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

From the Guidelines

For adults with newly diagnosed ITP, the initial therapy should be either prednisone (0.5-2.0 mg/kg per day) or dexamethasone (40 mg per day for 4 days) as the type of corticosteroid, with a preference for dexamethasone if a rapid platelet count response is desired. This recommendation is based on the American Society of Hematology 2019 guidelines for immune thrombocytopenia 1. The choice between prednisone and dexamethasone should consider the patient's individual circumstances, including the need for a rapid increase in platelet count.

Key Considerations in ITP Management

• ITP is an acquired autoimmune disorder characterized by a low platelet count resulting from platelet destruction and impaired platelet production 1.
• The clinical course of ITP can vary significantly among patients, with some experiencing spontaneous remission, while others may require ongoing treatment to manage symptoms and prevent bleeding complications.
• Treatment decisions should be individualized based on factors such as bleeding risk, platelet count severity, comorbidities, and patient preferences.

Treatment Options for ITP

• First-line treatment: Corticosteroids such as prednisone or dexamethasone 1.
• Second-line options: Thrombopoietin receptor agonists (TPO-RAs) like eltrombopag or romiplostim, rituximab, or splenectomy for refractory cases 1.
• The goal of treatment is to achieve a safe platelet count (>30,000/μL) rather than normal levels, focusing on preventing serious bleeding while minimizing treatment side effects.

Quality of Life and Bleeding Risk

• ITP can have a significant impact on health-related quality of life (HRQoL), particularly in the first year after diagnosis, related to restrictions on activities, anxiety due to the risk of bleeding, and the burden of treatment and monitoring 1.
• Bleeding events are often unpredictable, and patients with ITP may experience severe bleeding, including mucosal bleeding, gastrointestinal hemorrhage, or, rarely, intracranial hemorrhage (ICH) 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.

ITP Treatment: Nplate 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.

• Key Points:
  • Nplate is used to treat low blood platelet counts (thrombocytopenia) in patients with ITP.
  • It is used when other treatments, such as certain medicines or surgery to remove the spleen, have not worked well enough.
  • Nplate is given by injection under the skin (subcutaneous) one time each week.
  • The goal of treatment with Nplate is to keep the platelet count about 50,000 per microliter in order to lower the risk for bleeding 2.

From the Research

Definition and Treatment of ITP

• Immune thrombocytopenia (ITP) is an autoimmune disease affecting blood platelets that causes thrombocytopenia and an increased risk of bleeding 3.
• First-line therapy for ITP is indicated for patients with bleeding complications or who are at increased risk of bleeding, and the decision to initiate therapy depends on the platelet count and other endpoints including quality of life 3.
• The choice of first-line therapy depends primarily on how quickly a platelet count response is required, with intravenous immune globulin providing the more rapid response, followed by high-dose dexamethasone and prednisone 3.

First-Line Treatment Options

• High-dose dexamethasone (HDD) and conventional prednisone are two common first-line treatment options for adult patients with primary ITP 4.
• Initial responses to HDD and prednisone are similar, but patients in the HDD group respond earlier than the prednisone group 4.
• The sustained response at 6 months is lower in the HDD group than in the prednisone group, but the overall duration of response is longer in the prednisone group throughout the follow-up period 4.

Second-Line Treatment Options

• Romiplostim and eltrombopag are two relatively new drugs that have been shown to be successful in ITP treatment after standard treatment failure 5.
• These two medications are recommended as a second-line treatment for ITP, with romiplostim appearing to have slightly fewer adverse effects and higher costs 5.
• Switching between thrombopoietin receptor agonists (TRAs) is a successful way to overcome adverse effects and inadequacy according to the currently available literature 5.

Combination Therapy

• Simultaneous use of romiplostin, eltrombopag, and prednisone can be successful in severe thrombocytopenia of Evans syndrome refractory to other treatments 6.
• Combination therapy may be more reasonable than sequential monotherapies in some cases 6.

Ranking of Therapeutic Options

• A systematic review and network meta-analysis ranked romiplostim as the most suitable treatment for adults with previously treated ITP, followed by avatrombopag, eltrombopag, fostamatinib, and rituximab 7.
• Avatrombopag produced more satisfactory outcomes than romiplostim, eltrombopag, and rituximab in terms of early response, while severe adverse events profiles were similar across all treatment arms 7.