Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors
Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors
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DOI：
10.1111/hae.13877
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发表时间：
2019-11-20
期刊：
影响因子：
3.9
通讯作者：
Ragni, Margaret V.
中科院分区：
文献类型：
--
作者：
Ebbert, Patrick T.;Xavier, Frederico;Ragni, Margaret V.
Emicizumab是一种双特异性单克隆抗体，通过与因子IXa和X结合来模拟因子VIII（FVIII），以促进A型血友病（HA）和具有抑制剂的HA（HA-I）的止血。由于emicizumab与FVIII在生物化学上不同，因此人们对其真实世界的止血疗效感兴趣。目的通过回顾性图表审查描述emicizumab的真实患者经验。方法：我们回顾了在西PA血友病中心接受治疗的患者的病历，这些患者在获得许可后开始使用emicizumab，并且可以获得出血事件和因子使用情况。组间比较通过连续数据的Student t检验和离散数据的卡方或Fisher精确检验进行。结果42例患者中HA组18例（42.9%），HA-I组24例（52.1%）。各组年龄相似，17例（40.5%）
Introduction Emicizumab is a bispecific monoclonal antibody that mimics factor VIII (FVIII) by binding to factors IXa and X to promote hemostasis in haemophilia A (HA) and HA with inhibitors (HA-I). As emicizumab differs biochemically from FVIII, there is interest in its real-world haemostatic efficacy. Aim To describe real-world patient experience with emicizumab by retrospective chart review. Methods We reviewed medical records of patients cared for at the Hemophilia Center of Western PA, who initiated emicizumab following its licensure, and on whom bleeding events and factor use were available. Comparisons between groups were done by Student's t test for continuous data and by chi-square or Fisher's exact test for discrete data. Results A total of 42 patients whose charts were reviewed included 18 (42.9%) with HA and 24 (52.1%) with HA-I. Groups were similar in age, 17 (40.5%)