What are the treatment options for Immune thrombocytopenic purpura (ITP)?

From the Guidelines

The treatment of Immune thrombocytopenic purpura (ITP) should prioritize thrombopoietin receptor agonists (TPO-RAs) as second-line therapy due to their high efficacy and improved quality of life, as supported by the most recent study 1.

First-line Treatment

For newly diagnosed ITP with significant bleeding or platelet counts below 30,000/μL, first-line therapy includes:

• Corticosteroids (prednisone 1 mg/kg/day for 2-4 weeks with gradual taper)
• Intravenous immunoglobulin (IVIG 1 g/kg/day for 1-2 days) Patients with severe bleeding may require platelet transfusions alongside these treatments.

Second-line Treatment

For persistent or chronic ITP, second-line options include:

• Thrombopoietin receptor agonists (eltrombopag 25-75 mg daily or romiplostim 1-10 μg/kg weekly)
• Rituximab (375 mg/m² weekly for 4 weeks)
• Splenectomy Other immunosuppressants like azathioprine (1-2 mg/kg/day), mycophenolate mofetil (500-1000 mg twice daily), or cyclosporine may be used in refractory cases.

Key Considerations

• The treatment approach targets the underlying autoimmune process where antibodies destroy platelets and impair their production.
• Patients should avoid medications that affect platelet function (NSAIDs, aspirin) and activities with high bleeding risk.
• Regular monitoring of platelet counts is essential to assess treatment response and guide therapy adjustments, as highlighted in 1 and 1.
• TPO-RAs have been shown to improve quality of life and reduce bleeding events, making them a preferred second-line option, as noted in 1 and 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), a prescription medicine 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.
• Surgery to remove the spleen, which may be considered when other treatments have not worked well enough.
• Certain medicines, which may be used to treat ITP, although the specific medicines are not listed in the provided text 2.

From the Research

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

• First-line treatments for ITP include:
  • Corticosteroids, such as prednisone or dexamethasone 3
  • Intravenous immunoglobulins (IVIg) for severe forms 3
  • Anti-RhD immune globulin 4
• Second-line treatments for ITP include:
  • Splenectomy, which can increase platelet counts in approximately two thirds of patients, but may have significant morbidities and life-threatening sequelae 5, 6
  • Thrombopoietin-receptor agonists (TPO-RAs), such as eltrombopag, which have been shown to be effective and safe in clinical practice 6
  • Rituximab, an immunosuppressive agent 6, 4
• Third-line treatments for ITP include:
  • Immunosuppressive drugs, such as azathioprine, ciclosporin, and cyclophosphamide 6, 7
  • Fostamatinib, a newer treatment option 4
• Other treatment options for ITP include:
  • Danazol, a synthetic androgen 7
  • Colchicine, an anti-inflammatory agent 7
  • High-dose immunoglobulin G 7
  • Anti-FcRn monoclonal antibodies and recombinant FcγR, which are currently in development 3