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

Management of Immune Thrombocytopenic Purpura (ITP)

First-line treatment for ITP should include corticosteroids, with the addition of IVIG when rapid platelet count increase is required. 1

Diagnostic Approach

• Testing for HCV and HIV is recommended for all patients with suspected ITP (grade 1B) 1
• Bone marrow examination is not necessary for patients presenting with typical ITP (grade 2C) 1
• Further investigations are suggested if there are abnormalities other than thrombocytopenia in the blood count or smear 1
• Secondary causes of ITP should be ruled out, including antiphospholipid syndrome, autoimmune disorders, common variable immune deficiency, drug-induced thrombocytopenia, and infections 1

First-Line Treatment Options

Corticosteroids

• Standard initial therapy for adult ITP with platelet counts <30 × 10^9/L or with bleeding symptoms 2
• Options include:
  • Prednisone (0.5-2 mg/kg/day) until platelet count increases 2
  • Dexamethasone (high-dose regimens) showing response rates up to 90% 2
• Corticosteroids should be rapidly tapered and discontinued after 4 weeks in non-responders to avoid complications 2

Intravenous Immunoglobulin (IVIG)

• Recommended when a more rapid increase in platelet count is required (grade 2B) 1
• Initial dose should be 1 g/kg as a one-time dose; may be repeated if necessary 1
• Produces rapid responses in up to 80% of patients, often within 24 hours 2
• Can be used as first-line treatment if corticosteroids are contraindicated 1

Anti-D Immunoglobulin

• May be used as a first-line treatment in appropriate patients if corticosteroids are contraindicated (grade 2C) 1
• Only effective in Rh-positive, non-splenectomized patients 1

Second-Line Treatment Options

Splenectomy

• Recommended for patients who have failed corticosteroid therapy (grade 1B) 1
• Provides long-term responses in 60-70% of patients 2
• Both laparoscopic and open splenectomy offer similar efficacy (grade 1C) 1
• Appropriate vaccinations should be administered prior to splenectomy 1

Thrombopoietin Receptor Agonists (TPO-RAs)

• 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
• May be considered for patients who have failed one line of therapy such as corticosteroids or IVIG without splenectomy (grade 2C) 1
• Romiplostim has shown durable platelet responses in patients with ITP duration >1 year 2, 3
• TPO-RAs have demonstrated efficacy in patients with antibody deficiencies, with 85.7% response rate after 6 weeks 4
• Potential side effects include risk of blood clots if platelet count becomes too high 3

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 rates range from 31-79% 2

Management of Secondary ITP

HCV-Associated ITP

• Antiviral therapy should be considered in the absence of contraindications (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

• Treatment of HIV infection with antiviral therapy should be considered before other treatment options unless clinically significant bleeding is present (grade 1A) 1
• If ITP treatment is required, initial treatment should consist of corticosteroids, IVIG, or anti-D (grade 2C) 1
• Splenectomy is preferred for symptomatic patients who fail initial therapies 1

H. pylori–Associated ITP

• Screening for H. pylori should be considered in patients with ITP (grade 2C) 1
• Eradication therapy is recommended for patients who test positive for H. pylori (grade 1B) 1

Special Populations

Pregnancy

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

Children

• Treatment approach is similar to adults but with more caution regarding long-term corticosteroid use 2
• Response rates to IVIG, high-dose steroids, and low-dose steroids are similar (78-89%), but time to response is faster with IVIG and high-dose steroids 5
• Observation alone may be appropriate for children without significant bleeding, with lower recurrence rates compared to pharmacotherapy 5

Emergency Treatment for Severe Bleeding

• High-dose corticosteroids combined with IVIG are recommended 2
• Platelet transfusions at larger-than-usual doses may be considered in life-threatening situations 2

Treatment Algorithm

1. Assess bleeding risk and platelet count
2. First-line therapy:
   • For non-bleeding patients with platelets >30 × 10^9/L: Consider observation
   • For patients with bleeding or platelets <30 × 10^9/L: Start corticosteroids
   • If rapid platelet increase needed: Add IVIG (1 g/kg)
3. If no response to first-line therapy after 4 weeks:
   • Consider splenectomy (traditional second-line)
   • Consider TPO-RAs or rituximab before splenectomy
4. For refractory ITP:
   • TPO-RAs if not tried previously
   • Consider combination therapies
5. Always treat underlying causes in secondary ITP