艾美赛珠单抗预防治疗中国幼儿甲型血友病的真实世界经验：回顾性数据
Real-world experience of emicizumab prophylaxis in young children with hemophilia A: retrospective data from China.
作者信息
Liu Guoqing, Huang Kun, Li Gang, Zhen Yingzi, Li Zhengping, Chen Zhenping, Wu Runhui
机构信息
Hematology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Hematologic Disease Laboratory, Hematology Center, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Healt, Beijingh, China.
出版信息
Front Pediatr. 2022 Oct 19;10:992267. doi: 10.3389/fped.2022.992267. eCollection 2022.
BACKGROUND
As a new non-factor therapy for hemophilia A (HA), real-world study of emicizumab is still scarce. This study aimed to investigate the real-world use of emicizumab in Chinese boys with HA.
METHODS
Patients with moderate or severe HA were enrolled at Beijing Children's Hospital. They take emicizumab weekly (3 mg/kg) for a month and then went into a maintenance period with a different dosing regimen. After obtaining platelet-poor plasma at end of the loading period and during the maintenance period, coagulation ability and FVIII inhibitor were determined using human and bovine chromogenic Bethesda assay. Patients' bleeding rates were calculated through patients' records from 24 weeks before to at least 6 months after the switch (to emicizumab).
RESULT
In total, 13 pediatric patients with HA (severe: moderate = 11:2) were enrolled in this study. The patients' age was 3.51 (0.73-6.65) years. Eight had FVIII inhibitors at enrollment and one of them developed FVIII inhibitors again during the switch. The coagulation level of the maintenance period was 19.6 (13.5-32.8) IU/dL ( = 10). The median dose of each emicizumab injection was 2.7 (1.3-3.8) mg/kg and the monthly consumption of emicizumab was 5.2 (3.2-6.8) mg/kg/month. After switching to emicizumab, reduced annualized bleeding rate (ABR) [0.5 (0-4) vs. 4 (0-18), < 0.01], annualized joint bleeding rate (AJBR) [0 (0-1.1) vs. 1.0 (0-12), < 0.01], and annualized spontaneous bleeding rate (ASBR) [0 (0-1) vs. 2.0 (0-18), < 0.01] were observed. In patients with or without FVIII inhibitor, similar ABR [0.33 (0-4) vs. 0.5 (0-3), = 0.78], AJBR [0 (0-1.1) vs. 0 (0-0.5), = 0.63], and ASBR [0 (0-1) vs. 0 (0-1.5), = 0.73] were also noticed. Five inhibitor-positive patients (at enrollment) all had their inhibitor titer reduced. In addition, all target joints vanished after switching to emicizumab.
CONCLUSION
Emicizumab could reduce bleeds in pediatric patients with/without FVIII inhibitors and eliminate target joints.
背景
作为一种治疗甲型血友病（HA）的新型非因子疗法，emicizumab的真实世界研究仍然较少。本研究旨在调查emicizumab在中国HA男孩中的真实世界应用情况。
方法
在北京儿童医院招募中重度HA患者。他们每周服用emicizumab（3mg/kg），持续一个月，然后进入维持期，采用不同的给药方案。在负荷期结束时和维持期采集乏血小板血浆后，使用人和牛的发色底物贝塞斯达试验测定凝血能力和FVIII抑制剂。通过患者从转换（至emicizumab）前24周至少至转换后6个月的记录计算患者的出血率。
结果
本研究共纳入13例HA儿科患者（重度：中度=11:2）。患者年龄为3.51（0.73 - 6.65）岁。8例患者在入组时有FVIII抑制剂，其中1例在转换期间再次出现FVIII抑制剂。维持期的凝血水平为19.6（13.5 - 32.8）IU/dL（n = 10）。每次emicizumab注射的中位剂量为2.7（1.3 - 3.8）mg/kg，emicizumab的月消耗量为5.2（3.2 - 6.8）mg/kg/月。转换为emicizumab后，年化出血率（ABR）降低[0.5（0 - 4）对4（0 - 18），P < 0.01]，年化关节出血率（AJBR）降低[0（0 - 1.1）对1.0（0 - 12），P < 0.01]，年化自发性出血率（ASBR）降低[0（0 - 1）对2.0（0 - 18），P < 0.01]。在有或无FVIII抑制剂的患者中，也观察到类似的ABR[0.33（0 - 4）对0.5（0 - 3），P = 0.78]、AJBR[0（0 - 1.1）对0（0 - 0.5），P = 0.63]和ASBR[0（0 - 1）对0（0 - 1.5），P = 0.73]。5例抑制剂阳性患者（入组时）的抑制剂滴度均降低。此外，转换为emicizumab后，所有靶关节均消失。
结论
Emicizumab可减少有/无FVIII抑制剂的儿科患者的出血，并消除靶关节。
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