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Immunotherapy. 2018 Jan;10(1):9-25. doi: 10.2217/imt-2017-0097. Epub 2017 Oct 2.

Fostamatinib for persistent/chronic adult immune thrombocytopenia.

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Academic Haematology Unit, Blizard Institute, Barts & The London School of Medicine & Dentistry, Queen Mary University of London, London, UK.
Division of Hematology/Oncology, Department of Medicine, Weill Cornell Medicine, New York, NY, USA.
Department of Haematology, The Royal London Hospital, Barts Health NHS Trust, London, UK.
Division of Pediatric Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA.


Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by phagocytosis and destruction of autoantibody-coated platelets via spleen tyrosine kinase (Syk)-mediated signal transduction in macrophages. Effectiveness of existing therapies varies, and even leading treatments (e.g., IVIg, splenectomy, rituximab, thrombopoietic agents) do not provide optimal management for a substantial number of patients with chronic ITP. Fostamatinib disodium is an orally-bioavailable investigational agent being developed for treatment of primary persistent/chronic adult ITP. Fostamatinib inhibits FcR-triggered, Syk-dependent cytoskeletal rearrangement during phagocytosis. Promising findings have been described in several autoimmune diseases, including rheumatoid arthritis, and sustained responses with manageable adverse events observed with ongoing treatment in patients with heavily treated chronic ITP. Fostamatinib represents an active therapy targeting a previously unexplored mechanism of ITP pathogenesis.


FcR; Syk; autoimmune; fostamatinib; immune thrombocytopenia; phagocytosis; signal transduction; spleen tyrosine kinase; targeted therapy; tyrosine kinase inhibitors

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