How to treat thrombocytopenia?

Treatment of Thrombocytopenia

Treatment of thrombocytopenia should be tailored to the underlying cause, with corticosteroids as first-line therapy for immune thrombocytopenia (ITP), the most common form requiring intervention. 1

Determining When to Treat

• Treatment is indicated for:

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

• The goal is to achieve a safe platelet count (>30 × 10⁹/L) to prevent bleeding, not to normalize platelet counts 1

First-Line Treatment Options

Corticosteroids

• Prednisone: 0.5-2 mg/kg/day until platelet count increases to 30-50 × 10⁹/L 2, 1

  • Should be rapidly tapered and stopped in responders, and especially in non-responders after 4 weeks to avoid complications 2

• Dexamethasone: 40 mg/day for 4 days (equivalent to 400 mg prednisone/day) 2

  • Can be given in cycles every 14 days for up to 4 cycles
  • Higher initial response rates (up to 90%) with sustained response in 50-80% of patients 2, 1

• Methylprednisolone: For patients failing first-line therapies

  • High-dose (30 mg/kg/day for 7 days) shows response rates up to 95% 2
  • Faster response (4.7 days vs 8.4 days) compared to prednisone 2

Intravenous Immunoglobulin (IVIg)

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

IV Anti-D

• Appropriate for Rh(D) positive, non-splenectomized ITP patients 2
• Should be avoided in patients with autoimmune hemolytic anemia 2
• Blood group, DAT, and reticulocyte count required before treatment 2

Second-Line Treatment Options

Thrombopoietin Receptor Agonists (TPO-RAs)

• Romiplostim (Nplate):

  • Initial dose: 1 mcg/kg subcutaneously once weekly 3
  • Adjust dose to maintain platelet count ≥50 × 10⁹/L
  • Maximum dose: 10 mcg/kg weekly 3
  • Indicated for adults and children ≥1 year with ITP who had insufficient response to corticosteroids, immunoglobulins, or splenectomy 3

• Eltrombopag (ALVAIZ):

  • Initial dose: 36 mg orally once daily (18 mg for East/Southeast Asian patients or those with hepatic impairment) 4
  • Indicated for adults and children ≥6 years with persistent or chronic ITP who had insufficient response to corticosteroids, immunoglobulins, or splenectomy 4

Other Second-Line Options

• Rituximab: 60% response rate with 40% achieving complete response 1
• Azathioprine: Complete responses in 45% of patients treated with 150 mg/day 1
• Cyclosporin A: Clinical improvement in >80% of patients resistant to first-line therapy 1
• Mycophenolate mofetil: Response rate of 78% in retrospective studies 1
• Dapsone: Can delay splenectomy for up to 32 months in patients not responding to corticosteroids 1

Splenectomy

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

Special Considerations

Emergency Management (Life-threatening Bleeding)

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

Secondary Causes of Thrombocytopenia

• HCV-associated ITP: Consider antiviral therapy if no contraindications 1
• HIV-associated ITP: Treat HIV infection with antiretrovirals before other treatments unless significant bleeding 1
• H. pylori-associated ITP: Screen for H. pylori and administer eradication therapy if positive 1

Monitoring

• Weekly complete blood counts during dose adjustment of TPO-RAs 1
• Monthly complete blood counts after stable dose is achieved 1
• Monitor for corticosteroid side effects (hyperglycemia, hypertension, mood changes) 1

Common Pitfalls to Avoid

• Don't attempt to normalize platelet counts - aim for safe levels (>30 × 10⁹/L) 1, 3, 4
• Don't continue corticosteroids beyond 4 weeks in non-responders 2
• Don't use TPO-RAs for thrombocytopenia due to myelodysplastic syndrome 3, 4
• Don't forget to screen for underlying causes (HIV, HCV, H. pylori) before diagnosing primary ITP 1
• Don't use IV anti-D in patients with autoimmune hemolytic anemia 2

By following this treatment algorithm and considering the specific characteristics of each patient's condition, clinicians can effectively manage thrombocytopenia while minimizing risks and complications.