Fostamatinib in chronic immune thrombocytopenia: a profile of its use in the USA
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Oral fostamatinib is an orally administered small molecule spleen tyrosine kinase (SYK) inhibitor approved for the treatment of adults with chronic immune thrombocytopenia (ITP) who have an inadequate response to a previous treatment. Fostamatinib has a unique mechanism of action, whereby its active metabolite targets the SYK-mediated pathway of platelet destruction. In clinical trials, fostamatinib provided durable responses in adults with chronic ITP who had not responded or had relapsed following treatment with one or more prior ITP therapies, including corticosteroids, thrombopoietin receptor agonists, rituximab, and/or splenectomy. Most patients who respond to fostamatinib maintain platelet counts of > 50 × 109/L for periods of ≥ 12 months. The most common adverse events reported with fostamatinib in clinical trials were diarrhea, hypertension, nausea, and increased transaminase levels.
The manuscript was reviewed by: W. Homenda, Wojewódzki Szpital Specjalistyczny im. J. Korczaka i Akademia Pomorska w Słupsku, Slupsk, Poland; A. A. Khalafallah, Menzies Institute for Medical Research and Faculty of Health Sciences, University of Tasmania, Launceston, TAS, Australia. During the peer review process, Rigel Pharmaceuticals Inc., the marketing-authorization holder of fostamatinib, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.
Compliance with ethical standards
The preparation of this review was not supported by any external funding.
Conflict of interest
K. McKeage and K.A. Lyseng-Williamson are employees of Adis/Springer, are responsible for the article content and declare no conflicts of interest.
- 5.Terrell DR, Beebe LA, Vesely SK, et al. The incidence of immune thrombocytopenic purpura in children and adults: a critical review of published reports. Am J Hematol. 2010;85(3):174–80.Google Scholar
- 8.Tavalisse™ (fostamatinib disodium hexahydrate): US prescribing information. South San Francisco: Rigel Pharmaceuticals Inc. 2018.Google Scholar
- 11.Bussel JB, Arnold DM, Cooper N, et al. Long-term maintenance of platelet responses in adult patients with persistent/chronic immune thrombocytopenia treated with fostamatinib: 1-year efficacy and safety results [abstract]. Blood. 2017;130(Suppl 1):16.Google Scholar