What is the initial approach to treating thrombocytopenia?

Initial Approach to Treating Thrombocytopenia

The initial approach to treating thrombocytopenia should focus on determining the cause while simultaneously assessing bleeding risk, with corticosteroids being the standard first-line therapy for immune thrombocytopenia (ITP) when treatment is indicated. 1, 2

Diagnostic Workup

• Diagnosis of thrombocytopenia is primarily based on history, physical examination, complete blood count, and peripheral blood smear examination to exclude other causes 2
• Testing for HCV and HIV is strongly recommended for all patients with suspected ITP (grade 1B) 1, 2
• A bone marrow examination is not necessary for patients presenting with typical ITP (grade 2C) 1, 2
• Further investigations are only suggested if there are abnormalities other than thrombocytopenia in the blood count or smear 2
• Screening for H. pylori should be considered in patients with ITP in whom eradication therapy would be used if testing is positive (grade 2C) 1, 2

Treatment Indications

• Treatment is rarely indicated in patients with platelet counts above 50 × 10^9/L unless they have bleeding due to platelet dysfunction, trauma, surgery, comorbidities for bleeding, or require anticoagulation therapy 1
• Treatment is indicated for patients with platelet counts <10,000/μL regardless of symptoms 2, 3
• Treatment is indicated for patients with platelet counts <20,000/μL with significant mucous membrane bleeding 2, 3
• Patients with severe, life-threatening bleeding require immediate intervention regardless of platelet count 2

First-Line Treatment Options

Corticosteroids

• Prednisone is the standard initial first-line therapy for ITP patients, usually given at 0.5 to 2 mg/kg/d until the platelet count increases (30-50 × 10^9/L) 1
• To avoid corticosteroid-related complications, prednisone should be rapidly tapered and usually stopped in responders, and especially in non-responders after 4 weeks 1
• Dexamethasone 40 mg/day for 4 days (equivalent to 400 mg of prednisone per day) is an alternative that has shown high initial response rates and substantial sustained response rates 1

Intravenous Immunoglobulin (IVIG)

• IVIG should be used with corticosteroids when a more rapid increase in platelet count is required (grade 2B) 1, 2
• IVIG should be administered at an initial dose of 1 g/kg as a one-time dose; this dosage may be repeated if necessary (grade 2B) 1, 2
• IVIG or anti-D (in appropriate Rh-positive, non-splenectomized patients) should be used as first-line treatment if corticosteroids are contraindicated (grade 2C) 1, 2

Management of Severe or Life-Threatening Bleeding

• Patients with severe, life-threatening bleeding should receive high-dose parenteral glucocorticoid therapy 2
• These patients should also receive IVIG 2
• Platelet transfusions should be administered despite their limited effectiveness due to ongoing destruction 2, 3
• Hospitalization is appropriate for patients with platelet counts <20,000/μL who have significant mucous membrane bleeding 2, 3

Management of Secondary ITP

• For HCV-associated ITP, antiviral therapy should be considered in the absence of contraindications (grade 2C); if ITP treatment is required, the initial treatment should be IVIG (grade 2C) 1, 2
• For HIV-associated ITP, treatment of the HIV infection with antiviral therapy should be considered before other treatment options unless the patient has clinically significant bleeding complications (grade 1A) 1, 2
• Eradication therapy should be administered for patients who are found to have H. pylori infection (grade 1B) 1, 2

Common Pitfalls and Caveats

• Avoid prolonged corticosteroid use as this can lead to significant complications; prednisone should be tapered and stopped in responders, and especially in non-responders after 4 weeks 1
• Remember that thrombocytopenia does not protect against thrombosis; antithrombotic therapy should not be withheld because of thrombocytopenia alone when clinically indicated 4
• Some conditions can present with both bleeding and thrombosis, such as antiphospholipid syndrome, heparin-induced thrombocytopenia, and thrombotic microangiopathies 3
• Platelet transfusions should be used judiciously and are recommended only when patients have active hemorrhage or when platelet counts are less than 10 × 10^3 per μL 3

Second-Line Treatment Considerations

• For patients who fail first-line therapy, options include:
  • Thrombopoietin receptor agonists (TPO-RAs) like eltrombopag or romiplostim for patients who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy 5, 6, 5
  • Splenectomy, which provides a high initial response rate (85%) but carries risks of surgical complications and long-term adverse effects 1
  • Rituximab, which may be considered for patients at risk of bleeding who have failed one line of therapy 2