What are the first-line and subsequent treatment options for Immune Thrombocytopenic Purpura (ITP) in pediatric patients?

Pediatric ITP Treatment Guidelines

First-line treatment for pediatric ITP should include intravenous immunoglobulin (IVIg), IV anti-D immunoglobulin (for Rh-positive patients), or short-course corticosteroids, with the choice based on bleeding severity and platelet count. 1

Initial Assessment and Management Approach

When to Treat

• Treatment is necessary for all children with severe bleeding symptoms
• Treatment should be considered for children with:
  • Moderate bleeding
  • Platelet counts <10 × 10^9/L
  • Increased risk of bleeding

Watch and Wait Approach

• Approximately two-thirds of children will improve spontaneously within 6 months 1
• Many children stabilize with platelet counts of 20-30 × 10^9/L without symptoms unless injured
• This approach is appropriate for children without significant bleeding symptoms

First-Line Treatment Options

1. Intravenous Immunoglobulin (IVIg)

• Dosage: 0.8-1 g/kg as a single dose
• Efficacy: Raises platelet count in >80% of children
• Time to response: 1-2 days (more rapid than corticosteroids)
• Side effects: Headache (can be severe), fever, nausea/vomiting
• Duration: One-third of patients fall below acceptable platelet counts after 2-6 weeks 1

2. IV Anti-D Immunoglobulin

• Only for Rh(D)-positive children
• Dosage: 50-75 μg/kg as a short infusion
• Efficacy: 50-77% achieve platelet response
• Time to response: ≥50% respond within 24 hours
• Side effects: Headache, fever, chills (less common than with IVIg)
• Caution: Risk of mild extravascular hemolysis; rare cases of intravascular hemolysis, DIC, and renal failure reported in pediatric patients with comorbidities 1

3. Corticosteroids

• Prednisone:
  • Conventional dose: 1-2 mg/kg/day for maximum of 14 days
  • Higher dose: 4 mg/kg/day for 3-4 days
  • Efficacy: Up to 75% response rate, higher doses effective in 72-88% of children
  • Time to response: 2-7 days
  • Side effects: Transient mood changes, gastritis, weight gain
  • Caution: Use with care in presence of active infection (especially varicella) or GI bleeding 1

Emergency Treatment for Life-Threatening Bleeding

For organ- or life-threatening situations:

• Larger-than-usual dose (2-3 fold) of platelets
• IV high-dose corticosteroids
• IVIg or IV anti-D
• Consider emergency splenectomy in special circumstances 1

Treatment for Persistent or Chronic ITP (>6 months)

Goals

• Maintain hemostatic platelet count with first-line therapies
• Minimize prolonged corticosteroid therapy
• All children with persistent/chronic ITP should be managed by a hematologist experienced in pediatric ITP 1

Second-Line Treatment Options

1. High-Dose Methylprednisolone (HDMP)

   • 30 mg/kg/day for 3 days followed by 20 mg/kg/day for 4 days
   • At least as effective as IVIg with 60-100% response rate
   • Response within 2-7 days 1

2. Dexamethasone

   • 28-40 mg/m²/day
   • Response rate up to 80% in previously untreated patients
   • Side effects include sleeplessness, aggressive behavior, loss of concentration 1

3. Rituximab

   • 100 mg or 375 mg/m²/week for 4 weeks
   • Response rates between 31-79%
   • Generally well tolerated; potential side effects include serum sickness, rash, arthralgia
   • 63% achieved complete response lasting 4-30 months 1

4. TPO-receptor agonists

   • Romiplostim has shown efficacy in pediatric patients ≥1 year of age with ITP for at least 6 months who have had insufficient response to corticosteroids, immunoglobulins, or splenectomy 2
   • In clinical studies, 88.2% of pediatric patients achieved platelet counts ≥50 × 10^9/L for 2 consecutive weeks 2

5. Splenectomy

   • 60-70% long-term response rate
   • 80% of responders maintain platelet response over 4 years
   • Should be deferred as long as possible due to risk of post-splenectomy infections
   • Consider for children with symptomatic, severe thrombocytopenia who have failed primary therapy 1, 3

Special Considerations

• Menstruation: Can be managed with antifibrinolytic agents and hormonal medication
• Safety precautions: Family should carry information about the condition; medical bracelet or pendant may be appropriate
• Quality of life: Consider treatment for children with platelet counts 10-30 × 10^9/L who are troubled by purpura, especially adolescents conscious of appearance 1

Common Pitfalls and Caveats

1. Prolonged corticosteroid use: Should be avoided in children due to serious side effects; use only to maintain hemostatic platelet count for as short a time as possible 1

2. Cytotoxic drugs: Should be used with extreme caution in children 1

3. Bone marrow examination: Not necessary for patients presenting with typical ITP, but should be performed in children with atypical features (e.g., hepatosplenomegaly) 4

4. Treatment goals: Aim to increase platelet count to safe levels (>30-50 × 10^9/L) to prevent bleeding, not normalize counts 5

5. Activity restrictions: Children should not participate in competitive contact activities with high risk of head trauma, but other activities need not be restricted 1