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

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

The treatment of ITP should follow a stepwise approach starting with corticosteroids or IVIG as first-line therapy, followed by thrombopoietin receptor agonists or splenectomy as second-line options, with consideration of underlying causes in secondary ITP. 1

Initial Diagnosis and Assessment

Before initiating treatment, proper diagnosis is essential:

• Testing for HCV and HIV is strongly recommended (grade 1B) 1
• Bone marrow examination is not necessary for patients with typical ITP presentation regardless of age (grade 2C) 1
• Further investigations should be performed if there are abnormalities in the blood count or smear beyond thrombocytopenia 1
• Consider screening for H. pylori in patients where eradication therapy would be used if positive (grade 2C) 1

First-Line Treatment Options

Corticosteroids

• Prednisone (standard initial therapy): 0.5-2 mg/kg/day until platelet count increases to 30-50 × 10^9/L 1
  • Should be rapidly tapered and stopped in responders within 4 weeks to avoid complications
  • Response rate: 70-80% initially, but sustained responses only in 20-40% 1

Alternative Corticosteroid Regimens

• Dexamethasone: 40 mg/day for 4 days (equivalent to 400 mg prednisone/day)
  • May be given in 1-4 cycles every 14 days
  • Higher initial response rates (up to 90%) with potentially better sustained response (50-80%) 1
• Methylprednisolone: High-dose parenteral administration for patients failing first-line therapies
  • Response rate: up to 80% but typically short-term 1

Intravenous Immunoglobulin (IVIG)

• Recommended when rapid increase in platelet count is required (grade 2B) 1
• Dosing: 1 g/kg as a one-time dose; may be repeated if necessary 1
• Can be used with corticosteroids for enhanced effect 1
• Response rate: up to 80% initially, typically within 24-48 hours 1

Anti-D Immunoglobulin

• For Rh(D) positive, non-splenectomized patients 1
• Alternative to IVIG if corticosteroids are contraindicated (grade 2C) 1
• Avoid in patients with autoimmune hemolytic anemia 1
• Requires blood group, DAT, and reticulocyte count before administration 1

Second-Line Treatment Options

Thrombopoietin Receptor Agonists (TPO-RAs)

• Recommended for patients who relapse after splenectomy or have contraindications to splenectomy and have failed at least one other therapy (grade 1B) 1
• May be considered for patients who have failed first-line therapy without splenectomy (grade 2C) 1
• Options include:
  • Romiplostim: Weekly subcutaneous injection, individualized dosing 2
  • Eltrombopag: Daily oral administration 1
• High response rates: durable platelet response in 38-61% of patients 2

Splenectomy

• Recommended for patients who have failed corticosteroid therapy (grade 1B) 1
• Both laparoscopic and open approaches offer similar efficacy (grade 1C) 1
• Response rate: 80% initially, with approximately two-thirds achieving lasting response 1
• No further treatment needed in asymptomatic patients after splenectomy with platelet counts >30 × 10^9/L (grade 1C) 1

Rituximab

• May be considered for patients who have failed corticosteroids, IVIG, or splenectomy (grade 2C) 1
• Response rate: 60% overall, with complete response in 40% of patients 1
• Standard dosing: 375 mg/m² weekly for 4 weeks (lower doses may also be effective) 1

Management of Secondary ITP

HCV-Associated ITP

• Consider antiviral therapy if no contraindications exist (grade 2C) 1
• Monitor platelet count closely due to risk of worsening thrombocytopenia with interferon 1
• Initial treatment should be IVIG if ITP treatment is required (grade 2C) 1

HIV-Associated ITP

• Treat HIV infection with antiretroviral therapy before other options unless significant bleeding is present (grade 1A) 1
• If ITP treatment is required, use corticosteroids, IVIG, or anti-D (grade 2C) 1
• Consider splenectomy for symptomatic patients who fail these therapies (grade 2C) 1

H. pylori-Associated ITP

• Administer eradication therapy for patients with confirmed H. pylori infection (grade 1B) 1

Emergency Treatment for Severe Bleeding

For patients with uncontrolled bleeding or at high risk:

• Combine prednisone with IVIG 1
• Consider high-dose methylprednisolone 1
• Other rapid-acting options include platelet transfusion (possibly with IVIG) and emergency splenectomy 1

Special Populations

Pregnancy

• Pregnant patients requiring treatment should receive corticosteroids or IVIG (grade 1C) 1
• Mode of delivery should be based on obstetric indications (grade 2C) 1

Treatment Algorithm

1. First-line therapy:

   • Corticosteroids (prednisone or dexamethasone) ± IVIG if rapid response needed
   • For contraindications to steroids: IVIG or anti-D (if Rh+)

2. If inadequate response or relapse:

   • TPO-RAs (romiplostim or eltrombopag) OR
   • Splenectomy (consider patient preference, comorbidities)
   • Rituximab as alternative

3. For refractory ITP:

   • Consider immunosuppressive agents: azathioprine, cyclosporine, cyclophosphamide, mycophenolate mofetil, or danazol 1

Common Pitfalls and Caveats

• Prolonged corticosteroid use leads to significant adverse effects; taper rapidly and limit to 6-8 weeks maximum 1
• TPO-RAs may increase risk of thrombosis if platelet counts become too high 2
• Splenectomy carries lifelong risk of infection and should be carefully considered
• Treatment goal is to achieve a safe platelet count to prevent bleeding, not necessarily a normal count 3
• Always rule out secondary causes of ITP before committing to long-term therapy