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

From the Guidelines

Treatment for immune thrombocytopenia (ITP) should prioritize thrombopoietin receptor agonists (TPO-RAs) as second-line therapy due to their high efficacy and favorable safety profile, as suggested by the American Society of Hematology 2019 guidelines 1. The choice of treatment for ITP depends on the severity of symptoms and platelet count. First-line treatment typically involves corticosteroids such as prednisone (1 mg/kg/day for 2-4 weeks) or dexamethasone (40 mg daily for 4 days) to rapidly achieve safe platelet counts and prevent bleeding events 1. However, prolonged exposure to corticosteroids can lead to severe adverse events, and initial responsiveness is often not sustained in the long-term 1.

For persistent ITP, second-line options include:

• TPO-RAs like eltrombopag (starting at 50 mg daily) or romiplostim (1-10 μg/kg weekly subcutaneously), which have been shown to be highly effective in clinical trials with response rates of 70-80% 1
• Rituximab (375 mg/m² weekly for 4 weeks), which can trigger short-term responses in 50-60% of patients, but is associated with infusion-related toxicities and rare but lethal complications 1
• Splenectomy, which is associated with initial response in 85% of cases, but also carries a risk of short-term and long-term complications 1

Severe bleeding requires hospitalization and possibly platelet transfusions. Treatment aims to increase platelet production and decrease platelet destruction by modulating the immune response that targets platelets. Lifestyle modifications to reduce bleeding risk include avoiding contact sports and certain medications like aspirin and NSAIDs. Regular monitoring of platelet counts is essential to assess treatment response and adjust therapy accordingly. The American Society of Hematology 2019 guidelines suggest a TPO-RA rather than rituximab for adults with ITP lasting >3 months who are corticosteroid-dependent or have no response to corticosteroids 1.

From the FDA Drug Label

Nplate is a prescription medicine used to treat low blood platelet counts (thrombocytopenia) in: adults with immune thrombocytopenia (ITP) when certain medicines or surgery to remove your spleen have not worked well enough children 1 year of age and older with ITP for at least 6 months when certain medicines or surgery to remove your spleen have not worked well enough.

Treatment for ITP: Nplate (romiplostim) is used to treat adults and children 1 year of age and older with immune thrombocytopenia (ITP) when certain medicines or surgery to remove the spleen have not worked well enough.

• The goal of Nplate treatment is to keep the platelet count about 50,000 per microliter in order to lower the risk for bleeding.
• Nplate is given by a healthcare provider as an injection under the skin (subcutaneous) one time each week.
• The dose of Nplate is adjusted based on the patient's platelet count, and the healthcare provider will closely monitor the patient's platelet count and adjust the dose as needed 2.

From the Research

Treatment Options for ITP

• First-line treatments for ITP include corticosteroids, immunoglobulins, or other therapies 3, 4, 5
• Corticosteroids are the standard initial treatment for ITP, with high-dose dexamethasone and conventional prednisone being commonly used options 4
• Intravenous immunoglobulin or anti-D immunoglobulin can also increase platelet counts and are useful for stimulating rapid platelet increases before planned procedures 3, 6
• Splenectomy is still commonly used for patients who do not have long-term responses to steroid therapy, but it is an invasive procedure with potential risks and complications 3, 7

Second-Line and Third-Line Treatments

• Second-line treatments for ITP include immunosuppressive drugs and splenectomy 5, 7
• Third-line treatments aim to stimulate platelet production by megakaryocytes, with options including thrombopoietin receptor agonists (TPO-RAs) and fostamatinib 5, 7
• Rituximab is also a treatment option for patients with ITP, particularly those who are unresponsive to or dependent on corticosteroid treatment 7, 6

Treatment Considerations

• Treatment should be tailored according to the patient's age, lifestyle, comorbidities, and compliance 5
• The goal of treatment is to prevent severe or life-threatening bleeding, and treatment decisions should be based on the individual patient's risk of bleeding and response to therapy 3, 5, 7
• Patient involvement, preferences, and values should be central to the process of choosing the appropriate therapy 7