An updated evaluation of avatrombopag for the treatment of chronic immune thrombocytopenia

ABSTRACT

Introduction

Multiple agents are available for the management of chronic immune thrombocytopenia (ITP), including thrombopoietin-receptor agonists (TPO-RAs), rituximab, and fostamatinib. Although TPO-RAs are often selected as treatments for chronic ITP, when choosing between the TPO-RAs, clinicians must balance safety profile, dosing restrictions, and method of administration incorporating patient preference.

Areas covered

We provide an overview of the thrombopoietin receptor agonists with a particular focus on avatrombopag, the newest agent in this class. In phase II and III clinical trials, avatrombopag was shown to offer durable improvement in platelet counts. We also include recent real-world evidence describing avatrombopag effectiveness in patients with poor response to prior treatments (including other TPO-RAs).

Expert opinion

Compared with other TPO-RAs used to treat ITP, avatrombopag offers practical oral dosing with a single pill strength, does not require long-term dietary restrictions, and has no warning for hepatotoxicity. It is frequently effective after use of other TPO-RAs in ITP. The primary downside with avatrombopag use at present is the lack of longer-term safety data in ITP that presently exists for romiplostim and eltrombopag.

KEYWORDS:

• Avatrombopag
• immune thrombocytopenia
• ITP
• thrombopoietin receptor agonist
• thrombocytopenia
• TPO-RA

Article highlights

• Thrombopoietin receptor agonists (TPO-RAs) are an important class of medications utilized for treatment of chronic immune thrombocytopenia.

• Avatrombopag was recently approved for use in adults with ITP and became the third TPO-RA available for use in ITP.

• In Phase 2 and Phase 3 clinical trials comparing avatrombopag with placebo for treatment of ITP, avatrombopag demonstrated clinical efficacy in raising platelet counts, durability of response, and a favorable safety profile.

• Compared with other TPO-RAs, avatrombopag is administered orally, does not require dietary restrictions, and does not have a safety warning of hepatotoxicity.

• Avatrombopag can also be an effective treatment for patients who need to switch from romiplostim or eltrombopag due to poor clinical response, adverse effects, or patient preference.

Acknowledgments

H Al-Samkari is the recipient of the American Society of Hematology Scholar Award.

Declaration of interest

H Al-Samkari declares research funding (Agios, Dova/Sobi, Amgen); consultancy (Agios, Dova/Sobi, Forma, argenx, Rigel, Moderna, Novartis). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

A Song contributed to concept and design, data collection, creation of tables and figures, writing of the first draft of the manuscript, critical revision of the manuscript, and final approval. H Al-Samkari contributed to concept and design, creation of tables and figures, critical revision of the manuscript, and final approval. All authors agree for the final version of the manuscript to be published.

Additional information

Funding

This paper was not funded.