From the Guidelines
The treatment for Immune Thrombocytopenic Purpura (ITP) should prioritize corticosteroids as first-line therapy, with a recommended duration of no longer than 6-8 weeks, followed by a switch to second-line treatments such as thrombopoietin receptor agonists (TPO-RAs) like eltrombopag or romiplostim, which have shown improved clinical benefits and a safer profile compared to prolonged corticosteroid use 1.
First-Line Treatment
- Corticosteroids, including prednisone and high-dose dexamethasone, are commonly administered to rapidly achieve safe platelet counts and prevent bleeding events 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 the importance of switching to second-line treatments promptly 1.
Second-Line Treatment
- TPO-RAs, such as eltrombopag and romiplostim, have been shown to be highly effective in clinical trials, with initial responses observed after 1-2 weeks of treatment and significantly reducing bleeding events and emergency hospitalizations 1.
- Rituximab, a monoclonal antibody targeting CD-20, may also be considered for the treatment of ITP, especially in relapsed/refractory cases, but its use is associated with infusion-related toxicities and rare but lethal complications 1.
- Splenectomy remains an effective option for refractory cases, with approximately 60-70% of patients achieving long-term remission, but it is associated with an increased chance of relapse and short-term and long-term complications 1.
Quality of Life and Safety Considerations
- The introduction of TPO-RAs has a positive impact on the quality of life of ITP patients, and their long-term use does not affect bone marrow tolerability 1.
- Prolonged exposure to corticosteroids can trigger severe adverse events, such as weight gain, cataract, mood alterations, hypertension, and infections, highlighting the importance of switching to second-line treatments promptly 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 treatment for Immune Thrombocytopenic Purpura (ITP) is Nplate (romiplostim), which is used to treat low blood platelet counts in adults and children 1 year of age and older when certain medicines or surgery to remove the spleen have not worked well enough 2.
- Key points:
- Nplate is given by injection under the skin one time each week.
- The dose of Nplate is adjusted to maintain platelet counts between 50 × 10^9/L to 200 × 10^9/L.
- Patients receiving Nplate should be closely monitored for changes in platelet counts and for signs of bleeding.
- Important considerations:
- Nplate may increase the risk of blood clots.
- Patients with chronic liver disease may be at increased risk for blood clots in the veins of the liver.
From the Research
Treatment Options for Immune Thrombocytopenic Purpura (ITP)
The primary goal of treatment for ITP is to prevent serious bleeding by increasing platelet counts. The following are some of the treatment options available:
- Corticosteroids: Traditionally used as first-line therapy to increase platelet counts 3, 4, 5, 6, 7
- Splenectomy: May be considered for patients who do not respond to corticosteroids, as it can produce a long-lasting response in a majority of patients 3, 4, 6, 7
- Intravenous immunoglobulin (IVIG) or Rho(D) immune globulin (anti-RhD): Can be used to rapidly increase platelet counts, particularly before planned procedures 4, 5, 6
- Rituximab: A monoclonal antibody against the CD20 antigen, used as a second-line therapy for refractory or non-responsive ITP 4, 5
- Thrombopoietic agents: May be used to stimulate platelet production in patients who do not respond to other treatments 5
- Fostamatinib: A potential treatment option for patients with chronic refractory ITP 5
- Other treatments: Such as androgens, immunomodulatory drugs, cytotoxic chemotherapy, and bone marrow transplantation, may be considered in certain cases 7
Treatment Approach
The treatment approach for ITP should be tailored to the individual patient and disease phase, aiming to elevate platelet counts to a safe level while minimizing toxicity and improving quality of life 5. The decision to treat ITP should be based on the presence of bleeding or severe thrombocytopenia, rather than platelet count alone 6.