What is the treatment for Immune Thrombocytopenic Purpura (ITP)?

From the Guidelines

The treatment for Immune Thrombocytopenic Purpura (ITP) should prioritize corticosteroids as first-line therapy, with a recommended duration of no longer than 6-8 weeks, followed by a switch to second-line treatments such as thrombopoietin receptor agonists (TPO-RAs) if necessary, as supported by the most recent evidence 1.

First-Line Treatment

• Corticosteroids, including prednisone and high-dose dexamethasone, are commonly administered in adult ITP patients as first-line therapy to rapidly achieve safe platelet counts and prevent bleeding events 1.
• Initial responsiveness to corticosteroids is observed in about 60-80% of patients, but sustained responses are seen only in 20-40% of cases, highlighting the importance of switching to second-line treatments if necessary 1.

Second-Line Treatment

• Second-line options include TPO-RAs like eltrombopag (25-75 mg daily) or romiplostim (1-10 μg/kg weekly), rituximab (375 mg/m² weekly for 4 weeks), or immunosuppressants like azathioprine or mycophenolate mofetil 1.
• TPO-RAs have been shown to be highly effective in clinical trials, with initial responses observed in 70-80% of patients and a median exposure time of 2 years, making them a preferred second-line option 1.

Additional Considerations

• Patients should avoid medications that affect platelet function (aspirin, NSAIDs) and activities with high bleeding risk.
• Treatment aims to maintain safe platelet counts while minimizing medication side effects, as ITP results from autoantibodies causing increased platelet destruction and impaired production.
• Splenectomy remains effective for refractory cases but is now less commonly used due to effective medical therapies 1.

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 for Immune Thrombocytopenic Purpura (ITP) is 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.

• Key points:
  • Nplate is given by injection under the skin one time each week.
  • The dose of Nplate is adjusted to maintain platelet counts between 50 × 10^9/L to 200 × 10^9/L.
  • Patients receiving Nplate should be closely monitored for changes in platelet count and for signs of bleeding.
  • Nplate may cause serious side effects, including blood clots and bone marrow changes. 2

From the Research

Treatment Options for ITP

The primary goal of treatment for Immune Thrombocytopenic Purpura (ITP) is to prevent serious bleeding, with treatment typically depending on symptomology rather than the platelet count itself 3. The treatment options can be categorized into first-line, second-line, and third-line therapies.

First-Line Therapies

• Corticosteroids have been the standard first-line treatment for symptomatic patients, aiming to increase platelet counts 4, 3, 5, 6.
• Intravenous immune globulin (IVIG) or Rho(D) immune globulin (anti-RhD) may be added for steroid-resistant cases 3.
• The use of corticosteroids is recommended for the shortest possible duration to minimize side effects 6.

Second-Line Therapies

• Splenectomy, which can produce a long-lasting response in a majority of patients, is considered for those who do not respond to steroid therapy 4, 5.
• Rituximab, a monoclonal antibody against the CD20 antigen (anti-CD20), is another option for second-line therapy 3.
• Thrombopoietin receptor agonists are often used as a second-line treatment, aiming to stimulate platelet production 5, 6.

Third-Line Therapies and Emerging Treatments

• For patients with chronic refractory ITP, third-line treatments are evaluated, including new emerging therapies that target various aspects of ITP pathophysiology 3, 7, 6.
• These new strategies may change the order of treatment lines and offer alternative options for patients who do not respond to traditional therapies 7, 6.

Considerations for Treatment

• Treatment should be tailored according to the patient's age, lifestyle, comorbidities, and compliance 7.
• The decision to treat ITP is based on the risk of bleeding rather than the platelet count itself, with treatment indicated for patients with significant bleeding symptoms or low platelet counts (<20 x 10^9-30 x 10^9/L) 5.