Australian Haemophilia Centre Directors' Organisation, Melbourne, Australia.
Perth Children's Hospital, Perth, Australia.
Haemophilia. 2023 Sep;29(5):1283-1290. doi: 10.1111/hae.14842. Epub 2023 Aug 11.
Extended half-life (EHL) factor VIII and IX concentrates as prophylaxis against bleeds have been available to selected persons with haemophilia (PWH) in Australia since March 2018. Preliminary analysis of switching to EHL demonstrated increased treatment adherence, fewer injections and improved bleeding outcomes.
To characterise clinical practices regarding the use of EHL in Australia, to further evaluate treatment regimens and bleeding outcomes, and to analyse the influence of EHL product pharmacokinetics on clinical decision-making.
A national, retrospective study was conducted using the Australian Bleeding Disorders Registry (ABDR). Patients on EHL products during the entire 2019 calendar year were included for analysis.
A complete and validated dataset of 174 PWH was analysed, 115 Haemophilia A (HA) and 59 Haemophilia B (HB). Adherence to EHL therapy was 85.7% in HA and 87.2% in HB. About 63.5% of HA and 64.4% of HB PWH reported zero spontaneous bleeds over 12months. Ankles were the most frequent spontaneous bleed site. Approximately one-third patients underwent dose adjustments, with most frequent reasons being pharmacokinetics, body weight change and breakthrough bleeds. About 19.5% of PWH had target joint history, with spontaneous bleeds reported in 58% of that cohort on EHL. Multivariate regression showed significant impact of non-adherence, target joint history and short half-life on spontaneous bleeds in the HA cohort; however only short half-life had significant impact in the HB cohort.
EHL usage in Australia shows excellent treatment adherence and bleeding outcomes. This study affirms the use and value of widely available population-based pharmacokinetics as a clinical tool.
自 2018 年 3 月以来，澳大利亚为选定的血友病患者（PWH）提供了延长半衰期（EHL）VIII 和 IX 因子浓缩物作为预防出血的手段。初步分析表明，改用 EHL 可提高治疗依从性、减少注射次数并改善出血结局。
描述澳大利亚使用 EHL 的临床实践，进一步评估治疗方案和出血结局，并分析 EHL 产品药代动力学对临床决策的影响。
使用澳大利亚出血性疾病登记处（ABDR）进行了一项全国性、回顾性研究。纳入 2019 年全年使用 EHL 产品的患者进行分析。
分析了 174 名 PWH 的完整且经过验证的数据集，其中 115 名为血友病 A（HA），59 名为血友病 B（HB）。HA 中 EHL 治疗的依从性为 85.7%，HB 中为 87.2%。约 63.5%的 HA 和 64.4%的 HB PWH 在 12 个月内报告零自发性出血。踝关节是最常见的自发性出血部位。约三分之一的患者进行了剂量调整，最常见的原因是药代动力学、体重变化和突破性出血。约 19.5%的 PWH 有靶关节病史，在该队列中，58%的患者在使用 EHL 时有自发性出血。多变量回归显示，非依从性、靶关节病史和半衰期短对 HA 队列的自发性出血有显著影响；然而，半衰期短对 HB 队列有显著影响。
澳大利亚 EHL 的使用情况显示出极佳的治疗依从性和出血结局。本研究证实了广泛可用的基于人群的药代动力学作为临床工具的使用和价值。
