What is the management of thrombocytopenia (low platelet count)?

From the Guidelines

Management of low platelets (thrombocytopenia) should prioritize treatment based on the severity, cause, and presence of bleeding, with a focus on increasing platelet production, decreasing platelet destruction, or replacing platelets directly through transfusion, as recommended by the American Society of Hematology 2019 guidelines for immune thrombocytopenia 1.

Key Considerations

• For mild thrombocytopenia (>50,000/μL) without bleeding, observation may be sufficient.
• For moderate to severe cases (<50,000/μL) or when bleeding is present, treatment should address the underlying cause.
• Common interventions include stopping medications that cause thrombocytopenia, treating infections, or managing autoimmune conditions.

Treatment Options

• For immune thrombocytopenia (ITP), first-line treatment includes corticosteroids such as prednisone (1 mg/kg/day for 2-4 weeks followed by tapering) or dexamethasone (40 mg daily for 4 days) 1.
• Intravenous immunoglobulin (IVIG) at 1 g/kg for 1-2 days can provide rapid but temporary platelet increase in emergency situations.
• For refractory cases, thrombopoietin receptor agonists like eltrombopag (starting at 50 mg daily) or romiplostim (1 μg/kg weekly subcutaneously) may be used.

Platelet Transfusions

• Platelet transfusions are generally reserved for active bleeding or counts below 10,000/μL, or before procedures 1.
• Patients should avoid activities with high bleeding risk and medications that affect platelet function (NSAIDs, aspirin).

Recent Guidelines

• The American Society of Clinical Oncology clinical practice guideline update in 2018 recommends prophylactic platelet transfusion for patients with thrombocytopenia resulting from impaired bone marrow function to reduce the risk of hemorrhage when the platelet count falls below a predefined threshold level 1.
• The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia recommends treatment based on the presence of bleeding rather than the platelet count alone 1.

From the FDA Drug Label

2 DOSAGE AND ADMINISTRATION

2.1 Patients with Immune Thrombocytopenia (ITP) Use the lowest dose of Nplate to achieve and maintain a platelet count ≥ 50 × 10^9/L as necessary to reduce the risk for bleeding.

INDICATIONS AND USAGE

ALVAIZ is a thrombopoietin receptor agonist indicated: for the treatment of thrombocytopenia in adult and pediatric patients 6 years and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy

The management of low platelet count involves the use of thrombopoietin receptor agonists such as romiplostim (Nplate) and eltrombopag (ALVAIZ).

• The goal is to achieve and maintain a platelet count ≥ 50 × 10^9/L to reduce the risk of bleeding.
• Romiplostim (Nplate) is administered as a weekly subcutaneous injection, with dose adjustments based on platelet count response.
• Eltrombopag (ALVAIZ) is taken orally once daily, with dose adjustments to maintain a platelet count ≥ 50 × 10^9/L.
• It is essential to monitor platelet counts regularly and adjust the dose accordingly to minimize the risk of bleeding or thrombotic complications 2, 3.

From the Research

Low Platelet Management Overview

• Low platelet count, also known as thrombocytopenia, is a condition characterized by an increased risk of bleeding due to decreased platelet counts 4.
• Primary immune thrombocytopenia (ITP) is an immune-mediated bleeding disorder that results in accelerated platelet destruction and suppressed platelet production 4.

Treatment Options

• Initial treatments for newly diagnosed ITP in adults include corticosteroids, intravenous immunoglobulin, and anti-RhD immunoglobulin, but these agents generally cannot induce a long-term response in most patients 4.
• Subsequent treatments for patients who fail initial therapy include thrombopoietic agents, rituximab, fostamatinib, splenectomy, and several older immunosuppressive agents 4.
• Romiplostim and eltrombopag are two relatively new drugs that have been shown to be successful in ITP treatment after standard treatment failure, and are recommended as a second-line treatment 5.
• A systematic review and network meta-analysis found that romiplostim appears to be the most suitable treatment in terms of overall response, followed by avatrombopag, eltrombopag, fostamatinib, and rituximab 6.

Combination Therapies

• Simultaneous use of romiplostin, eltrombopag, and prednisone was successful in severe thrombocytopenia of Evans syndrome refractory to other treatments, suggesting that combined treatments may be more effective than sequential monotherapies 7.
• Upfront treatment strategies for newly diagnosed patients with ITP aim to enhance remission rates and prevent relapse, and may involve alteration or intensification of first-line therapy 8.

Treatment Goals

• The goal of treatment is to elevate platelet counts to a safe level with minimal toxicity and improve patient health-related quality of life, and should be tailored to the individual patient and disease phase 4.
• Treatment decisions should take into account the risks and benefits of each approach, and consider factors such as the desire to increase platelet count, induce remission, and improve health-related quality of life 8.