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

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

Glucocorticoids are the first-line treatment for ITP, with second-line options including thrombopoietin receptor agonists (TPO-RAs) or splenectomy for patients who are corticosteroid-dependent or unresponsive to initial therapy. 1

Initial Treatment Approach

First-Line Therapy

• Glucocorticoids:
  • Adults: Prednisone 1-2 mg/kg/day 1
  • Children: Prednisone 4 mg/kg/day for 7 days followed by gradual reduction 1
  • Alternative: High-dose methylprednisolone (30 mg/kg/day for 3 days) for rapid response 1
  • Important limitation: Limit duration to 4-6 weeks maximum to avoid significant adverse effects 1

Treatment Goals

• Target platelet count of ≥30-50 × 10⁹/L 1
• Focus on achieving adequate hemostasis rather than normalizing platelet count 2
• Approximately 70% of patients achieve platelet response to initial treatment 1

Monitoring During Treatment

• Weekly platelet count monitoring during treatment initiation 1
• Monthly monitoring after establishing stable platelet counts 1
• Monitor for adverse effects of corticosteroids: weight gain, osteoporosis, cataracts, hypertension, infections 1

Second-Line Treatment Options

Thrombopoietin Receptor Agonists (TPO-RAs)

• Preferred second-line option for patients who are corticosteroid-dependent or unresponsive 1
• Options include:
  • Romiplostim: Weekly subcutaneous injection 3
  • Eltrombopag: Daily oral medication 1
• If no response to one TPO-RA, switching to another may be effective 1

Important Safety Considerations for TPO-RAs

• Risk of blood clots if platelet count becomes too high 3
• Regular monitoring of platelet counts required before, during, and after therapy 3
• Potential for liver complications in patients with chronic liver disease 3
• Not for use in patients with myelodysplastic syndrome (MDS) 3

Splenectomy

• Consider for eligible patients who prefer surgical treatment 1
• 60-70% long-term response rate 1
• 80% of responders maintain platelet response over 4 years post-procedure 1
• Potential complications include risk of infections and surgical complications 4

Other Second-Line Options

• Rituximab (anti-CD20): Immunosuppressive therapy recommended in international guidelines 2
• Intravenous immunoglobulin (IVIG): Useful for rapid platelet increase before planned procedures 4
• Anti-D immunoglobulin: For Rh-positive, non-splenectomized patients 5

Special Considerations

Pediatric Patients

• Should be managed by a hematologist experienced in pediatric ITP 1
• Cytotoxic drugs should be used with extreme caution 1
• Hospital admission recommended for:
  • Platelet counts <20,000/μL with mucosal bleeding
  • Severe or life-threatening bleeding 1

Indications for Hospital Admission

• Adult patients with platelet counts <20 × 10⁹/L or active bleeding 1
• Follow-up with a hematologist within 24-72 hours of diagnosis or disease relapse 1

Platelet Transfusions

• Reserved for:
  • Active bleeding with thrombocytopenia
  • Very severe thrombocytopenia (<10,000/μL) with high bleeding risk 1

Initial Diagnostic Workup

• Complete blood count with peripheral smear examination
• Coagulation profile, liver function tests, renal function tests
• Reticulocyte count
• HIV serology, hepatitis B and C serology (mandatory regardless of risk factors)
• H. pylori testing
• Blood type and Rh(D) typing 1

Treatment Algorithm

1. Assess bleeding risk:

   • If platelet count <30 × 10⁹/L or active bleeding → initiate treatment
   • If platelet count >30 × 10⁹/L without bleeding → consider observation

2. First-line therapy:

   • Start glucocorticoids (prednisone 1-2 mg/kg/day)
   • Limit duration to 4-6 weeks maximum

3. Evaluate response after 4-6 weeks:

   • If adequate response → taper and discontinue
   • If inadequate response or relapse → proceed to second-line therapy

4. Second-line options:

   • TPO-RAs (romiplostim or eltrombopag) OR
   • Splenectomy (for eligible patients who prefer surgical approach) OR
   • Rituximab

5. For refractory cases:

   • Try alternative TPO-RA if first one fails
   • Consider combination therapies
   • Enroll in clinical trials for newer agents

The decision between treatment options should be based on bleeding severity, bleeding risk, activity level, potential side effects, and patient preferences, with the ultimate goal of achieving a platelet count associated with adequate hemostasis 2.