Dunn Amy, Dargaud Yesim, Abajas Yasmina, Carcao Manuel, Castaman Giancarlo, Giermasz Adam, Hermans Cédric, Jiménez-Yuste Victor, Lewandowska Magdalena, Mahlangu Johnny, Meeks Shannon, Miesbach Wolfgang, Recht Michael, Salinas Vanessa, Chrisentery-Singleton Tammuella, Bonzo Daniel, Mitchell Ian S, Wilkinson Thomas A, Young Guy
Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, Ohio, USA.
Université Lyon, Lyon, France.
Haemophilia. 2025 Jan;31(1):78-86. doi: 10.1111/hae.15109. Epub 2024 Dec 15.
Eptacog beta is a novel human recombinant FVIIa approved for use in the United States, European Union, United Kingdom and Mexico for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors (≥12 years). It is also indicated for perioperative care in the same patient population in Europe and the United Kingdom.
To assess the incidence of rebleeding and review treatment outcomes in subjects with haemophilia with inhibitors enrolled in the phase 3 PERSEPT 1 clinical trial.
To treat mild/moderate bleeding episodes (BEs), subjects administered an initial 75 or 225µg/kg dose of eptacog beta, followed (if necessary) by additional 75µg/kg doses at predefined intervals until bleed control. This analysis used subject-reported rebleeding to determine a rebleeding incidence for the first 24 h. Rebleeding through later timepoints was an exploratory, intention-to-treat analysis of bleed treatment data.
Four hundred and sixty-five BEs were analysed. Through 24 h, the proportion of rebleeds was 0% (initial 75µg/kg dose) and 0.5% (initial 225µg/kg dose). Through 48 h, the proportion of rebleeds was 3.2% (75µg/kg initial dose) and 5.6% (225µg/kg initial dose); the difference between initial dose strategies was not statistically significant. The majority of rebleeds were controlled with a single dose of eptacog beta and no subject who treated a rebleed required hospitalization.
Subjects with haemophilia with inhibitors who used eptacog beta to treat mild/moderate BEs experienced a low incidence of rebleeding. Rebleeds that did occur were effectively controlled with eptacog beta (median, one dose) without the need for hospitalization.
eptacog beta是一种新型人重组FVIIa，已在美国、欧盟、英国和墨西哥获批，用于治疗和控制患有抑制剂（≥12岁）的A型或B型血友病患者的出血。在欧洲和英国，它也适用于同一患者群体的围手术期护理。
评估参加3期PERSEPT 1临床试验的患有抑制剂的血友病患者再次出血的发生率，并回顾治疗结果。
为治疗轻度/中度出血事件（BEs），受试者最初给予75或225μg/kg剂量的eptacog beta，必要时按预定间隔额外给予75μg/kg剂量，直至出血得到控制。该分析使用受试者报告的再次出血情况来确定前24小时的再次出血发生率。通过后续时间点进行的再次出血分析是对出血治疗数据的探索性意向性分析。
分析了46次出血事件。至24小时，再次出血的比例在初始剂量为75μg/kg时为0%，在初始剂量为225μg/kg时为0.5%。至48小时，再次出血的比例在初始剂量为75μg/kg时为3.2%，在初始剂量为225μg/kg时为5.6%；初始剂量策略之间的差异无统计学意义。大多数再次出血通过单剂量eptacog beta得到控制，且治疗再次出血的受试者均无需住院治疗。
使用eptacog beta治疗轻度/中度出血事件的患有抑制剂的血友病患者再次出血的发生率较低。确实发生的再次出血通过eptacog beta（中位数，一剂）得到有效控制，无需住院治疗。
