Estimated prophylactic dose required to achieve 3% trough as a function of age and concentrate class in multi‐country severe WAPPS‐Hemo haemophilia patients

AbstractIntroductionWeb‐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.AimTo 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.MethodsPK 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.ResultsAverage 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.ConclusionBaseline usage patterns from 2017 to early 2023 provide a benchmark for assessing the impact of emerging technologies in haemophilia.

Estimated prophylactic dose required to achieve 3% trough as a function of age and concentrate class in multi‐country severe WAPPS‐Hemo haemophilia patients Journal Articles