Platelets floating above a damaged liver.

Avatrombopag: A Game-Changer for Thrombocytopenia in Adults with Chronic Liver Disease?

"Discover how this new treatment is transforming the management of low platelet counts in patients undergoing procedures in the USA."


For adults battling chronic liver disease, low platelet counts (thrombocytopenia) can turn routine medical procedures into high-stakes events. The risk of bleeding escalates, often leading to delays, cancellations, and the need for platelet transfusions, which themselves carry risks. This challenging landscape has long awaited a solution—and it may have arrived.

Enter Avatrombopag (Doptelet®), the first thrombopoietin receptor agonist approved in the USA specifically to address thrombocytopenia in adults with chronic liver disease scheduled for a procedure. This medication represents a significant step forward, offering a way to reduce the need for transfusions and improve the safety of these essential interventions.

This article dives into the details of Avatrombopag, exploring how it works, its benefits, potential side effects, and how it's changing the landscape of care for patients with chronic liver disease. We'll break down the science in an accessible way, providing you with the information you need to understand this important new treatment option.

How Does Avatrombopag Work to Boost Platelet Counts?

Platelets floating above a damaged liver.

Avatrombopag stimulates the production of platelets by targeting megakaryocytes, the cells in bone marrow responsible for platelet formation. It mimics the action of thrombopoietin, a key growth factor that regulates platelet production. By activating thrombopoietin receptors, Avatrombopag effectively boosts platelet counts, reducing the risk of bleeding during and after medical procedures.

The treatment regimen is straightforward: a once-daily oral dose for five consecutive days, starting 10 to 13 days before the scheduled procedure. The dosage (40 mg or 60 mg) depends on the patient's initial platelet count. This proactive approach aims to increase platelet levels to a safer range before the procedure takes place.

  • Increases platelet production by stimulating megakaryocytopoiesis.
  • Dosage (40 or 60 mg once daily × 5 days) is based on the patient's presurgery platelet count, with treatment starting 10-13 days prior to the scheduled procedure.
  • Reduces the need for platelet transfusion or rescue procedure after a scheduled procedure.
  • Has a tolerability profile similar to that of placebo.
Clinical trials (ADAPT-1 and ADAPT-2) have demonstrated Avatrombopag's effectiveness in reducing the need for platelet transfusions and rescue procedures. More patients receiving Avatrombopag achieved the target platelet count of >50×109/L on the day of their procedure compared to those receiving a placebo. This benefit was observed across various patient subgroups, regardless of age, sex, race, or severity of liver disease.

Avatrombopag: A Promising Step, But Not a Perfect Solution

Avatrombopag represents a significant advancement in managing thrombocytopenia for adults with chronic liver disease undergoing procedures. By stimulating platelet production, it reduces the need for transfusions and improves patient outcomes. However, like all medications, it's essential to consider potential risks and interactions.

While Avatrombopag is generally well-tolerated, it's crucial to be aware of potential side effects and the increased risk of thrombotic events in patients with known risk factors. A thorough evaluation by a healthcare professional is necessary to determine if Avatrombopag is the right choice for each individual.

Avatrombopag offers a valuable tool for managing thrombocytopenia in specific clinical settings. Its effectiveness and ease of use make it a favorable option for many patients, but careful patient selection and monitoring are essential to ensure the best possible outcomes.

About this Article -

This article was crafted using a human-AI hybrid and collaborative approach. AI assisted our team with initial drafting, research insights, identifying key questions, and image generation. Our human editors guided topic selection, defined the angle, structured the content, ensured factual accuracy and relevance, refined the tone, and conducted thorough editing to deliver helpful, high-quality information.See our About page for more information.

This article is based on research published under:

DOI-LINK: 10.1007/s40267-018-0593-0, Alternate LINK

Title: Avatrombopag In The Treatment Of Thrombocytopenia In Adults With Chronic Liver Disease Scheduled To Undergo A Procedure: A Profile Of Its Use In The Usa

Subject: Pharmacology (medical)

Journal: Drugs & Therapy Perspectives

Publisher: Springer Science and Business Media LLC

Authors: Emma H. Mccafferty, Katherine A. Lyseng-Williamson

Published: 2018-12-08

Everything You Need To Know

1

What is Avatrombopag (Doptelet®) and what does it do?

Avatrombopag, known under the brand name Doptelet®, is the first FDA-approved thrombopoietin receptor agonist specifically designed to treat thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a medical procedure. It helps reduce the risk of bleeding by increasing platelet counts.

2

How does Avatrombopag work to increase platelet counts in individuals with chronic liver disease?

Avatrombopag works by mimicking the effects of thrombopoietin, a key growth factor that regulates platelet production. It targets megakaryocytes in the bone marrow, stimulating them to produce more platelets. This helps to increase platelet counts to a safer level before a scheduled procedure, reducing the risk of bleeding.

3

What did clinical trials reveal about the effectiveness of Avatrombopag?

In clinical trials such as ADAPT-1 and ADAPT-2, Avatrombopag significantly reduced the need for platelet transfusions and rescue procedures in patients with chronic liver disease undergoing medical procedures. A greater proportion of patients treated with Avatrombopag achieved the target platelet count of greater than 50×109/L on the day of their procedure compared to those who received a placebo.

4

How is Avatrombopag administered to patients before a medical procedure?

Avatrombopag is administered orally once daily for five consecutive days, starting 10 to 13 days before the scheduled procedure. The dosage, either 40 mg or 60 mg, depends on the patient's initial platelet count. This proactive approach is designed to elevate platelet levels to a safer range prior to the intervention.

5

Is Avatrombopag a perfect solution for thrombocytopenia, and what are its limitations?

While Avatrombopag offers significant benefits in reducing the need for platelet transfusions and improving outcomes for adults with chronic liver disease and thrombocytopenia, it is not a perfect solution and it's crucial to consider potential risks and interactions. The tolerability profile of Avatrombopag is similar to that of a placebo. This highlights the importance of a comprehensive approach to managing thrombocytopenia, including careful patient selection and monitoring.

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