Journal
IMMUNOTHERAPY
Volume 10, Issue 1, Pages 9-25Publisher
FUTURE MEDICINE LTD
DOI: 10.2217/imt-2017-0097
Keywords
autoimmune; FcR; fostamatinib; immune thrombocytopenia; phagocytosis; signal transduction; spleen tyrosine kinase; Syk; targeted therapy; tyrosine kinase inhibitors
Categories
Funding
- Amgen
- GSK
- Novartis
- Rigel Pharmaceuticals, Inc.
- Baxter
- Alexion
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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.
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