Abstract：
BACKGROUND: Web-Accessible Population-Pharmacokinetic Service-Haemophilia (WAPPS-Hemo) data are available to study factor-concentrate usage, defined as the required weekly dose to achieve a 3% trough (WD3T), across standard and extended half-life (SHL/EHL) products.
OBJECTIVE: To provide baseline usage data including (i) differences across plasma-derived (pdSHL) versus recombinant (rSHL) products, (ii) SHL versus EHL, and (iii) effect of age and positive inhibitor history.
METHODS: PK profiles (n = 14,416 patients, 0.3-85.2 years) and linear mixed effects models were used to estimate usage versus age, controlling for significant factors, using 95% confidence intervals to perform comparisons across all ages and posthoc tests to assess the differences.
RESULTS: Average usage was significantly higher for pdSHL versus rSHL in patients with a positive inhibitor history (PIH; 1.9-2.5 times higher), for SHL versus EHL (4-10 times), and was significantly associated with age.
CONCLUSIONS: Baseline usage patterns from 2017 to early 2023 provide a benchmark for assessing the impact of emerging technologies in haemophilia.
OBJECTIVE: To provide baseline usage data including (i) differences across plasma-derived (pdSHL) versus recombinant (rSHL) products, (ii) SHL versus EHL, and (iii) effect of age and positive inhibitor history.
METHODS: PK profiles (n = 14,416 patients, 0.3-85.2 years) and linear mixed effects models were used to estimate usage versus age, controlling for significant factors, using 95% confidence intervals to perform comparisons across all ages and posthoc tests to assess the differences.
RESULTS: Average usage was significantly higher for pdSHL versus rSHL in patients with a positive inhibitor history (PIH; 1.9-2.5 times higher), for SHL versus EHL (4-10 times), and was significantly associated with age.
CONCLUSIONS: Baseline usage patterns from 2017 to early 2023 provide a benchmark for assessing the impact of emerging technologies in haemophilia.
摘要：
背景：网络可访问的人群-药代动力学服务-血友病（WAPPS-Hemo）数据可用于研究因子浓缩物的使用，定义为达到3%波谷(WD3T)所需的每周剂量，跨标准和延长半衰期（SHL/EHL）产品。
目的：提供基线使用数据，包括（i）血浆衍生产品（pdSHL）与重组产品（rSHL）之间的差异，(ii)SHL与EHL,和(iii)年龄和阳性抑制剂史的影响。
方法：PK概况（n=14,416例患者，0.3-85.2年)和线性混合效应模型用于估计使用量与年龄，控制重要因素，使用95%置信区间在所有年龄之间进行比较,并进行posthoc检验以评估差异.
结果：在抑制剂病史阳性的患者中，pdSHL的平均使用率明显高于rSHL（PIH；高1.9-2.5倍），对于SHL与EHL（4-10次），并且与年龄显著相关。
结论：2017年至2023年初的基线使用模式为评估新兴技术在血友病中的影响提供了基准。
目的：提供基线使用数据，包括（i）血浆衍生产品（pdSHL）与重组产品（rSHL）之间的差异，(ii)SHL与EHL,和(iii)年龄和阳性抑制剂史的影响。
方法：PK概况（n=14,416例患者，0.3-85.2年)和线性混合效应模型用于估计使用量与年龄，控制重要因素，使用95%置信区间在所有年龄之间进行比较,并进行posthoc检验以评估差异.
结果：在抑制剂病史阳性的患者中，pdSHL的平均使用率明显高于rSHL（PIH；高1.9-2.5倍），对于SHL与EHL（4-10次），并且与年龄显著相关。
结论：2017年至2023年初的基线使用模式为评估新兴技术在血友病中的影响提供了基准。
