What is the management for severe thrombocytopenia?

From the Guidelines

The management of severe thrombocytopenia requires prompt intervention with first-line treatment typically involving corticosteroids such as prednisone at 1-2 mg/kg/day for 2-4 weeks with subsequent tapering, as recommended by the most recent study 1.

Treatment Approach

The treatment approach depends on the underlying cause of thrombocytopenia, with immune thrombocytopenia (ITP) managed differently than thrombocytopenia due to bone marrow failure.

• For ITP, corticosteroids are the standard initial treatment, and they may also reduce bleeding by means of a direct effect on blood vessels, as stated in 1.
• Second-line options include thrombopoietin receptor agonists like eltrombopag or romiplostim, rituximab, or immunosuppressants such as azathioprine or mycophenolate mofetil, as discussed in 1 and 1.

Management of Refractoriness to Platelet Transfusion

For patients with alloimmune refractory thrombocytopenia, the management involves platelet transfusions from donors who are HLA-A and HLA-B antigen selected, as recommended in 1.

• The transfusion of HLA-matched platelets results in adequate increments in approximately 50% to 60% of transfusion events.

Quality of Life Considerations

The treatment approach should prioritize the patient's quality of life, avoiding prolonged exposure to corticosteroids, which can trigger severe adverse events, as highlighted in 1.

• Patients should avoid medications that affect platelet function (NSAIDs, aspirin) and activities with high bleeding risk.

Recent Recommendations

Recent studies recommend tapering and discontinuation of thrombopoietin receptor agonists in immune thrombocytopenia, with a focus on real-world recommendations, as discussed in 1.

• Initial corticosteroid treatment should be administered for no longer than 6-8 weeks, and excessively fast tapering should be avoided, as recommended in 1.

From the FDA Drug Label

In patients with chronic hepatitis C, ALVAIZ in combination with interferon and ribavirin may increase the risk of hepatic decompensation [see Warnings and Precautions (5. 1)]. ALVAIZ may increase the risk of severe and potentially life-threatening hepatotoxicity. Monitor hepatic function and discontinue dosing as recommended [see Warnings and Precautions (5.2)]. 1 INDICATIONS AND USAGE

1. 1 Treatment of Thrombocytopenia in Patients with Persistent or Chronic Immune Thrombocytopenia ALVAIZ® (eltrombopag tablets) are 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 ALVAIZ should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

The management for severe thrombocytopenia includes the use of eltrombopag (ALVAIZ), which is 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 initial dose regimen for adult and pediatric patients 6 years and older with ITP is 36 mg orally once daily, except in patients who are of East-/Southeast-Asian ancestry or who have mild to severe hepatic impairment (Child-Pugh Class A, B, C)
• Dose adjustments are based upon the platelet count response
• Romiplostim (Nplate) is also used for the treatment of thrombocytopenia in adult patients with ITP, with a recommended dose of 1 mcg/kg subcutaneously once weekly, with individual dose adjustments to maintain platelet counts (50 × 10^9/L to 200 × 10^9/L) 2 3.

From the Research

Management of Severe Thrombocytopenia

The management of severe thrombocytopenia involves several steps, including:

• Excluding pseudothrombocytopenia by collecting blood in a tube containing heparin or sodium citrate and repeating the platelet count 4
• Distinguishing acute from chronic thrombocytopenia by obtaining or reviewing previous platelet counts 4
• Identifying the underlying cause of thrombocytopenia, such as heparin-induced thrombocytopenia, thrombotic microangiopathies, or immune thrombocytopenia 4, 5, 6
• Transfusing platelets in patients with active hemorrhage or platelet counts less than 10 × 10^3 per μL 4
• Treating the underlying cause of thrombocytopenia, such as discontinuing the offending drug or administering immunosuppressive therapy 4, 6

Treatment Options

Treatment options for severe thrombocytopenia include:

• Platelet transfusions to increase platelet counts and reduce the risk of bleeding 4
• Corticosteroids to reduce inflammation and increase platelet production 7, 8
• Immunoglobulins to reduce platelet destruction and increase platelet production 8
• Thrombopoietin receptor agonists, such as romiplostim, to stimulate platelet production 8
• Rituximab to reduce platelet destruction and increase platelet production 7

Activity Restrictions

Patients with severe thrombocytopenia should adhere to activity restrictions to avoid trauma-associated bleeding, including:

• Avoiding contact sports and other high-risk activities 4
• Avoiding heavy lifting and bending 4
• Avoiding activities that may cause trauma, such as riding a bicycle or playing rough sports 4

Monitoring and Follow-up

Patients with severe thrombocytopenia should be monitored closely for signs of bleeding and platelet count should be checked regularly 4, 6. Regular monitoring of ADAMTS13 activity in remission and administration of rituximab when ADAMTS13 activity is less than 20% reduces risk of relapse 7.