high bleeding risk and medications that affect platelet function like aspirin and NSAIDs during treatment.

From the FDA Drug Label

Nplate is a prescription medicine used to treat low blood platelet counts (thrombocytopenia) in: adults with immune thrombocytopenia (ITP) when certain medicines or surgery to remove your spleen have not worked well enough children 1 year of age and older with ITP for at least 6 months when certain medicines or surgery to remove your spleen have not worked well enough.

The treatment options for Immune Thrombocytopenic Purpura (ITP) include Nplate (romiplostim), which is used to treat low blood platelet counts in adults and children 1 year of age and older with ITP when certain medicines or surgery to remove the spleen have not worked well enough 2.

• Key points:
  • Nplate is used to treat ITP in adults and children 1 year of age and older.
  • It is used when certain medicines or surgery to remove the spleen have not worked well enough.
  • The goal of Nplate treatment is to keep the platelet count about 50,000 per microliter to lower the risk for bleeding.
• Other treatment options are not explicitly mentioned in the provided drug labels, but it is mentioned that patients may have received other treatments prior to Nplate, including:
  • Corticosteroids
  • Immunoglobulins
  • Rituximab
  • Cytotoxic therapies
  • Danazol
  • Azathioprine
  • Surgery to remove the spleen (splenectomy) 2 2

From the Research

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

• The primary goal of treatment for ITP is to prevent serious bleeding, with treatment initiation recommended when the thrombocyte count has dropped below 20-30 × 10^9/l, or when hemorrhagic manifestations occur 3.
• First-line treatment options include:
  • Corticosteroids, such as prednisone, which can increase platelet counts 4, 5.
  • Intravenous immunoglobulin (IVIG) or anti-D immunoglobulin, which can also increase platelet counts and are particularly useful for stimulating rapid platelet increases before planned procedures 4, 6, 3.
• Second-line treatment options include:
  • Splenectomy, which can produce a long-lasting response in a majority of patients, but is an invasive procedure with potential risks 4, 7, 6, 3.
  • Thrombopoietin-receptor agonists (TPO-RAs), such as eltrombopag, which have been shown to be effective and safe as second-line treatment 7.
  • Rituximab, an immunosuppressive therapy that can be used in patients who do not respond to first-line treatment 6, 3.
  • Fostamatinib, a new drug in the treatment of ITP 3.
• Third-line treatment options include a mix of immunosuppressive drugs, such as azathioprine and ciclosporin 7.

Response to Treatment

• The overall response rate to eltrombopag as second-line treatment has been reported to be 94%, with a complete response rate of 76% and a partial response rate of 23% 7.
• The sustained response at 6 months has been reported to be lower in patients treated with high-dose dexamethasone compared to those treated with conventional prednisone 5.
• The presence of anti-GPIb-IX autoantibodies has been identified as a predictive factor for a poor initial response to corticosteroids therapy 5.