From the Guidelines
For adults with newly diagnosed Immune Thrombocytopenia (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 increase in platelet count is desired. This recommendation is based on the American Society of Hematology 2019 guidelines for ITP, which suggests that both prednisone and dexamethasone are effective for initial therapy, but dexamethasone may have a more rapid effect on platelet count response 1.
Key Considerations in ITP Management
- ITP is an acquired autoimmune disorder characterized by low platelet counts due to immune-mediated platelet destruction and impaired production, with an incidence of 2 to 5 per 100,000 and variable clinical symptoms 1.
- The likelihood of spontaneous remission from ITP is age-related, with higher remission rates in children than in adults, and natural history data in adults are less well-studied 1.
- Bleeding events in ITP can be unpredictable, and while some patients may not exhibit bleeding beyond bruising and petechiae, others may experience more serious mucosal bleeding, including menorrhagia, epistaxis, gastrointestinal hemorrhage, hematuria, or rarely, intracranial hemorrhage (ICH) 1.
- ITP has 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.
Treatment Approach
- First-line treatment typically involves corticosteroids, with the choice between prednisone and dexamethasone depending on the need for a rapid increase in platelet count 1.
- For patients requiring immediate platelet increase, intravenous immunoglobulin (IVIG) at 1 g/kg for 1-2 days can be added to corticosteroid therapy.
- Second-line options include thrombopoietin receptor agonists (TPO-RAs) like eltrombopag or romiplostim, rituximab, or splenectomy for refractory cases, with the choice of therapy individualized based on bleeding risk, platelet count, comorbidities, and patient preferences 1.
Patient Management
- Patients with ITP should avoid activities with high bleeding risk, limit NSAID use, and seek immediate medical attention for signs of significant bleeding.
- Treatment decisions should consider the patient's overall health status, including comorbidities, medications, and age, which all impact the risk of bleeding.
- The management approach may vary based on disease duration, access to care, quality-of-life implications, and patient and provider preferences, among other factors 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 with Nplate: Nplate is used to treat adults and children 1 year of age and older with immune thrombocytopenia (ITP) who have had an insufficient response to other treatments, such as medicines or splenectomy. The goal of Nplate treatment is to increase platelet counts to reduce the risk of bleeding.
- Key Points:
- Nplate is used for ITP treatment in adults and children 1 year of age and older.
- It is used when other treatments have not worked well enough.
- The goal is to increase platelet counts and reduce bleeding risk.
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 not only on the platelet count, but also on 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
- Corticosteroids, such as prednisone, are a common first-line treatment for ITP 4, 5.
- High-dose dexamethasone (HDD) has been shown to provide a more rapid response than conventional prednisone, but with a lower sustained response at 6 months 4.
- Intravenous immune globulin (IVIG) is also used as a first-line treatment, particularly in cases where a rapid response is needed 3, 5.
Second-Line Treatment Options
- Romiplostim and eltrombopag are two thrombopoietin receptor agonists (TRAs) that have been shown to be effective as second-line treatments for ITP 6.
- These medications have been shown to be safe and effective, with romiplostim having slightly fewer adverse effects and higher costs 6.
- Splenectomy and rituximab are also used as second-line treatments for ITP, particularly in cases where first-line treatments have failed 5.
Special Considerations
- The presence of anti-GPIb-IX autoantibodies has been shown to be a predictive factor for a poor initial response to corticosteroids therapy 4.
- Combined treatments, such as the use of prednisone, eltrombopag, and romiplostin, may be effective in severe cases of ITP that are refractory to other treatments 7.