What are the treatment options for thrombocytopenia?

Treatment Options for Thrombocytopenia

The treatment of thrombocytopenia should be tailored to the underlying cause, with corticosteroids as first-line therapy for immune thrombocytopenia (ITP), followed by splenectomy, thrombopoietin receptor agonists, or rituximab for refractory cases. 1, 2

Initial Evaluation and Diagnosis

• Confirm true thrombocytopenia (exclude pseudothrombocytopenia)
• Essential testing:
  • Complete blood count with peripheral blood smear examination
  • Testing for HCV and HIV (grade 1B) 1
  • Consider bone marrow examination if abnormalities other than thrombocytopenia are present in blood count or smear 1
  • Note: Bone marrow examination is NOT necessary for patients presenting with typical ITP (grade 2C) 1, 2

Treatment Algorithm Based on Cause

1. Immune Thrombocytopenia (ITP)

First-line Treatment:

• Corticosteroids (grade 2B) 1, 2
  • Preferred: Longer courses of prednisone 1 mg/kg orally daily for 21 days followed by taper
  • Alternative: Dexamethasone 40 mg daily for 4 days (may repeat for 1-4 cycles every 2-4 weeks) 2
• Intravenous Immunoglobulin (IVIg) 1, 2
  • When rapid increase in platelet count is required
  • Initial dose: 1 g/kg as one-time dose (may be repeated if necessary) (grade 2B)
  • Can be used with corticosteroids for faster response
• Anti-D immunoglobulin (in appropriate patients) if corticosteroids are contraindicated (grade 2C) 1

Second-line Treatment (for those failing corticosteroids):

• Splenectomy (grade 1B) 1
  • Both laparoscopic and open splenectomy offer similar efficacy (grade 1C)
  • Should be delayed for at least 12 months unless severe disease is present (grade 2C)
• Thrombopoietin receptor agonists (e.g., romiplostim) 1, 3
  • For patients who relapse after splenectomy or have contraindications to splenectomy (grade 1B)
  • May be considered for patients who have failed one line of therapy without splenectomy (grade 2C)
  • Dosing: Initial dose 1 mcg/kg weekly SC, adjust to maintain platelet count ≥50 × 10⁹/L
  • Maximum dose: 10 mcg/kg weekly
• Rituximab 1, 2
  • May be considered for patients who have failed one line of therapy (grade 2C)
  • Response rate approximately 80% 2

2. Drug-Induced Thrombocytopenia

• Discontinue the suspected causative drug 4, 5
• Common culprits: cinchona alkaloids (quinine, quinidine), sulfonamides, NSAIDs, anticonvulsants, disease-modifying antirheumatic drugs, and diuretics 5
• For severe cases with bleeding: platelet transfusion may be necessary 5

3. HCV-Associated Thrombocytopenia

• Consider antiviral therapy in the absence of contraindications (grade 2C) 1
• Monitor platelet count closely due to risk of worsening thrombocytopenia with interferon
• If ITP treatment is required, initial treatment should be IVIg (grade 2C) 1

4. HIV-Associated Thrombocytopenia

• Treat underlying HIV infection 1
• Consider ITP-directed therapies as needed

5. Pregnancy-Associated ITP

• Treat with corticosteroids or IVIg (grade 1C) 1
• Mode of delivery should be based on obstetric indications (grade 2C) 1

Platelet Transfusion Guidelines

• Consider for patients with active bleeding and platelet count <20,000/mm³ 2
• Consider before procedures:
  • Central venous catheter placement (if platelets <20 × 10⁹/L)
  • Lumbar puncture (if platelets <50 × 10⁹/L) 2
• Not recommended for asymptomatic patients after splenectomy with platelet counts >30 × 10⁹/L (grade 1C) 1

Monitoring and Follow-up

• During dose adjustment phase: Weekly complete blood counts including platelet counts
• After stable dose established: Monthly complete blood counts
• After discontinuation of treatment: Weekly complete blood counts for at least 2 weeks 3
• Treatment response definitions:
  • Adequate response: Platelet count ≥50 × 10⁹/L
  • Complete response: Platelet count ≥100 × 10⁹/L 2

Important Caveats

• Thrombopoietin receptor agonists are not indicated for thrombocytopenia due to myelodysplastic syndrome or causes other than ITP 3
• Use the lowest dose of thrombopoietin receptor agonists to achieve and maintain platelet count ≥50 × 10⁹/L 3
• Discontinue thrombopoietin receptor agonists if platelet count doesn't increase sufficiently to avoid clinically important bleeding after 4 weeks at maximum dose 3
• Patients with low platelet counts should avoid activities with high risk of trauma and avoid antiplatelet medications and NSAIDs 2