What medications are available to treat thrombocytopenia (low platelet count)?

From the Guidelines

Thrombocytopenia can be treated with various medications, including corticosteroids, intravenous immunoglobulin (IVIG), thrombopoietin receptor agonists, and rituximab, with the choice of treatment depending on the underlying cause and severity of the condition. The most recent and highest quality study 1 suggests that first-line treatment often includes corticosteroids like prednisone, which can be effective in reducing immune system activity in immune thrombocytopenia (ITP). However, prolonged exposure to corticosteroids can trigger severe adverse events, and tapering and interruption of corticosteroids in favor of an early switch towards a second-line treatment such as thrombopoietin receptor agonists (TPO-RAs) can lead to substantially improved clinical benefits.

Some key points to consider when treating thrombocytopenia include:

• The use of corticosteroids, such as prednisone, as a first-line treatment for ITP, with a typical dose of 1-2 mg/kg/day for 2-4 weeks with gradual tapering 1
• The use of IVIG at 1 g/kg for 1-2 days to provide rapid but temporary platelet increases in more severe or refractory cases 1
• The use of TPO-RAs, such as eltrombopag (starting at 50 mg daily) or romiplostim (1-10 μg/kg weekly subcutaneous injection), to stimulate platelet production and effectively treat chronic ITP 1
• The use of rituximab (375 mg/m² weekly for 4 weeks) for persistent cases of ITP 1
• The importance of addressing the underlying cause of thrombocytopenia, such as stopping implicated medications, treating infections, or managing liver disease, and individualizing therapy based on bleeding risk, side effects, and patient preferences 1

Overall, the goal of treatment is to maintain safe platelet counts (above 30,000/μL) rather than normalizing counts, and to improve the patient's quality of life while minimizing the risk of adverse events.

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.

ALVAIZ is a prescription medicine used to treat: adults and children 6 years of age and older with low blood platelet counts due to persistent or chronic immune thrombocytopenia (ITP), when other medicines to treat ITP or surgery to remove the spleen have not worked well enough adults with low blood platelet counts due to chronic hepatitis C virus (HCV) infection before and during treatment with interferon. severe aplastic anemia (SAA) when other medicines to treat SAA have not worked well enough.

The medications available to treat thrombocytopenia (low platelet count) are:

• Romiplostim (Nplate): used to treat low blood platelet counts in adults and children with immune thrombocytopenia (ITP) 2
• Eltrombopag (ALVAIZ): used to treat low blood platelet counts in adults and children with persistent or chronic ITP, chronic hepatitis C virus infection, and severe aplastic anemia (SAA) 3

From the Research

Medications for Thrombocytopenia

The following medications are available to treat thrombocytopenia (low platelet count):

• Corticosteroids, such as prednisone and dexamethasone, which are used as first-line therapy to stop bleeding and raise the platelet count acutely in patients with newly diagnosed or newly relapsed disease 4, 5, 6, 7, 8
• Intravenous immunoglobulin (IVIG) and anti-RhD immunoglobulin, which are used as first-line therapy in case of corticosteroid refractoriness or the need of rapid platelet elevation 4, 5, 6, 7, 8
• Thrombopoietin receptor agonists, such as romiplostim and eltrombopag, which are used as second-line therapy to maintain a hemostatic platelet count in patients who failed first-line therapy 4, 5, 6, 7, 8
• Rituximab, which is used as second-line therapy with high response and relapse rates, although its use is based on small studies and it is not approved by leading medical agencies for this indication 4, 5, 6, 7, 8
• Fostamatinib, a tyrosine kinase inhibitor recently approved by the U.S. Food and Drug Administration, which may be useful in patients with disease unresponsive to standard therapies or in specific clinical circumstances 7, 8
• Older immunosuppressive agents, such as azathioprine, cyclophosphamide, cyclosporine, danazol, dapsone, mycophenolate mofetil, and the Vinca alkaloids, which may be useful in patients with disease unresponsive to standard therapies or in specific clinical circumstances 8

Treatment Approach

The treatment approach for thrombocytopenia should be tailored to the individual patient and clinical setting, taking into account the patient's platelet count, bleeding risk factors, and preference 4, 5, 6, 7, 8. The goal of treatment is to provide a hemostatic platelet count while minimizing treatment-related toxicity 4.