Hajducek Dagmar M, Sinkovic Oceana, Chelle Pierre, Iorio Alfonso, Edginton Andrea
School of Pharmacy, University of Waterloo, Waterloo, Ontario, Canada.
Faculty of Health Sciences, McMaster University, Waterloo, Ontario, Canada.
Haemophilia. 2025 Jul;31(4):646-656. doi: 10.1111/hae.70061. Epub 2025 May 10.
Haemophilia treatment is costly and only 25% of patients receive adequate care. Although not optimal, Factor VIII (FVIII) low-dose prophylaxis (LDP) may reduce annual joint bleeding rates. Understanding FVIII usage, collected through the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) platform, and its association with Gross National Income per capita (GNI) and Universal Health Coverage index (UHCI) may provide insights in global disparities.
To provide insights in FVIII use to advocate for LDP in low-income countries by providing: (i) statistical summary of FVIII usage, LDP prevalence, GNI and UHCI in WAPPS-Hemo in 2017-2023; (ii) estimation of the relationship between LDP probability (P) and GNI/UHCI for children (≤12 years) and adults; (iii) exploratory comparison of pharmacokinetics (PKs) across LDP/non-LDP.
Descriptive statistics/graphical summaries for (i) and (iii), mixed-effects logistic regression for (ii).
Data from 6223 severe haemophilia patients (ages 0.1-92 years) showed that 18% and 32% of countries used ≤1% LDP infusions in children and adults. LDP prevalence rose annually, peaking at 7% for children and 14% for adults. GNI was found lower in LDP-prevalent countries in children. In both children and adults, P demonstrated an inverse association with GNI and UHCI. PK outcomes were similar across LDP status, except potentially for plasma-derived products in children, however limited by sample size.
Underrepresentation of low-resource countries in WAPPS-Hemo underscores the financial challenges in haemophilia treatment. The association between GNI/UHCI and P suggests cost-driven adoption of LDP in low-resource settings, especially in children. PK outcomes average similarities may facilitate LDP-usage in WAPPS-Hemo.
NCT02061072, NCT03533504 (ClinicalTrials.gov).
血友病治疗费用高昂，仅有25%的患者能得到充分治疗。尽管并非最佳方案，但低剂量因子VIII（FVIII）预防治疗（LDP）可能会降低年度关节出血率。通过网络可及人群药代动力学服务 - 血友病（WAPPS - Hemo）平台收集的FVIII使用情况及其与人均国民总收入（GNI）和全民健康覆盖指数（UHCI）的关联，可能有助于了解全球差异。
通过提供以下内容，深入了解FVIII的使用情况，以倡导低收入国家采用LDP：（i）2017 - 2023年WAPPS - Hemo中FVIII使用情况、LDP患病率、GNI和UHCI的统计摘要；（ii）儿童（≤12岁）和成人中LDP概率（P）与GNI/UHCI之间关系的估计；（iii）LDP/非LDP之间药代动力学（PK）的探索性比较。
对（i）和（iii）进行描述性统计/图形摘要，对（ii）进行混合效应逻辑回归。
来自6223例重度血友病患者（年龄0.1 - 92岁）的数据显示，18%和32%的国家在儿童和成人中使用≤1%的LDP输注。LDP患病率逐年上升，儿童达到7%，成人达到14%时达到峰值。在儿童中，LDP患病率较高的国家GNI较低。在儿童和成人中，P均与GNI和UHCI呈负相关。除儿童中血浆源性产品可能存在差异外，LDP状态下的PK结果相似，但受样本量限制。
低资源国家在WAPPS - Hemo中的代表性不足，凸显了血友病治疗的经济挑战。GNI/UHCI与P之间的关联表明，在低资源环境中，尤其是儿童中，LDP的采用受成本驱动。PK结果平均相似性可能有助于在WAPPS - Hemo中使用LDP。
NCT02061072，NCT03533504（ClinicalTrials.gov）
