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

Treatment of Immune Thrombocytopenic Purpura (ITP)

Corticosteroids are the first-line treatment for symptomatic ITP, with IVIG added when rapid platelet increase is required, followed by splenectomy for corticosteroid-refractory cases, and thrombopoietin receptor agonists reserved for patients who fail splenectomy or have contraindications to it. 1, 2

First-Line Treatment

Initial Therapy Selection

• Corticosteroids (prednisone or dexamethasone) should be initiated for symptomatic patients requiring treatment 1, 2
• Treatment is indicated when platelet counts are <20-30 × 10⁹/L or when counts are <50 × 10⁹/L with substantial mucous membrane bleeding 3
• The goal is to maintain platelet counts around 50,000 per microliter to reduce bleeding risk, not to normalize counts 4

When to Add IVIG

• IVIG (1 g/kg as a one-time dose) should be added when rapid platelet increase is required (grade 2B) 1, 2
• IVIG is particularly useful before planned surgical procedures or in emergency settings with active bleeding 1
• For emergency treatment with uncontrolled bleeding, combine prednisone and IVIG together 1
• IVIG typically increases platelets within 24 hours at the 1 g/kg dose 1

Important Caveats for First-Line Therapy

• Corticosteroids should be used for the shortest duration possible to minimize long-term morbidity 5
• Anti-D immunoglobulin can be used as an alternative in Rh-positive, non-splenectomized patients 1
• Treatment should be based on bleeding symptoms, not platelet count alone 6, 3

Second-Line Treatment

Splenectomy

• Splenectomy is recommended for patients who have failed corticosteroid therapy (grade 1B) 1, 2
• Provides long-term responses in 60-70% of patients 2
• Both laparoscopic and open splenectomy offer similar efficacy for medically suitable patients (grade 1C) 1
• Vaccination should be completed prior to splenectomy 1

Thrombopoietin Receptor Agonists (TPO-RAs)

• TPO-RAs (romiplostim, eltrombopag) are recommended for patients at risk of bleeding who relapse after splenectomy or have contraindications to splenectomy and have failed at least one other therapy (grade 1B) 1, 2
• TPO-RAs may be considered earlier for patients at risk of bleeding who have failed one line of therapy (such as corticosteroids or IVIG) and have not had splenectomy (grade 2C) 1
• Romiplostim demonstrated 61% durable platelet response in non-splenectomized patients and 38% in splenectomized patients 4

Rituximab

• Rituximab may be considered for patients at risk of bleeding who have failed one line of therapy such as corticosteroids, IVIG, or splenectomy (grade 2C) 1
• Response rate approximately 60%, with complete response in 40% of patients 1
• Response typically occurs within 1-8 weeks 1

Additional Second-Line Options

When splenectomy, TPO-RAs, and rituximab are not suitable, other immunosuppressive agents can be considered 1:

• Azathioprine (1-2 mg/kg): Response in up to two-thirds of patients, but requires 3-6 months 1
• Cyclosporin A (5 mg/kg/day initially): 50-80% response rate in 3-4 weeks 1
• Mycophenolate mofetil (1000 mg twice daily): Up to 75% response in 4-6 weeks 1
• Dapsone (75-100 mg): Response in up to 50% of patients within 3 weeks 1

Management of Secondary ITP

Screen for Underlying Causes

• Testing for HCV and HIV is recommended for all patients with suspected ITP (grade 1B) 2
• Screen for H. pylori in patients where eradication therapy would be used if positive (grade 2C) 1, 2

Treatment of Secondary ITP

• For HCV-associated ITP: Consider antiviral therapy in the absence of contraindications (grade 2C), with close platelet monitoring due to interferon-related thrombocytopenia risk 1, 2
• For HIV-associated ITP: Treat HIV infection with antiviral therapy before other options unless clinically significant bleeding is present (grade 1A) 1, 2
• For H. pylori-positive patients: Administer eradication therapy (grade 1B) 1, 2

Special Populations

Pregnancy

• Pregnant patients requiring treatment should receive either corticosteroids or IVIG (grade 1C) 1, 2
• Mode of delivery should be based on obstetric indications, not platelet count (grade 2C) 1, 2

Post-Splenectomy Management

• No further treatment is recommended for asymptomatic patients after splenectomy with platelet counts >30 × 10⁹/L (grade 1C) 1

Critical Pitfalls to Avoid

• Do not treat based solely on platelet count—bleeding risk and symptoms should guide therapy 6, 3
• Avoid prolonged corticosteroid use due to significant long-term morbidity 7, 5
• Monitor for thrombosis risk with TPO-RAs, especially when platelet counts become elevated 4
• Do not use TPO-RAs in patients with myelodysplastic syndrome (MDS) 4
• Bone marrow examination is not necessary for patients presenting with typical ITP (grade 2C) 2