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Long-term sustained response to fostamatinib in two patients with chronic refractory immune thrombocytopenia (ITP).
British Journal of Haematology ( IF 6.5 ) Pub Date : 2020-01-03 , DOI: 10.1111/bjh.16328
Eun-Ju Lee 1, 2, 3 , Marina Izak 1, 2, 3 , James B Bussel 1, 2, 3
Affiliation  

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.

中文翻译:

两名难治性免疫性血小板减少症(ITP)患者对福司他替尼的长期持续反应。

对现有治疗难以治疗的慢性免疫性血小板减少症(ITP)患者的护理可能具有很大的挑战性。口服Syk抑制剂Fostamatinib是FDA批准的最新ITP药物。3期临床试验表明,接受fostamatinib治疗的患者中有43%的患者总体反应良好,并且已有两年使用的报道。在本文中,我们报道了两名ITP历史悠久的患者,但对众多的一线,二线和三线疗法没有持续的反应,而对正在进行的福司他替尼的反应却延长了。这表明对其他药物无反应的患者可能对fostamatinib有反应,并且可以持续受益。
更新日期:2020-01-03
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