What is the treatment approach for thrombocytopenia?

Treatment Approach for Thrombocytopenia

The treatment of thrombocytopenia must be tailored to the underlying cause, with corticosteroids as first-line therapy for immune thrombocytopenia (ITP), followed by thrombopoietin receptor agonists for those who fail initial therapy. 1

Diagnosis and Initial Assessment

Before initiating treatment, it's essential to:

• Determine the underlying cause through:

  • Complete blood count with peripheral blood smear
  • Testing for Hepatitis C and HIV (for suspected ITP)
  • Evaluation for secondary causes (antiphospholipid syndrome, drug-induced thrombocytopenia, infections)

• Assess bleeding risk based on:

  • Platelet count
  • Presence of active bleeding
  • Comorbidities that increase bleeding risk
  • Need for procedures or surgery

Treatment Indications

Treatment is generally indicated in the following scenarios:

• Platelet count <30 × 10⁹/L
• Active bleeding
• High risk of bleeding due to comorbidities
• Need for procedures/surgery
• Occupation or lifestyle with high bleeding risk 1

No treatment is typically needed for:

• Asymptomatic patients with platelet counts >50 × 10⁹/L
• Asymptomatic post-splenectomy patients with counts >30 × 10⁹/L 1

Treatment Algorithm for Immune Thrombocytopenia (ITP)

First-Line Treatment

1. Corticosteroids:

   • Prednisone: 0.5-2 mg/kg/day until platelet count increases to 30-50 × 10⁹/L
   • Dexamethasone: 40 mg/day for 4 days 2, 1
   • Taper rapidly to avoid complications, especially in non-responders after 4 weeks 2

2. Intravenous Immunoglobulin (IVIg):

   • Used with corticosteroids when rapid platelet increase is needed
   • Initial dose: 1 g/kg as one-time dose (may be repeated if necessary)
   • First-line option if corticosteroids are contraindicated 1

Second-Line Treatment

1. Thrombopoietin Receptor Agonists (TPO-RAs):

   • Recommended for patients who relapse after splenectomy or have contraindications to splenectomy 1

   • May be considered after failure of first-line therapy 1

   • Romiplostim:

     • Initial dose: 1 mcg/kg subcutaneously once weekly
     • Adjust dose by increments of 1 mcg/kg to achieve platelet count ≥50 × 10⁹/L
     • Maximum dose: 10 mcg/kg weekly
     • Most patients respond with 2-3 mcg/kg 3

   • Eltrombopag:

     • Initial dose: 36 mg orally once daily (18 mg for East/Southeast Asian patients or those with hepatic impairment)
     • Adjust based on platelet count response 4

2. Splenectomy:

   • Recommended for patients who have failed corticosteroid therapy
   • High initial response rate (85%) but up to 30% relapse within 10 years
   • Both laparoscopic and open approaches offer similar efficacy 1

3. Other Immunosuppressive Agents:

   • Rituximab: 60% response rate with 40% achieving complete response 2
   • Azathioprine: Complete responses in 45% of patients treated with 150 mg/day 2
   • Cyclosporin A: Clinical improvement in >80% of patients resistant to first-line therapy 2
   • Mycophenolate mofetil: Response rate of 78% in retrospective studies 2
   • Dapsone: Can delay splenectomy for up to 32 months in patients not responding to corticosteroids 2

Treatment for Cancer-Associated Thrombocytopenia

For patients with cancer-associated thrombosis (CAT) and thrombocytopenia:

1. Platelet count >50 × 10⁹/L: Full-dose anticoagulation is safe 2

2. Platelet count <50 × 10⁹/L:

   • High-risk features (symptomatic PE, proximal DVT, history of recurrent/progressive thrombosis):

     • Therapeutic anticoagulation with platelet transfusion support to maintain counts above 40-50 × 10⁹/L 2

   • Lower-risk events (distal DVT, incidental subsegmental PE, catheter-related thrombosis):

     • Dose-modified anticoagulation (50% or prophylactic-dose LMWH)
     • Withhold anticoagulation if platelet count <25 × 10⁹/L 2

Emergency Management

For life-threatening bleeding:

• IVIg has the most rapid onset of action
• Consider platelet transfusions (effects may be short-lived)
• Consider recombinant factor VIIa in severe bleeding 1

Monitoring

• During dose adjustment of TPO-RAs: Weekly complete blood counts
• After stable dose: Monthly complete blood counts
• For patients on corticosteroids: Monitor for side effects including hyperglycemia, hypertension, and mood changes

Special Considerations

• Pregnancy: Treat with corticosteroids or IVIg; delivery mode based on obstetric indications 1
• HCV-associated ITP: Consider antiviral therapy if no contraindications 1
• HIV-associated ITP: Treat HIV infection with antivirals before other treatments unless significant bleeding 1
• H. pylori-associated ITP: Screen and administer eradication therapy if positive 1

The goal of treatment is to achieve a safe platelet count to prevent bleeding, not to normalize platelet counts. Treatment decisions should be guided by bleeding risk rather than absolute platelet counts alone 5, 6.