Abstract：
UNASSIGNED: Emicizumab is a bispecific monoclonal antibody that mimics the function of factor VIII by binding to factor IXa and factor X to achieve haemostasis in haemophilia A. The long half-life and subcutaneous mode of administration makes emicizumab a compelling treatment option for bleeding prophylaxis. There is still limited real-world data on its use and management considerations, especially during surgical procedures. The objective of the study is to describe the real-world experience of emicizumab in a cohort of adult and paediatric haemophilia A patients in Singapore, including its use in the periprocedural setting.
UNASSIGNED: This was an observational study conducted at the 2 main haemophilia treatment centres in Singapore. All haemophilia A patients who commenced treatment with emicizumab before 1 July 2022 were recruited.
UNASSIGNED: A total of 18 patients with haemophilia A were included in this study. Ten (55.6%) patients had active inhibitors. The median annual bleeding rate for all patients before emicizumab use was 4.5 events (interquartile range [IQR] 2.8-8.3) compared with 0 events (IQR 0-0) after emicizumab was commenced (P=0). There were no adverse events of venous or arterial thrombosis, thrombotic microangiopathy, or death. A total of 6 procedures in 5 patients were performed during the study period with no major bleeding complications.
UNASSIGNED: Emicizumab effectively protects against bleeding in haemophilia A patients with and without inhibitors, including in children less than 12 years old. More studies are required to address clinical nuances, such as periprocedural management and the role of immune tolerance in patients with inhibitors on emicizumab.
UNASSIGNED: This was an observational study conducted at the 2 main haemophilia treatment centres in Singapore. All haemophilia A patients who commenced treatment with emicizumab before 1 July 2022 were recruited.
UNASSIGNED: A total of 18 patients with haemophilia A were included in this study. Ten (55.6%) patients had active inhibitors. The median annual bleeding rate for all patients before emicizumab use was 4.5 events (interquartile range [IQR] 2.8-8.3) compared with 0 events (IQR 0-0) after emicizumab was commenced (P=0). There were no adverse events of venous or arterial thrombosis, thrombotic microangiopathy, or death. A total of 6 procedures in 5 patients were performed during the study period with no major bleeding complications.
UNASSIGNED: Emicizumab effectively protects against bleeding in haemophilia A patients with and without inhibitors, including in children less than 12 years old. More studies are required to address clinical nuances, such as periprocedural management and the role of immune tolerance in patients with inhibitors on emicizumab.
摘要：
■Emicizumab是一种双特异性单克隆抗体，通过与IXa因子和X因子结合来模拟VIII因子的功能，从而在血友病A中实现止血。长半衰期和皮下给药模式使emicizumab成为预防出血的令人信服的治疗选择。关于其使用和管理考虑的实际数据仍然有限，尤其是在手术过程中。该研究的目的是描述新加坡成人和儿科A型血友病患者队列中emicizumab的真实经历，包括其在围手术期设置中的使用。
■这是一项在新加坡2个主要血友病治疗中心进行的观察性研究。所有在2022年7月1日前开始使用埃米珠单抗治疗的A型血友病患者均被招募。
■本研究共纳入18例A型血友病患者。10例（55.6%）患者有活性抑制剂。使用emicizumab前所有患者的中位年出血率为4.5个事件（四分位距[IQR]2.8-8.3），而开始使用emicizumab后的0个事件（IQR0-0）（P=0）。没有静脉或动脉血栓形成的不良事件，血栓性微血管病,或死亡。在研究期间，共有5例患者进行了6次手术，无严重出血并发症。
■Emicizumab可有效防止有或没有抑制剂的A型血友病患者出血，包括12岁以下的儿童。需要更多的研究来解决临床上的细微差别，例如围手术期管理和免疫耐受在使用emicizumab抑制剂的患者中的作用。
■这是一项在新加坡2个主要血友病治疗中心进行的观察性研究。所有在2022年7月1日前开始使用埃米珠单抗治疗的A型血友病患者均被招募。
■本研究共纳入18例A型血友病患者。10例（55.6%）患者有活性抑制剂。使用emicizumab前所有患者的中位年出血率为4.5个事件（四分位距[IQR]2.8-8.3），而开始使用emicizumab后的0个事件（IQR0-0）（P=0）。没有静脉或动脉血栓形成的不良事件，血栓性微血管病,或死亡。在研究期间，共有5例患者进行了6次手术，无严重出血并发症。
■Emicizumab可有效防止有或没有抑制剂的A型血友病患者出血，包括12岁以下的儿童。需要更多的研究来解决临床上的细微差别，例如围手术期管理和免疫耐受在使用emicizumab抑制剂的患者中的作用。
