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

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

The treatment of Immune Thrombocytopenic Purpura (ITP) should follow a stepwise approach, beginning with corticosteroids as first-line therapy, followed by thrombopoietin receptor agonists (TPO-RAs), rituximab, or splenectomy as second-line options based on response and bleeding risk. 1, 2

First-Line Treatment Options

• Corticosteroids are the standard initial therapy for ITP, with prednisone typically administered at 0.5-2 mg/kg/day until platelet count increases 2
• Corticosteroid treatment should be limited to 6-8 weeks to avoid severe adverse events such as weight gain, cataract, mood alterations, hypertension, infections, hyperglycemia, and osteoporosis 3
• Dexamethasone shows high initial response rates up to 90% with sustained responses in 50-80% of patients when given in 1-4 cycles 2
• Intravenous immunoglobulin (IVIg) should be added when a more rapid increase in platelet count is required, with response rates up to 80% and many patients responding within 24 hours 1, 2
• IVIg is typically administered at 0.4 g/kg/day for 5 days or 1 g/kg/day for 1-2 days 2
• IV anti-D (50-75 μg/kg) can be used in non-splenectomized Rh(D)-positive patients as an alternative to IVIg 3

Second-Line Treatment Options

• Thrombopoietin Receptor Agonists (TPO-RAs) such as romiplostim are recommended for patients who relapse after splenectomy or have contraindications to splenectomy and have failed at least one other therapy 1, 2
• TPO-RAs should be considered early in patients who require on-demand administration of corticosteroids after completing first-line treatment, as they show a safer profile compared to prolonged corticosteroid use 3
• Rituximab (anti-CD20 monoclonal antibody) has response rates of 31-79%, with short-term responses in 50-60% of patients and long-term responses in 20-30% of cases 3, 2
• Splenectomy remains highly effective with initial response in 85% of cases and durable responses in 60-70% of patients, making it the gold standard for those who fail corticosteroid therapy 3, 1, 4
• Splenectomy should be considered carefully due to short-term complications (within 30 days of surgery) and long-term complications in approximately 10% of patients 3

Third-Line and Alternative Treatment Options

• Immunosuppressive agents including azathioprine, cyclosporin A, mycophenolate mofetil, and danazol can be considered for refractory cases with response rates ranging from 40-80% 2
• Combining first-line therapies may be appropriate for refractory patients, such as high-dose methylprednisolone with IVIg 3, 2
• In emergency situations with severe bleeding, high-dose corticosteroids combined with IVIg or IV anti-D are recommended, and platelet transfusions at larger-than-usual doses may be considered 2

Special Populations

• For pregnant patients, corticosteroids (prednisone 10-20 mg/day) or IVIg are recommended first-line treatments 3, 1
• Mode of delivery for pregnant ITP patients should be based on obstetric indications rather than platelet count 1
• In secondary ITP, addressing the underlying cause is essential:
  • Antiviral therapy for HCV-associated ITP 1
  • Antiretroviral therapy for HIV-associated ITP 1
  • Eradication therapy for H. pylori-positive patients 1, 2

Important Safety Considerations

• TPO-RAs like romiplostim carry risks of blood clots, especially if platelet counts become too high during treatment 5
• Patients on romiplostim require regular monitoring of platelet counts to adjust dosing appropriately 5
• Rituximab can reduce the efficacy of concomitant vaccinations and is associated with rare but serious complications including hepatitis B reactivation and multifocal leukoencephalopathy 3
• The decision to treat should be based primarily on bleeding risk rather than platelet count alone, with treatment generally indicated for patients with platelet counts <20-30 × 10^9/L or <50 × 10^9/L with significant mucous membrane bleeding 4