Clinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Waehringer Guertel 18-20, 1090, Vienna, Austria.
Department of Internal Medicine V-Haematology and Oncology, Medical University Innsbruck, Innsbruck, Austria.
Sci Rep. 2021 Jun 21;11(1):12967. doi: 10.1038/s41598-021-92245-5.
To prevent bleeding in severe haemophilia A [SHA, defined as factor VIII (FVIII) activity < 1%] regular prophylactic FVIII replacement therapy is required, and the benefits of factor products with extended half-life (EHL) over traditional standard half-life (SHL) are still being debated. We performed a multi-centre, retrospective cohort study of persons with SHA in Austria aiming to compare clinical outcomes and factor utilization in patients with SHA, who switched from prophylaxis with SHL to an EHL. Data were collected from haemophilia-specific patient diaries and medical records. Twenty male persons with SHA (median age: 32.5 years) were included. The most common reason for switching to the EHL was a high bleeding rate with SHL. Switch to rFVIII-Fc resulted in a significantly decreased annualized bleeding rate (ABR; median difference (IQR): - 0.3 (- 4.5-0); Wilcoxon signed-rank test for matched pairs: Z = - 2.7, p = 0.008) and number of prophylactic infusions per week (- 0.75 (- 1.0-0.0); Z = - 2.7, p = 0.007). Factor utilization was comparable to prior prophylaxis with SHL (0.0 (- 15.8-24.8) IU/kg/week; Z = - 0.4, p = 0.691). In summary, switch to EHL (rFVIII-Fc) was associated with an improved clinical outcome, reflected by ABR reduction, and less frequent infusions, without significantly higher factor usage.
为预防重度甲型血友病(SHA,定义为因子 VIII [FVIII]活性 < 1%)患者出血,需要进行常规预防性 FVIII 替代治疗,而具有延长半衰期(EHL)的因子产品相对于传统标准半衰期(SHL)的优势仍存在争议。我们对奥地利的 SHA 患者进行了一项多中心、回顾性队列研究,旨在比较从 SHL 预防性治疗转为 EHL 的 SHA 患者的临床结局和因子利用情况。数据来自血友病患者日记和病历。共纳入 20 名男性 SHA 患者(中位年龄:32.5 岁)。转为 EHL 的最常见原因是 SHL 时出血率较高。转为 rFVIII-Fc 后,年化出血率(ABR;中位数差值(IQR):-0.3 (-4.5-0);配对符号秩检验:Z=-2.7,p=0.008)和每周预防性输注次数(-0.75 (-1.0-0.0);Z=-2.7,p=0.007)显著降低。因子利用情况与 SHL 预防性治疗相当(0.0 (-15.8-24.8) IU/kg/周;Z=-0.4,p=0.691)。总之,转为 EHL(rFVIII-Fc)与 ABR 降低和输注频率降低相关,临床结局改善,而因子使用量没有显著增加。