ArticleHaemophilia : the official journal of the World Federation of Hemophilia2025
A Global Cross-Sectional Database Study of Low Dose FVIII SHL Prophylaxis in Haemophilia A.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
The Development of the Web-based Application for the Population Pharmacokinetic Service - Hemophilia (WAPPS-Hemo) - Phase 1
Exploring Sources of Variability in Individual Pharmacokinetics of Factor VIII and IX Concentrates in Hemophilia in the Web Accessible Population Pharmacokinetics Service - Hemophilia (WAPPS-Hemo) Database
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5 authors.
Funding
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Abstract
introductionHaemophilia 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.
aimsTo 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
methodsDescriptive statistics/graphical summaries for (i) and (iii), mixed-effects logistic regression for (ii).
resultsData 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
conclusionUnderrepresentation of low-resource countries in WAPPS-Hemo underscores the financial challenges in haemophilia treatment. The association between GNI/UHCI and P
trial registrationNCT02061072, NCT03533504 (ClinicalTrials.gov).
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