What is the approach to Immune Thrombocytopenic Purpura (ITP)?

From the Guidelines

The approach to Immune Thrombocytopenic Purpura (ITP) should prioritize a patient-centered strategy, focusing on bleeding risk, platelet count, and symptoms, with first-line treatment options including corticosteroids or intravenous immunoglobulin (IVIG) for rapid platelet increase in emergency situations, as supported by the most recent and highest quality study 1.

Introduction to ITP Management

The management of ITP involves a comprehensive assessment of the patient's condition, including the severity of bleeding, the rapidity of desired platelet count rise, and possible side effects of treatments. According to the American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia 1, treatment should be tailored to the individual patient.

First-Line Treatment Options

For newly diagnosed ITP patients with platelet counts above 30,000/μL and no significant bleeding, observation alone may be appropriate. However, for those with counts below 30,000/μL or with bleeding symptoms, first-line treatment includes:

• Corticosteroids (prednisone 1 mg/kg/day for 2-4 weeks with subsequent taper) 1
• Intravenous immunoglobulin (IVIG 1 g/kg/day for 1-2 days) for rapid platelet increase in emergency situations

Second-Line Treatment Options

If patients fail to respond to first-line therapy, second-line options include:

• Thrombopoietin receptor agonists (eltrombopag 50 mg daily or romiplostim 1-10 μg/kg weekly) 1
• Rituximab (375 mg/m² weekly for 4 weeks)
• Splenectomy, which provides the highest long-term remission rates (60-70%) but is typically reserved for patients who have failed medical management due to its irreversibility and infection risks

Quality of Life and Treatment Considerations

The introduction of thrombopoietin receptor agonists (TPO-RAs) has a positive impact on the quality of life of ITP patients, with response rates of 70-80% documented, and a median exposure time of 2 years 1. Throughout treatment, patients should avoid medications that impair platelet function (NSAIDs, aspirin) and activities with high bleeding risk. The pathophysiology of ITP involves autoantibody-mediated platelet destruction and impaired platelet production, which explains why therapies target either reducing antibody production, preventing platelet destruction, or enhancing platelet production.

Key Considerations

• Increasing age is a major risk factor for bleeding in ITP patients 1
• The use of rituximab reduces the efficacy of concomitant vaccinations and is associated with rare but lethal complications, including hepatitis B reactivation and multifocal leukoencephalopathy 1
• TPO-RAs are well tolerated, especially in the long-term, and are not linked to increased thrombotic risk, although the correlation between TPO-RA usage and thrombotic risk is still under debate 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 Nplate is used to try to keep your platelet count about 50,000 per microliter in order to lower the risk for bleeding.

The approach to Immune Thrombocytopenic Purpura (ITP) using Nplate involves administering the medication via subcutaneous injection once a week, with dose adjustments as needed to maintain a platelet count of 50,000 per microliter, in order to lower the risk of bleeding. The treatment is intended for adults with ITP who have not responded well to other medicines or splenectomy 2.

From the Research

Approach to Immune Thrombocytopenic Purpura (ITP)

The approach to ITP involves several treatment options, including:

• First-line treatments:
  • Corticosteroids, such as prednisone or dexamethasone, to increase platelet counts 3, 4, 5, 6, 7
  • Intravenous immunoglobulins (IVIg) for severe forms 3, 5, 6, 7
  • Intravenous anti-D immunoglobulin for rapid platelet increases 5, 6, 7
• Second-line treatments:
  • Splenectomy for patients who do not respond to first-line treatments 4, 5, 6, 7
  • Rituximab, a monoclonal antibody, for patients with chronic ITP 6, 7
  • Thrombopoietin receptor agonists, which are currently under clinical investigation 5, 7
• Other treatment options:
  • Anti-FcRn monoclonal antibodies and recombinant FcγR, which are currently in development 3
  • Danazol, azathioprine, cyclophosphamide, vinca alkaloids, and cyclosporin A, which have limited evidence of efficacy 6

Treatment Goals and Considerations

The goal of treatment for ITP is to prevent serious bleeding and increase platelet counts 4, 5, 6, 7. The treatment approach should be individualized, taking into account the patient's specific needs and circumstances 7. The benefits and risks of each treatment option should be carefully considered, including the potential for side effects and the risk of relapse 4, 5, 6, 7.