What are the latest treatments for Immune Thrombocytopenic Purpura (ITP)?

From the Guidelines

The latest treatment for Immune Thrombocytopenia (ITP) follows a stepwise approach based on bleeding risk and platelet count, with first-line therapy typically including corticosteroids such as prednisone (1 mg/kg/day for 2-4 weeks with gradual taper) or dexamethasone (40 mg daily for 4 days), and second-line options including thrombopoietin receptor agonists (TPO-RAs) like eltrombopag (25-75 mg daily), romiplostim (1-10 μg/kg weekly), or avatrombopag (20-40 mg daily) 1.

First-line Therapy

• Corticosteroids, including prednisone and high-dose dexamethasone, are commonly administered in adult ITP patients as first-line therapy to rapidly achieve safe platelet counts, prevent or interrupt bleeding events, and ensure an acceptable quality of life 1.
• Initial responsiveness to corticosteroids is observed in about 60–80% of patients, but sustained responses are seen only in 20–40% of cases, highlighting an important loss of efficacy in the long-term 1.

Second-line Therapy

• TPO-RAs, such as eltrombopag and romiplostim, are therapeutic options approved for second-line treatment of ITP, with a mechanism of action that involves agonistic activity on the thrombopoietin receptor, triggering an increase of megakaryopoiesis and platelet production 1.
• TPO-RAs have been shown to be highly effective, with initial responses observed already after 1–2 weeks of treatment, while significantly reducing bleeding events and emergency hospitalizations, and are well tolerated, especially in the long-term 1.

Treatment Selection

• Treatment selection depends on patient factors including age, comorbidities, bleeding risk, and treatment goals, with the aim of maintaining platelet counts above 30,000/μL or higher if bleeding is present 1.
• The use of TPO-RAs, rituximab, and other second-line therapies should be considered in patients who do not respond to first-line treatments, with the goal of achieving a durable response and improving quality of life 1.

From the FDA Drug Label

The safety and efficacy of Nplate in adults with ITP were assessed in two double-blind, placebo-controlled clinical studies, an open-label single-arm study, and in an open-label extension study ALVAIZ® (eltrombopag tablets) are indicated for the treatment of thrombocytopenia in adult and pediatric patients 6 years and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy

The latest treatments for Immune Thrombocytopenic Purpura (ITP) include:

• Romiplostim (SC): a thrombopoietin receptor agonist that stimulates platelet production
• Eltrombopag (PO): a thrombopoietin receptor agonist that stimulates platelet production These treatments are used in patients with ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. The goal of treatment is to increase platelet counts and reduce the risk of bleeding. 2 3

From the Research

Treatment Options for Immune Thrombocytopenic Purpura (ITP)

• First-line treatments for ITP include corticosteroids, intravenous immunoglobulin, and intravenous anti-D immunoglobulin 4, 5, 6, 7.
• Second-line treatments consist of rituximab, thrombopoietin receptor agonists, and splenectomy 4, 6, 7.
• For patients who fail initial therapy, subsequent treatments may include thrombopoietic agents, fostamatinib, and several older immunosuppressive agents 7.
• Emerging therapeutic approaches for refractory ITP target various pathways, including macrophages, platelets/megakaryocytes, T cells, B cells, and endothelial cells 6.
• Novel agents being developed for ITP treatment include splenic tyrosine kinase inhibitor, Bruton kinase inhibitor, plasma cell targeting therapies, neonatal Fc receptor inhibitor, platelet desialylation inhibitor, and inhibition of the classical complement pathway 8.

Specific Treatment Strategies

• Corticosteroids have been the standard first-line treatment for symptomatic patients, with the addition of intravenous immune globulin (IVIG) or Rho(D) immune globulin for steroid-resistant cases 4.
• Rituximab, a monoclonal antibody against the CD20 antigen, is used as a second-line therapy for refractory or non-responsive ITP 4, 6, 7.
• Splenectomy is also considered as a second-line treatment option for patients with refractory ITP 4, 6, 7.
• Thrombopoietin receptor agonists are used to stimulate platelet production in patients with ITP 6, 7, 8.

Treatment Goals and Considerations

• The goal of treatment is to elevate platelet counts to a safe level with minimal toxicity and improve patient health-related quality of life 7.
• Treatment strategies should be tailored to individual patients and disease phases 7.
• The management of refractory ITP patients remains a challenge, and emerging therapeutic approaches are being developed to address this issue 6, 8.