What are the treatment options for thrombocytopenia (low platelet count)?

From the Guidelines

The treatment of thrombocytopenia should prioritize the use of thrombopoietin receptor agonists, such as eltrombopag or romiplostim, for persistent immune thrombocytopenia (ITP), as they have been shown to be effective in increasing platelet counts and reducing bleeding events 1. When managing thrombocytopenia, it is essential to consider the underlying cause, severity, and the patient's risk of bleeding. For ITP, first-line therapy typically includes corticosteroids, such as prednisone or dexamethasone, and intravenous immunoglobulin (IVIG) for rapid platelet increase in emergency situations. However, for persistent ITP, second-line treatment options, including thrombopoietin receptor agonists, rituximab, and splenectomy, should be considered. Some key points to consider when treating thrombocytopenia include:

• The use of antifibrinolytic agents, such as aminocaproic acid and tranexamic acid, may be considered as an adjunct treatment for bleeding in thrombocytopenic patients, although their efficacy is unproven 1.
• Recombinant factor VIIa (rfVIIa) has been used in some cases of ITP, but its use is associated with a risk of thrombosis and should be used with caution 1.
• Emergent splenectomy may be considered in life-threatening bleeding, but it should be regarded as a heroic measure due to the associated risks 1.
• The goal of treatment is to increase platelet counts to safe levels while addressing the primary cause of thrombocytopenia, and the choice of treatment should be guided by the underlying mechanism of platelet destruction or decreased production. In terms of specific treatment options, the following may be considered:
• Thrombopoietin receptor agonists, such as eltrombopag (starting at 50 mg daily) or romiplostim (1 μg/kg weekly, adjusted based on response), for persistent ITP 1.
• Rituximab (375 mg/m² weekly for 4 weeks) for refractory cases of ITP.
• Splenectomy for patients who fail medical management.
• Discontinuation of the offending medication for drug-induced thrombocytopenia, and immediate cessation of heparin and switching to direct thrombin inhibitors for heparin-induced thrombocytopenia.
• Plasma exchange therapy for thrombotic thrombocytopenic purpura, often with adjunctive corticosteroids.

From the FDA Drug Label

Nplate is indicated for the treatment of thrombocytopenia in: Adult patients 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. 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.

Treatment of Thrombocytopenia

• The drug romiplostim (Nplate) is indicated for the treatment of thrombocytopenia in adult and pediatric patients with immune thrombocytopenia (ITP) who have had an insufficient response to other treatments.
• The recommended dose is the lowest dose to achieve and maintain a platelet count ≥ 50 × 10^9/L.
• Dose adjustments are based on platelet count response and should not exceed a maximum weekly dose of 10 mcg/kg 2.

From the Research

Treatment Options for Thrombocytopenia

• The primary goal of treatment is to achieve a hemostatic platelet count (> 20-30 × 10(9) L(-1)) while minimizing treatment-related toxicity 3
• First-line therapy for immune thrombocytopenia (ITP) typically includes corticosteroids, intravenous immune globulin G, or anti-Rh(D) 3, 4, 5, 6
• Second-line treatment options for ITP include splenectomy, rituximab, and thrombopoietin receptor agonists (such as romiplostim and eltrombopag) 3, 4, 5, 6
• Novel drugs, such as fostamatinib, are also being explored as potential treatment options for ITP 5, 6

Management of Thrombocytopenia

• Patients with a platelet count greater than 50 × 10(3) per μL are generally asymptomatic, while those with platelet counts between 20 and 50 × 10(3) per μL may experience mild skin manifestations 7
• Patients with platelet counts of less than 10 × 10(3) per μL are at high risk of serious bleeding and may require hospitalization 7
• Transfusion of platelets is recommended for patients with active hemorrhage or platelet counts less than 10 × 10(3) per μL, in addition to treatment of underlying causative conditions 7
• Activity restrictions are recommended for patients with platelet counts of less than 50 × 10(3) per μL to avoid trauma-associated bleeding 7

Diagnosis and Evaluation

• The diagnosis of ITP is based on the exclusion of other causes of isolated thrombocytopenia using patient history, physical examination, blood count, and evaluation of the peripheral blood film 5
• The clinical treatment goals should be to resolve bleeding events and prevent severe bleeding episodes, with a target platelet count of > 20-30 × 10(9) L(-1) 5
• Evaluation of thrombocytopenia should include distinguishing between acute and chronic thrombocytopenia, as well as identifying underlying causative conditions 7