■随着治疗景观的不断发展,血友病社区必须概述所有已发布的批准疗法数据,比如emicizumab,支持共同决策。
■为了将emicizumab在先天性血友病A患者中使用的临床和现实数据结合起来,不管年龄,疾病严重程度,或因子VIII抑制剂状态。重点领域是安全,功效,和生活质量(QoL)。
■此范围审查使用了引文数据库(PubMed,Embase,和Cochrane图书馆)和手动搜索抽象书籍。出版物报告了血友病A患者中emicizumab的原始数据,2014年12月后以英文发表,并纳入报告部分终点.这种叙事综合集中在零出血上,治疗年度出血率(ABR),不良事件,和QoL措施。
■总的来说,包括97种出版物(截止日期:2022年8月9日)。治疗ABR仍然很低(计算的平均和中位治疗ABR分别在0.7-1.3和0.0-1.4之间),零治疗出血患者的中位数百分比为66.7%。经历治疗相关不良事件的患者比例为0.0%至60.0%;大多数是注射部位反应。在37份报告安全性和招募超过2300名个人的出版物中,报告了11例血栓性事件和4例血栓性微血管病变。来自完善的工具的数据显示了使用美珠单抗的QoL益处。
这项范围审查整合了全球已发表的emicizumab在A型血友病患者中的应用经验,并支持了emicizumab具有可接受的安全性这一事实。在预防出血方面是有效的,并与QoL的改善有关。
UNASSIGNED: With the treatment landscape continually evolving, it is vital that the hemophilia community have an overview of all published data for approved therapies, such as
emicizumab, to support shared decision making.
UNASSIGNED: To bring together the clinical and real-world data for
emicizumab use in people with congenital hemophilia A, regardless of age, disease severity, or factor VIII inhibitor status. Key focus areas were safety, efficacy, and quality of life (QoL).
UNASSIGNED: This scoping review used citation databases (PubMed, Embase, and the Cochrane Library) and manual searches of abstract books. Publications reporting original data for
emicizumab in people with hemophilia A, published in English after December 2014, and reporting select endpoints were included. This narrative synthesis focused on zero bleeds, treated annualized bleeding rate (ABR), adverse events, and QoL measures.
UNASSIGNED: Overall, 97 publications were included (cut-off: August 9, 2022). Treated ABR remained low (calculated mean and median treated ABRs ranged between 0.7-1.3 and 0.0-1.4, respectively), and the median percentage of people with zero treated bleeds was 66.7%. The proportion of people experiencing treatment-related adverse events ranged from 0.0% to 60.0%; most were injection-site reactions. Across 37 publications reporting on safety and enrolling >2300 individuals, 11 thrombotic events and 4 thrombotic microangiopathies were reported. Data from well-established tools show QoL benefits with
emicizumab.
UNASSIGNED: This scoping review consolidates the global published experience for emicizumab in people with hemophilia A and supports the fact that
emicizumab has an acceptable safety profile, is effective and efficacious in bleed prevention, and is associated with improvements in QoL.