What is the treatment for thrombocytopenia (low platelet count)?

From the Guidelines

The treatment for thrombocytopenia should be individualized based on the underlying cause, severity, and presence of bleeding, with the goal of maintaining a safe platelet count to prevent serious bleeding complications, as recommended by the most recent guidelines 1.

Causes and Severity of Thrombocytopenia

The approach to treating thrombocytopenia varies significantly depending on its cause, which can range from impaired bone marrow production to increased platelet destruction or sequestration. For instance, in cases of immune thrombocytopenia (ITP), the treatment focuses on reducing immune-mediated platelet destruction, whereas in thrombocytopenia due to bone marrow failure, the approach may involve supporting bone marrow function or transfusing platelets.

Treatment Options

• Observation: For mild cases without bleeding, observation alone may be sufficient, especially if the platelet count is not severely low.
• Corticosteroids: First-line medication treatment often includes corticosteroids such as prednisone (typically 1-2 mg/kg/day for 2-4 weeks with gradual tapering) to reduce immune destruction of platelets, as supported by guidelines 1.
• Intravenous Immunoglobulin (IVIG): For ITP, IVIG at 1 g/kg for 1-2 days can provide rapid but temporary increases in platelet counts.
• Thrombopoietin Receptor Agonists: Like eltrombopag (25-75 mg daily) or romiplostim (1-10 μg/kg weekly subcutaneously), these stimulate platelet production and are effective for chronic ITP, with recommendations for their use found in recent studies 1.
• Rituximab: May be used in refractory cases of ITP.
• Platelet Transfusions: For severe bleeding, platelet transfusions may be necessary, typically given as single-donor apheresis units, with guidelines suggesting a threshold for transfusion based on the patient’s diagnosis, clinical condition, and treatment modality 1.
• Splenectomy: Remains an option for patients with ITP who fail medical therapy.

Recent Guidelines and Recommendations

Recent guidelines from the American Society of Clinical Oncology 1 and other studies 1 emphasize the importance of prophylactic platelet transfusions for patients with thrombocytopenia resulting from impaired bone marrow function to reduce the risk of hemorrhage. However, the threshold for transfusion varies according to the patient’s diagnosis, clinical condition, and treatment modality. For adult patients having minor invasive procedures, recommendations suggest prophylactic platelet transfusion for patients having elective central venous catheter placement with a platelet count less than 20 × 10^9 cells/L, based on low-quality evidence but with a weak recommendation for practice 1.

Considerations for Specific Patient Groups

• Patients with Liver Disease: The use of thrombopoietin receptor agonists like eltrombopag and lusutrombopag has been approved for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure, aiming to increase platelet counts to ≥50,000/μL before the procedure, as discussed in recent practice guidance 1.
• Drug-Induced Thrombocytopenia: Discontinuing the offending medication is essential.

In conclusion, the management of thrombocytopenia requires a tailored approach based on the underlying cause, severity of the condition, and the presence of bleeding, with the ultimate goal of preventing serious bleeding complications while minimizing the risks associated with treatments.

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 thrombocytopenia (low platelet count) is Nplate (romiplostim), which is used to treat:

• Adults with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy
• Pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy The goal of treatment with Nplate is to achieve and maintain a platelet count ≥ 50 × 10^9/L as necessary to reduce the risk for bleeding 2. Key points:
• The initial dose of Nplate is 1 mcg/kg
• The dose is adjusted based on platelet count response
• The maximum weekly dose is 10 mcg/kg
• Platelet counts and CBCs must be performed weekly during the dose adjustment phase and then monthly following establishment of a stable Nplate dose 2.

From the Research

Treatment for Thrombocytopenia

The treatment for thrombocytopenia (low platelet count) depends on the underlying cause and severity of the condition. Some key points to consider include:

• Transfusion of platelets is recommended when patients have active hemorrhage or when platelet counts are less than 10 × 10^3 per μL, in addition to treatment (when possible) of underlying causative conditions 3.
• Patients with platelet counts of less than 50 × 10^3 per μL should adhere to activity restrictions to avoid trauma-associated bleeding 3.
• Treatment options for immune thrombocytopenia (ITP) include corticosteroids, intravenous immunoglobulins, anti-D immunoglobulin, rituximab, thrombopoietin receptor agonists, immunosuppressants, and splenectomy 4.
• Romiplostim, a thrombopoietin receptor agonist, has been shown to be effective in treating ITP in patients with renal impairment and chronic HCV infection 5.

Management of Thrombocytopenia

Management of thrombocytopenia involves identifying the underlying cause and taking steps to prevent bleeding complications. Some key considerations include:

• Patients with severe thrombocytopenia are at high risk of bleeding and may require hospitalization 3.
• In patients with mild or moderately low platelet counts, additional factors such as platelet function defects, hyperfibrinolysis, invasive procedures, or catheters can increase the risk of bleeding 6.
• A slow and gradual fall in platelet counts may indicate consumptive coagulopathy or bone marrow failure, while an abrupt decrease in platelet counts may suggest immunologic causes such as heparin-induced thrombocytopenia or other drug-induced immune thrombocytopenia 6.

Emerging Therapies for ITP

Recent advances in the treatment of ITP include the development of novel agents targeting various mechanisms involved in the pathogenesis of the disease. Some of these emerging therapies include:

• Splenic tyrosine kinase inhibitor
• Bruton kinase inhibitor
• Plasma cell targeting therapies
• Neonatal Fc receptor inhibitor
• Platelet desialylation inhibitor
• Inhibition of the classical complement pathway 4.