What is the initial treatment for a patient with Immune Thrombocytopenic Purpura (ITP)?

Treatment for Immune Thrombocytopenic Purpura (ITP)

Corticosteroids are the standard initial treatment for newly diagnosed ITP in adults requiring therapy, with prednisone (0.5-2 mg/kg/day) or high-dose dexamethasone (40 mg/day for 4 days) as first-line options. 1, 2, 3

When to Initiate Treatment

Treatment is indicated when: 2, 3

• Platelet count <30 × 10⁹/L with bleeding symptoms
• Platelet count <20 × 10⁹/L regardless of bleeding symptoms
• Active CNS, GI, or genitourinary bleeding at any platelet count 4
• Urgent surgery is required 4
• Patients >60 years old or with previous hemorrhage have higher bleeding risk and may warrant earlier intervention 2

Treatment is rarely needed if platelet count >50 × 10⁹/L unless active bleeding, surgery, bleeding-prone comorbidities, or anticoagulation requirements exist. 3

First-Line Corticosteroid Options

Prednisone

• Dose: 0.5-2 mg/kg/day until platelet count reaches 30-50 × 10⁹/L, then rapidly taper 2
• Initial response rate: 70-80% 3
• Sustained long-term response: only 20-40% 2, 3
• Most commonly recommended initial option 3

High-Dose Dexamethasone

• Dose: 40 mg/day for 4 days 2
• Initial response rate: up to 90% 3
• Sustained response: 50-80% with 3-6 cycles 3
• Preferred for severe thrombocytopenia with active bleeding due to faster platelet response 2, 5
• Works faster than prednisone and appears safer with lower incidence of adverse events, likely due to shorter treatment duration 5
• Dexamethasone shows increased platelet count response at 7 days (RR 1.31; 95% CI 1.11-1.54) and higher remission rates (RR 2.96; 95% CI 1.03-8.45) compared to prednisone 1

Adjunctive First-Line Therapies

Intravenous Immunoglobulin (IVIg)

• Dose: 1 g/kg as a single dose 2, 4
• Achieves platelet increase within 24 hours 3, 4
• Use when rapid platelet increase is required 2, 3
• Can be combined with corticosteroids for enhanced response and reduced infusion reactions 3, 4

Anti-D Immunoglobulin

• Dose: 75 mcg/kg 2
• Only for Rh(D)-positive, non-splenectomized patients 2, 4
• Provides predictable, transient platelet increases 3, 6

Emergency Treatment Protocol

For severe bleeding or platelet count <10 × 10⁹/L with high bleeding risk: 2, 4

• Combine prednisone plus IVIg
• Consider high-dose methylprednisolone (15 mg/kg/day) for rapid response 2, 7
• Platelet transfusion, possibly with IVIg, in emergency settings 4
• Emergency splenectomy in life-threatening situations 4

High-dose methylprednisolone achieves safe platelet counts (>50 × 10⁹/L) within 2-5 days in refractory cases, though the effect may be transient. 7

Critical Corticosteroid Management

Corticosteroids should not be continued beyond 6-8 weeks for initial treatment. 2 Patients requiring on-demand corticosteroids after completing induction should be considered non-responders and switched to second-line therapy. 2

Short-term side effects to monitor: 3

• Mood swings, weight gain, fluid retention
• Cushingoid features, hyperglycemia

Long-term side effects to monitor: 3

• Osteoporosis, avascular necrosis
• Hypertension, diabetes
• Skin changes, cataracts
• Immunosuppression with opportunistic infections

Rituximab as Initial Treatment

The American Society of Hematology suggests corticosteroids alone rather than rituximab plus corticosteroids for initial therapy (conditional recommendation). 1 While rituximab combined with corticosteroids shows higher durable response (RR 1.70; 95% CI 1.34-2.16) and remission rates (RR 1.58; 95% CI 1.00-2.52), there is very low certainty in the evidence due to missing quality-of-life data and potential for increased adverse events. 1

Exception: If high value is placed on possibility for remission over concerns for rituximab side effects, initial corticosteroids with rituximab may be preferred. 1 Dexamethasone combined with rituximab in first-line treatment produces higher response rates with better long-term results, particularly in younger women. 5

Special Populations

Pregnancy

• Use corticosteroids or IVIg only 2, 3
• Mode of delivery should be based on obstetric indications, not maternal platelet count 2, 3

HIV-Associated ITP

• Treat underlying HIV with antivirals before ITP-specific therapy unless clinically significant bleeding 2, 4

HCV-Associated ITP

• Consider antiviral therapy first 4
• If ITP treatment required, use IVIg 4

H. pylori-Associated ITP

• Administer eradication therapy in patients with confirmed H. pylori infection 4

When First-Line Fails

Patients are considered corticosteroid failures if: 2

• No response after 4 weeks of treatment
• Platelet count drops below safe levels during taper
• Require continuous corticosteroids to maintain platelet count

Second-Line Options

• Splenectomy: 80% initial response, 60-65% long-term response 3
• Thrombopoietin receptor agonists (TPO-RAs): Increasingly preferred before splenectomy due to high response rates and potential for remission 3
• Romiplostim is indicated for adult and pediatric patients (≥1 year) with ITP who have had insufficient response to corticosteroids, immunoglobulins, or splenectomy 8
• Rituximab: May be considered for patients who have failed one line of therapy 4

Critical Pitfalls to Avoid

• Do not use corticosteroids to normalize platelet counts—the goal is to maintain counts around 50,000/μL to reduce bleeding risk 2, 8
• Do not continue corticosteroids beyond 6-8 weeks without reassessing for second-line therapy 2
• Monitor for thrombotic complications if platelet counts become excessively elevated, particularly with TPO-RA use 8
• Cessation of drugs reducing platelet function is essential 4
• Control blood pressure and inhibit menses to minimize bleeding risk 4