Oldenburg Johannes, Hay Charles, Peyvandi Flora, Tagliaferri Annarita, Holme Pål Andrè, Álvarez-Román María Teresa, Biron-Andréani Christine, Malmström Håkan, Bystrická Linda, Lethagen Stefan
Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany.
University Department of Haematology, Manchester Royal Infirmary, Manchester, UK.
Eur J Haematol. 2025 Feb;114(2):248-257. doi: 10.1111/ejh.14309. Epub 2024 Oct 21.
The 24-month, prospective, non-interventional, European multicenter A-SURE study evaluated the real-world effectiveness of prophylaxis using an extended half-life recombinant factor VIII (FVIII) Fc fusion protein, efmoroctocog alfa (hereinafter rFVIIIFc), compared with prophylaxis using standard half-life (SHL) FVIII products in patients with hemophilia A.
Primary endpoints were annualized bleeding rate (ABR), annualized injection frequency, and annualized factor consumption. A comparative study design unique for an observational hemophilia study was implemented to reduce potential confounding in effectiveness estimates, wherein each patient prescribed rFVIIIFc was matched with one receiving SHL FVIII. Propensity scores were used for adjustment in statistical analyses.
Outcomes for all primary endpoints were significantly better in the rFVIIIFc group (n = 184) compared with the SHL FVIII group (n = 170): mean ABR 1.5 versus 2.3 (difference of -0.8; p = 0.0147); mean annualized injection frequency 114.4 versus 169.2 (difference of -54.8; p < 0.0001); and mean annualized factor consumption 243 024.2 versus 288 718.6 International Units (difference of 45 694.5; p = 0.0003). rFVIIIFc was well tolerated, with no inhibitor development.
rFVIIIFc has superior prophylactic effectiveness versus SHL FVIII, providing higher bleed protection with fewer injections and lower factor consumption.
为期24个月的前瞻性、非干预性欧洲多中心A-SURE研究评估了与使用标准半衰期（SHL）FVIII产品进行预防相比，使用延长半衰期重组因子VIII（FVIII）Fc融合蛋白艾美罗凝血八因子（以下简称rFVIIIFc）对A型血友病患者进行预防的真实世界有效性。
主要终点为年化出血率（ABR）、年化注射频率和年化因子消耗量。实施了一项在血友病观察性研究中独特的比较研究设计，以减少有效性估计中的潜在混杂因素，其中每个开具rFVIIIFc处方的患者与一名接受SHL FVIII的患者进行匹配。倾向得分用于统计分析中的调整。
与SHL FVIII组（n = 170）相比，rFVIIIFc组（n = 184）所有主要终点的结果均显著更好：平均ABR为1.5对2.3（差值为-0.8；p = 0.0147）；平均年化注射频率为114.4对169.2（差值为-54.8；p < 0.0001）；平均年化因子消耗量为243024.2对288718.6国际单位（差值为45694.5；p = 0.0003）。rFVIIIFc耐受性良好，未出现抑制剂。
与SHL FVIII相比，rFVIIIFc具有更好的预防效果，通过更少的注射和更低的因子消耗提供更高的出血保护。
