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Review
. 2021 Nov;81(16):1905-1913.
doi: 10.1007/s40265-021-01613-y. Epub 2021 Oct 28.

Avatrombopag: A Review in Thrombocytopenia

Anthony Markham  1
Affiliations
Review

Avatrombopag: A Review in Thrombocytopenia

Anthony Markham. Drugs. 2021 Nov.

Erratum in

Abstract

Avatrombopag (Doptelet®) is an orally administered second generation thrombopoietin receptor agonist (TPO-RA) approved for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory or have an unsatisfactory response to other treatments, as well as for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo an invasive procedure. In phase III studies, avatrombopag was associated with a significantly greater platelet response than placebo in patients with chronic ITP, and was superior to placebo in reducing the requirement for platelet transfusion or rescue procedures for bleeding caused by surgery in patients with CLD with a platelet count < 50 × 109/L at baseline. Longer term data indicate that avatrombopag is associated with high durable response rates in ITP and may have corticosteroid-sparing effects. The drug was generally well tolerated in both indications. Avatrombopag thus represents a convenient and effective second-line treatment for patients with chronic ITP and can prevent bleeding events in patients with CLD scheduled to undergo a procedure, offering a useful alternative to other available treatments in both indications.

Plain language summary

Avatrombopag (Doptelet®) is an orally administered drug that mimics the natural compound (thrombopoietin) responsible for stimulating the production of platelets, an essential component of the clotting process that prevents excessive bleeding. Several conditions can cause reduced platelet levels (thrombocytopenia) to the point that intervention is needed to prevent excessive blood loss. Avatrombopag is approved for the treatment of primary chronic immune thrombocytopenia (ITP) and to prevent bleeding caused by surgery in patients with low platelet levels caused by chronic liver disease (CLD). Clinical trials in patients with ITP show that avatrombopag quickly increases platelet levels and that this increase is maintained in the longer term in many patients. Similarly, clinical trials in patients with low platelet levels because of CLD showed that giving avatrombopag prior to surgery reduced the need for platelet transfusions or rescue procedures for bleeding. Avatrombopag is thus a convenient and effective treatment for patients with chronic ITP and can prevent bleeding events in patients with CLD scheduled to undergo a procedure. In both indications avatrombopag offers a useful alternative to other available treatments.

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Conflict of interest statement

A. Markham is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Figures

Fig 1
Fig 1
Mechanism of action of avatrombopag
See this image and copyright information in PMC

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References

    1. Kuter D. MSD manual: professional version; overview of platelet disorders. 2020. https://www.msdmanuals.com/professional/hematology-and-oncology/thromboc.... Accessed 17 Sep 2021.
    1. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829–3866. doi: 10.1182/bloodadvances.2019000966. - DOI - PMC - PubMed
    1. Giannini EG. Review article: thrombocytopenia in chronic liver disease and pharmacologic treatment options. Aliment Pharmacol Ther. 2006;23(8):1055–1065. doi: 10.1111/j.1365-2036.2006.02889.x. - DOI - PubMed
    1. Qamar AA, Grace ND, Groszmann RJ, et al. Incidence, prevalence, and clinical significance of abnormal hematologic indices in compensated cirrhosis. Clin Gastroenterol Hepatol. 2009;7(6):689–695. doi: 10.1016/j.cgh.2009.02.021. - DOI - PMC - PubMed
    1. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780–3817. doi: 10.1182/bloodadvances.2019000812. - DOI - PMC - PubMed

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