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阿伐曲泊帕联合 fostamatinib 治疗多种难治性免疫性血小板减少症。

Avatrombopag plus fostamatinib combination as treatment in patients with multirefractory immune thrombocytopenia.

机构信息

Hematology Department, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBIS /CSIC), Universidad de Sevilla, Sevilla, Spain.

Hematology Department, Complejo Asistencial Universitario de Salamanca (CAUSA), Instituto de Investigación Biomédica de Salamanca (IBSAL), Universidad de Salamanca (USAL), Salamanca, Spain.

出版信息

Br J Haematol. 2024 Oct;205(4):1551-1555. doi: 10.1111/bjh.19602. Epub 2024 Jun 19.

Abstract

Immune thrombocytopenia (ITP) refractory to multiple therapies may require a combination of drugs targeting different mechanisms and targets. In this retrospective, multicentre, international study, we report the safety and effectiveness of avatrombopag and fostamatininb in combination administered to 18 patients with multirefractory ITP. Overall, the combination response was achieved in 15 patients (83.3%), with a median time from combination start to best response of 15 days (IQR: 8-35 days). After a median follow-up of 256 days (IQR: 142.8-319), 5 patients relapsed (26.7%), all during tapering or stopping one drug. Adverse events were described in 6 of 18 patients (33%).

摘要

免疫性血小板减少症(ITP)对多种治疗方法耐药可能需要联合针对不同机制和靶点的药物。在这项回顾性、多中心、国际研究中,我们报告了联合使用avatrombopag 和 fostamatininibb 治疗 18 例多药难治性 ITP 患者的安全性和有效性。总体而言,15 例患者(83.3%)达到了联合应答,从联合治疗开始到最佳应答的中位时间为 15 天(IQR:8-35 天)。中位随访 256 天(IQR:142.8-319)后,5 例患者复发(26.7%),均发生在减药或停药期间。18 例患者中有 6 例(33%)出现不良事件。

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