A Global Cross-Sectional Database Study of Low Dose FVIII SHL Prophylaxis in Haemophilia A.

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2025-05-10 DOI:10.1111/hae.70061

Dagmar M Hajducek, Oceana Sinkovic, Pierre Chelle, Alfonso Iorio, Andrea Edginton

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引用次数: 0

Abstract

Introduction: 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.

Aims: 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_LDP) and GNI/UHCI for children (≤12 years) and adults; (iii) exploratory comparison of pharmacokinetics (PKs) across LDP/non-LDP.

Methods: Descriptive statistics/graphical summaries for (i) and (iii), mixed-effects logistic regression for (ii).

Results: 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_LDP 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.

Conclusion: Underrepresentation of low-resource countries in WAPPS-Hemo underscores the financial challenges in haemophilia treatment. The association between GNI/UHCI and P_LDP suggests cost-driven adoption of LDP in low-resource settings, especially in children. PK outcomes average similarities may facilitate LDP-usage in WAPPS-Hemo.

Trial registration: NCT02061072, NCT03533504 (ClinicalTrials.gov).

查看原文本刊更多论文

血友病A低剂量FVIII SHL预防的全球横断面数据库研究。

血友病治疗费用昂贵，只有25%的患者得到适当的护理。虽然不是最佳的，因子VIII （FVIII）低剂量预防（LDP）可以减少每年的关节出血率。了解FVIII的使用情况，通过可访问的网络人群药代动力学服务血友病（WAPPS-Hemo）平台收集，及其与人均国民总收入（GNI）和全民健康覆盖指数（UHCI）的关系，可以提供全球差异的见解。目的：通过提供以下数据，提供低收入国家FVIII使用情况的见解：(i) 2017-2023年WAPPS-Hemo地区FVIII使用情况、LDP患病率、GNI和UHCI的统计摘要；（ii）估计儿童（≤12岁）和成人的LDP概率（PLDP）与GNI/UHCI之间的关系；（iii） LDP/非LDP药代动力学（PKs）的探索性比较。方法：对(1)和(3)进行描述性统计/图形总结，对(2)进行混合效应逻辑回归。结果：来自6223名严重血友病患者（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。自民党的流行率每年都在上升，在儿童中达到7%，在成人中达到14%。在流行ldp的国家，儿童GNI较低。在儿童和成人中，PLDP均与GNI和UHCI呈负相关。除了受样本量限制的儿童血浆来源产品外，LDP状态下的PK结果相似。结论：资源匮乏国家在WAPPS-Hemo项目中的代表性不足凸显了血友病治疗的财政挑战。GNI/UHCI与PLDP之间的关联表明，在低资源环境中，特别是在儿童中，应采用成本驱动的LDP。PK结果的平均相似性可能有助于在WAPPS-Hemo中使用ldp。试验注册：NCT02061072， NCT03533504 （ClinicalTrials.gov）。

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来源期刊

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

期刊介绍： Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.