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

From the Guidelines

Immune thrombocytopenia (ITP) treatment typically begins with corticosteroids like prednisone (1mg/kg/day for 2-4 weeks) as first-line therapy, as recommended by the American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia 1. For patients requiring rapid platelet increase, intravenous immunoglobulin (IVIG) at 1g/kg for 1-2 days can be used alongside steroids, as suggested by the 2011 guideline 1. If patients don't respond or relapse after initial treatment, second-line options include:

• Thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag (25-75mg daily) or romiplostim (1-10μg/kg weekly), which have been shown to be highly effective in clinical trials, with initial responses observed already after 1–2 weeks of treatment, and significantly reducing bleeding events and emergency hospitalizations 1.
• Rituximab (375mg/m² weekly for 4 weeks), which has been demonstrated to trigger short-term responses in 50–60% of patients, with long-term responses documented in 20–30% of cases 1.
• Splenectomy, which is associated with initial response in 85% of cases, although durable responses are documented in about 60–65% of patients, and is associated with an increased chance of relapse in 20–30% of responding patients 1. For refractory cases, immunosuppressants like mycophenolate mofetil or azathioprine may be considered. Treatment decisions should be individualized based on bleeding risk, platelet count, comorbidities, and patient preferences, as emphasized by the 2011 guideline 1. The goal is to maintain platelet counts above 30,000/μL to prevent serious bleeding rather than normalizing counts, as suggested by observational data of ITP patient cohorts 1. ITP treatment works by either reducing platelet destruction (steroids, IVIG, rituximab, splenectomy) or increasing platelet production (TPO-RAs), addressing the autoimmune nature of the disease where antibodies target platelets for premature destruction. Key considerations in treatment selection include the potential side effects of each option, such as the risk of thrombotic events with TPO-RAs, and the impact on quality of life, as noted in the 2020 review of tapering and discontinuation of thrombopoietin receptor agonists in immune thrombocytopenia 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 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 that may be used before Nplate include:
  • 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)

• First-line treatment options for ITP include:
  • Corticosteroids, which have been the standard first-line treatment for symptomatic patients 3
  • Intravenous immune globulin (IVIG) or Rho(D) immune globulin (anti-RhD) for steroid-resistant cases 3, 4, 5
• Second-line treatment options for ITP include:
  • Splenectomy, which has been shown to be effective in approximately two thirds of patients, although thrombocytopenia often recurs with longer follow-up 4
  • Rituximab, a monoclonal antibody against the CD20 antigen (anti-CD20), which has been shown to induce sustained remissions with minimal toxicity in patients with chronic ITP 3, 5
• Third-line treatment options for ITP include:
  • Thrombopoietic agents, which aim to increase platelet production 6
  • Fostamatinib, which is currently under clinical evaluation 6
  • Other immunosuppressive agents, such as danazol, azathioprine, cyclophosphamide, vinca alkaloids, and cyclosporin A, although evidence of their efficacy is limited 5

Special Considerations

• High-dose dexamethasone has been shown to be effective as a first- and second-line treatment of ITP in adults, with excellent acute effects and good long-term effects as a first-line therapy 7
• The treatment strategy should aim at elevating the platelet counts to a safety level with minimal toxicity and improving patient health-related quality of life, and should be tailored to the individual patient and disease phase 6