Abstract
Eltrombopag (Revolade®), an orally active thrombopoietin receptor agonist, fills an important formerly unmet clinical need in patients with severe aplastic anaemia refractory to prior immunosuppressive therapy. In this patient population, treatment with eltrombopag for 12–16 weeks produced a haematological response in at least one cell lineage in 40 % of patients and extended treatment produced tri-lineage responses in nearly 41 % of the responders. In robust responders, stable haematological counts were maintained after eltrombopag discontinuation. Treatment with eltrombopag is generally well tolerated.
Similar content being viewed by others
References
Marsh JCW, Ball SE, Cavenagh J, et al. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009;147(1):43–70.
Desmond R, Townsley DM, Dumitriu B, et al. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014;123(12):1818–25.
Marsh JCW, Kulasekararaj AG. Management of the refractory aplastic anemia patient: what are the options? Hematol Am Soc Hematol Educ Prog. 2013;2013:87–94.
Killick SB, Bown N, Cavenagh J, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172(2):187–207.
Revolade (eltrombopag): summary of product characteristics. London: European Medicines Agency; 2016.
Promacta (eltrombopag) tablets: US prescribing information. Research Triangle Park (NC): GlaxoSmithKline; 2014.
Olnes MJ, Scheinberg P, Calvo KR, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012;367(1):11–9.
Erickson-Miller CL, Delorme E, Tian SS, et al. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009;27(2):424–30.
Jenkins JM, Williams D, Deng Y, et al. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood. 2007;109(11):4739–41.
Shida Y, Takahashi N, Nohda S, et al. Pharmacokinetics and pharmacodynamics of eltrombopag in healthy Japanese males. Jpn J Clin Pharmacol Ther. 2011;42(1):11–20.
Erhardt JA, Erickson-Miller CL, Aivado M, et al. Comparative analyses of the small molecule thrombopoietin receptor agonist eltrombopag and thrombopoietin on in vitro platelet function. Exp Hematol. 2009;37(9):1030–7.
Townsley DM, Dumitriu B, Scheinberg P, et al. Eltrombopag added to standard immunosuppression for aplastic anemia accelerates count recovery and increases response rates [abstract no. LBA2]. Blood. 2015;126(23).
Kuter DJ, Mufti GJ, Bain BJ, et al. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009;114(18):3748–56.
Maciejewski JP, Selleri C. Evolution of clonal cytogenetic abnormalities in aplastic anemia. Leuk Lymphoma. 2004;45(3):433–40.
Matthys G, Park JW, McGuire S, et al. Eltrombopag does not affect cardiac repolarization: results from a definitive QTc study in healthy subjects. Br J Clin Pharmacol. 2010;70(1):24–33.
Desmond R, Townsley DM, Dunbar C, et al. Eltrombopag in aplastic anaemia. Semin Hematol. 2015;52(1):31–7.
Marsh JCW, Mufti GJ. Eltrombopag: a stem cell cookie? Blood. 2014;123(12):1774–5.
McCormack PL. Eltrombopag: a review of its use in patients with severe aplastic anaemia. Drugs. 2015;75(5):525–31.
Acknowledgments
The review updated from Drugs 2015;75(5):525–31 [18], and was reviewed by: E. Angelucci, Ospedale Oncologico di Riferimento Regionale, Cagliari, Italy; Meral Beksac, Ankara University, Ankara, Turkey; R. Desmond, Tallaght Hospital, Dublin, Ireland; P. Scheinberg, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA. During the peer review process, the manufacturer of eltrombopag was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Funding
The preparation of this review was not supported by any external funding.
Conflict of interest
S. Dhillon and P. McCormack are salaried employees of Adis/Springer, are responsible for the article content and declare no conflicts of interest.
Rights and permissions
About this article
Cite this article
Dhillon, S., McCormack, P.L. Eltrombopag in severe aplastic anaemia: a guide to its use in the EU. Drugs Ther Perspect 32, 232–237 (2016). https://doi.org/10.1007/s40267-016-0301-x
Published:
Issue Date:
DOI: https://doi.org/10.1007/s40267-016-0301-x