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

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

The treatment of ITP should follow a stepwise approach, with corticosteroids as first-line therapy, followed by thrombopoietin receptor agonists (TPO-RAs) as the most effective second-line option, and splenectomy or rituximab as alternative second-line therapies based on patient-specific factors. 1, 2

First-Line Treatment

Initial Therapy

• Corticosteroids: Standard initial treatment for adults with ITP and platelet counts <30 × 10^9/L or with bleeding symptoms
  • Prednisone 1-2 mg/kg/day for adults
  • Children: Prednisone 4 mg/kg/day for 7 days followed by gradual reduction 2
  • Goal: Increase platelet count to reduce bleeding risk
  • Duration: Shortest possible course to minimize side effects 2

Emergency Treatment for Severe Bleeding

• Intravenous Immunoglobulin (IVIg): For patients with severe bleeding or requiring rapid platelet increase

  • Particularly useful before planned procedures
  • First-line for patients with end-stage renal disease (ESRD) and ITP 2

• Anti-D Immunoglobulin: Alternative for Rh(D)-positive, non-splenectomized patients 1

Second-Line Treatment Options

For Patients Who Fail Corticosteroid Therapy

1. Thrombopoietin Receptor Agonists (TPO-RAs):

   • Strong recommendation for patients who relapse after splenectomy or have contraindications to splenectomy (Grade 1B) 1
   • Examples: Romiplostim (Nplate), Eltrombopag
   • Efficacy: Stably increase platelet counts in 59-80% of patients 2
   • Monitoring: Weekly platelet count monitoring during initiation, then monthly once stable 2
   • Caution: Risk of blood clots if platelet count becomes too high 3

2. Splenectomy:

   • Strong recommendation for patients who have failed corticosteroid therapy (Grade 1B) 1
   • Only treatment providing sustained remission off all treatments in a high proportion of patients 2
   • Both laparoscopic and open approaches offer similar efficacy 1
   • Requires vaccination prior to procedure
   • Post-splenectomy: No further treatment needed if asymptomatic with platelet counts >30 × 10^9/L 1

3. Rituximab:

   • Consider for patients who have failed corticosteroids, IVIg, or splenectomy (Grade 2C) 1
   • Complete response rate: ~47% by 6 months 2
   • Limited long-term remission: Only ~21% maintain remission at 5 years 2
   • Adverse effects: Infusion reactions (20%), hypogammaglobulinemia, rare serious complications 2

Special Populations

Pregnancy

• Recommended treatments: Corticosteroids or IVIg (Grade 1C) 1
• Mode of delivery should be based on obstetric indications, not ITP status 1

Secondary ITP

1. HCV-associated ITP:

   • Consider antiviral therapy if no contraindications (Grade 2C)
   • Initial ITP treatment: IVIg 1

2. HIV-associated ITP:

   • First approach: Treat HIV with antivirals unless significant bleeding present (Grade 1A)
   • If ITP treatment needed: Corticosteroids, IVIg, or anti-D 1

3. H. pylori-associated ITP:

   • Screen for H. pylori in appropriate patients (Grade 2C)
   • Provide eradication therapy if positive (Grade 1B) 1

Treatment Algorithm

1. Initial Assessment:

   • Confirm ITP diagnosis (platelet count <100 × 10^9/L)
   • Evaluate bleeding risk
   • Screen for secondary causes (HIV, HCV, H. pylori)

2. Treatment Decision:

   • Treat if: Platelet count <20-30 × 10^9/L or bleeding symptoms
   • Observation if: Asymptomatic with platelet count >30 × 10^9/L

3. First-Line Therapy:

   • Corticosteroids (prednisone 1-2 mg/kg/day)
   • Add IVIg for severe bleeding or if rapid platelet increase needed

4. If No Response or Relapse:

   • TPO-RAs (preferred second-line therapy for most patients)
   • OR Splenectomy (for eligible patients preferring surgical approach)
   • OR Rituximab (alternative for patients unsuitable for above options)

5. Monitoring:

   • Weekly platelet counts during treatment initiation
   • Monthly counts after stabilization
   • Watch for bleeding complications and treatment-specific side effects

Common Pitfalls and Caveats

• Overtreating asymptomatic patients: Treatment should be based on bleeding risk, not just platelet count
• Prolonged corticosteroid use: Leads to significant morbidity; use for shortest duration possible
• Delaying second-line therapy: Consider TPO-RAs early for patients failing corticosteroids
• Inadequate monitoring: Regular platelet count monitoring is essential, especially when starting TPO-RAs
• Missing secondary causes: Always screen for underlying conditions (HIV, HCV, H. pylori)
• Blood clot risk with TPO-RAs: Monitor closely if platelet counts become elevated 3
• Post-splenectomy infection risk: Ensure appropriate vaccinations and patient education

By following this evidence-based approach, clinicians can effectively manage ITP while minimizing complications and improving patient quality of life.