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

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_LDP) 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_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.

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.

NCT02061072, NCT03533504 (ClinicalTrials.gov).