What are the treatment options for a child with immune thrombocytopenia (ITP)?

Pediatric ITP Management

Initial Assessment and Observation Strategy

Children with no bleeding or only mild bleeding (bruising and petechiae only) should be managed with observation alone, regardless of platelet count. 1 This is the most important principle in pediatric ITP management, as the risk of intracranial hemorrhage is only 0.1-0.5%, and most cases resolve spontaneously. 2

Diagnostic Workup

• Bone marrow examination is NOT necessary in children with typical ITP features, even before starting corticosteroids or IVIg, and even if IVIg therapy fails. 1
• Testing for antinuclear antibodies is not required in the evaluation of suspected pediatric ITP. 1
• Parents should be counseled to avoid contact sports with high risk of head trauma, but other activities need not be restricted. 2

First-Line Pharmacologic Treatment

When treatment is indicated (moderate to severe bleeding), use either a single dose of IVIg (0.8-1 g/kg) OR a short course of corticosteroids as first-line therapy. 1

IVIg Therapy (Preferred for Rapid Response)

• IVIg should be used when a more rapid platelet increase is needed. 1
• Single dose of 0.8-1 g/kg raises platelet count in more than 80% of children. 1, 3
• Response time: 1-2 days, with peak effect at days 3-7. 1, 3
• Low-dose IVIg (0.6-1 g/kg single dose) is equally effective as high-dose IVIg (2 g/kg) with significantly fewer adverse reactions (3% vs 6%). 4
• Common side effects include fever, headache, nausea, and vomiting. 1, 3

Corticosteroid Therapy

• Prednisone 4 mg/kg/day for 3-4 days is effective in 72-88% of children. 1, 5
• Response time: 2-7 days, slower than IVIg. 1, 5
• Critical caveat: Use corticosteroids only for as short a time as possible due to serious side effects with prolonged use in children. 1
• Low-dose steroids (1-2 mg/kg/day) have similar response rates (89%) but slower recovery time (16.8 days). 5

Anti-D Immunoglobulin (Alternative Option)

• Can be used in Rh-positive, non-splenectomized children at 50-75 mcg/kg. 1
• Do NOT use if hemoglobin is already decreased from bleeding or if autoimmune hemolysis is present. 1
• Response is slower than IVIg (peak at 7-14 days) and cannot be recommended for children with platelet counts ≤20 × 10⁹/L. 3
• Mean hemoglobin decrease of 0.8 g/dL occurs in 61% of patients. 6

Emergency Treatment for Life-Threatening Bleeding

For organ-threatening or life-threatening bleeding, use immediate combination therapy: 1, 2

• Platelet transfusion (2-3 fold larger than usual dose) 1, 2
• IV high-dose methylprednisolone (30 mg/kg/day) 1, 2
• IVIg (0.8-1 g/kg) or IV anti-D 1, 2
• Fresh frozen plasma if coagulation studies are prolonged. 2

Never delay emergency treatment while awaiting complete diagnostic workup. 2

Second-Line Treatments for Persistent or Chronic ITP

Rituximab

• Consider for children with ongoing bleeding despite IVIg, anti-D, or conventional corticosteroids. 1
• Dose: 375 mg/m²/week for 4 weeks (or 100 mg/m² weekly). 1
• Response rates: 31-79%, with 63% achieving complete response lasting 4-30 months. 1
• Generally well tolerated with mild side effects (serum sickness, rash, arthralgia). 1

High-Dose Dexamethasone

• Consider for refractory cases: 28 mg/m²/day. 1
• Response rate: up to 80%. 1
• Side effects include sleeplessness, behavioral changes, hypertension, anxiety, gastric distress. 1
• Responses are of short duration unless cycles are repeated. 1

Splenectomy Considerations

Splenectomy should be reserved for children with chronic or persistent ITP who have significant bleeding, lack of response to other therapies, or quality of life concerns. 1

• Delay splenectomy for at least 12 months unless severe unresponsive disease. 1
• Long-term response rate: 60-70%, with 80% maintaining response over 4 years. 1
• Risk of postsplenectomy sepsis must be considered. 1
• Repeated doses of anti-D may serve as an alternative to splenectomy in patients with recurrent bleeding, maintaining platelet counts >50 × 10⁹/L in 75% at 1 week and 60% at 4 weeks. 6

Key Clinical Pitfalls to Avoid

• Do not treat based on platelet count alone—bleeding severity determines treatment need. 1
• Do not use prolonged corticosteroid courses due to significant toxicity in children. 1
• Do not use anti-D in children with anemia from bleeding or evidence of hemolysis. 1
• Observation alone has significantly lower recurrence rates (3%) compared to any pharmacotherapy (23-39%). 5

MMR Vaccination in Children with ITP History

• Children with a history of ITP who are unimmunized should receive their scheduled first MMR vaccine. 1
• If already vaccinated, check vaccine titers; if fully immune (90-95%), no further MMR vaccine needed. 1