Long‐term sustained response to fostamatinib in two patients with chronic refractory immune thrombocytopenia (ITP)

Summary Care of patients with chronic immune thrombocytopenia (ITP) who are refractory to available treatments can be quite challenging. Fostamatinib, an oral Syk inhibitor, is the newest FDA‐approved agent for ITP. Phase 3 clinical trials demonstrated an overall response in 43% of patients treated...

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Bibliographic Details
Published in:British journal of haematology 2020-04, Vol.189 (2), p.379-382
Main Authors: Lee, Eun‐Ju, Izak, Marina, Bussel, James B.
Format: Article
Language:English
Subjects:
Adult
Aged
Chronic Disease
Clinical trials
Female
Hematology
Humans
Idiopathic thrombocytopenic purpura
ITP
maintenance therapy
Oxazines - pharmacology
Oxazines - therapeutic use
platelets
Purpura, Thrombocytopenic, Idiopathic - drug therapy
Pyridines - pharmacology
Pyridines - therapeutic use
Syk inhibitor
Syk protein
Thrombocytopenia
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Summary:Summary Care of patients with chronic immune thrombocytopenia (ITP) who are refractory to available treatments can be quite challenging. Fostamatinib, an oral Syk inhibitor, is the newest FDA‐approved agent for ITP. Phase 3 clinical trials demonstrated an overall response in 43% of patients treated with fostamatinib and use for two years has been reported. Herein, we report two patients with long histories of ITP without lasting responses to numerous first‐, second‐ and third‐line therapies with prolonged responses to ongoing fostamatinib. This shows that patients unresponsive to other agents may respond to fostamatinib and can have sustained benefit.
ISSN:0007-1048
1365-2141
DOI:10.1111/bjh.16328