Children's Blood and Cancer Centre, KK Women's and Children's Hospital, Singapore.
Department of Haematology, Singapore General Hospital, Singapore.
Ann Acad Med Singap. 2023 Nov 29;52(11):580-589. doi: 10.47102/annals-acadmedsg.2023100.
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.
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.
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.
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.
依库珠单抗是一种双特异性单克隆抗体，通过结合因子 IXa 和因子 X 模拟因子 VIII 的功能，从而实现血友病 A 的止血。依库珠单抗半衰期长且可皮下给药，这使其成为血友病 A 出血预防的一种极具吸引力的治疗选择。目前，关于依库珠单抗的使用和管理注意事项，尤其是在手术过程中的应用，仍缺乏真实世界的数据。本研究的目的是描述依库珠单抗在新加坡一组血友病 A 成年和儿科患者中的真实世界应用经验，包括在围手术期的应用。
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 日之前开始接受依库珠单抗治疗的血友病 A 患者均被招募入组。
本研究共纳入 18 例血友病 A 患者。其中 10 例（55.6%）患者存在活性抑制物。在使用依库珠单抗之前，所有患者的年平均出血率为 4.5 次（四分位距 [IQR] 2.8-8.3），而在开始使用依库珠单抗后则为 0 次（IQR 0-0）（P=0）。无静脉或动脉血栓形成、血栓性微血管病或死亡的不良事件。在研究期间，有 5 例患者共进行了 6 次手术，均无重大出血并发症。
依库珠单抗可有效预防有或无抑制物的血友病 A 患者出血，包括 12 岁以下的儿童。需要进一步研究来解决临床细节问题，如围手术期管理以及抑制物患者应用依库珠单抗的免疫耐受作用。
