What is the diagnosis and treatment for Immune Thrombocytopenic Purpura (ITP)?

From the Guidelines

Immune thrombocytopenic purpura (ITP) should be diagnosed and treated with a focus on increasing platelet counts to a safe level and preventing bleeding complications, with first-line treatment typically including corticosteroids, such as prednisone 0.5-2.0 mg/kg per day or dexamethasone 40 mg per day for 4 days, as recommended by the American Society of Hematology 2019 guidelines 1.

Diagnosis and Treatment Overview

ITP is an autoimmune disorder characterized by low platelet counts, which can lead to easy bruising and bleeding. The primary treatment goal is to increase platelet counts to a safe level and prevent bleeding complications.

First-Line Treatment

First-line treatment for adults with ITP typically includes:

• Corticosteroids: Prednisone 0.5-2.0 mg/kg per day or dexamethasone 40 mg per day for 4 days, as recommended by the American Society of Hematology 2019 guidelines 1
• Intravenous immunoglobulin (IVIG): 1 g/kg/day for 1-2 days in cases of severe thrombocytopenia or bleeding

Second-Line Treatment

For patients who don't respond to first-line treatments or relapse, second-line options include:

• Thrombopoietin receptor agonists: Eltrombopag (50-75 mg daily) or Romiplostim (1-10 μg/kg weekly subcutaneously), as suggested by the American Society of Hematology 2019 guidelines 1
• Rituximab: 375 mg/m² IV weekly for 4 weeks
• Splenectomy (for refractory cases)

Patient Monitoring and Education

Patients should be monitored regularly with complete blood counts and advised to avoid activities with a high risk of injury. They should also be educated about signs of bleeding and when to seek medical attention.

Rationale Behind Treatments

The rationale behind these treatments is to reduce autoantibody production against platelets (corticosteroids, IVIG, rituximab) or stimulate platelet production (thrombopoietin receptor agonists). Splenectomy removes the primary site of platelet destruction and autoantibody production. Key considerations in the management of ITP include the severity of thrombocytopenia, the presence of bleeding symptoms, and the patient's overall health status, as outlined in the international consensus report on the investigation and management of primary immune thrombocytopenia 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.

The diagnosis of Immune Thrombocytopenic Purpura (ITP) is not explicitly stated in the provided drug labels, but it is implied that a diagnosis of ITP is necessary for treatment with Nplate. The treatment for ITP with Nplate involves weekly subcutaneous injections, with the goal of maintaining a platelet count of at least 50,000 per microliter to lower the risk of bleeding. The dose of Nplate is adjusted based on the patient's platelet count, and patients are closely monitored for side effects and changes in their platelet count 2.

• Key points:
  • Nplate is used to treat ITP in adults and children 1 year of age and older.
  • Treatment with Nplate involves weekly subcutaneous injections.
  • The goal of treatment is to maintain a platelet count of at least 50,000 per microliter.
  • Patients are closely monitored for side effects and changes in their platelet count.
• Important considerations:
  • Nplate is not used to make the platelet count normal, but rather to lower the risk of bleeding.
  • Patients with a history of blood clots or chronic liver disease may be at increased risk of complications.
  • Women who are pregnant or breastfeeding should discuss the risks and benefits of Nplate with their healthcare provider 2.

From the Research

Diagnosis of Immune Thrombocytopenic Purpura (ITP)

• ITP can be challenging to diagnose, as it is a diagnosis of exclusion, despite the ability to detect anti-platelet antibodies 3
• The diagnosis of ITP remains one of exclusion, and bleeding, not platelet count, should be the rationale for treatment 3

Treatment of Immune Thrombocytopenic Purpura (ITP)

• The goal of treatment for ITP is to prevent serious bleeding, and traditionally, corticosteroids have been used as first-line therapy, followed by splenectomy 4
• First-line treatment for ITP includes corticosteroids, immunoglobulins, or other therapies, with the aim of increasing platelet counts and preventing bleeding 3, 5
• 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 4, 3
• Other treatments, such as high-dose dexamethasone, intermittent anti-D immunoglobulin infusions, and rituximab, have been evaluated, but their efficacy and safety remain unclear 3, 6
• Thrombopoietin receptor agonists are currently under clinical investigation for the treatment of ITP and may represent an alternative treatment option in the future 3
• Treatment modalities target various aspects of ITP pathophysiology, including the inhibition of autoantibody production, modulation of T cell activity, and stimulation of platelet production 7
• The American Society of Hematology and the International Society of Thrombosis and Hemostasis have published guidelines on the treatment of ITP patients, with first-line treatment focusing on inhibition of autoantibody production and platelet degradation, second-line treatments including immunosuppressive drugs and splenectomy, and third-line treatments aiming to stimulate platelet production by megakaryocytes 7

Treatment Outcomes and Response

• Initial responses to first-line treatment are similar for different therapies, but sustained responses and overall duration of response may vary 5
• The presence of anti-GPIb-IX autoantibodies may be a predictive factor for a poor initial response to corticosteroids therapy 5
• Adverse events may be more frequent and long-lasting with certain treatments, such as prednisone 5
• Monoclonal antibodies, such as rituximab, may have a role in the treatment of ITP, particularly for refractory and chronic patients, and may spare splenectomy and possibly cure the disease in some patients 6