Sjoerd F Koopman, Tine M H J Goedhart, Laura H Bukkems, Trevor M Mulders, Frank W G Leebeek, Karin Fijnvandraat, Michiel Coppens, Mary Mathias, Peter W Collins, R Campbell Tait, Catherine N Bagot, Nicola Curry, Jeanette Payne, Pratima Chowdary, Marjon H Cnossen, Ron A A Mathôt
BACKGROUND: Recombinant factor IX Fc fusion protein(rFIX-Fc) is an extended half-life (EHL) factor concentrate administered to haemophilia B patients. So far, a population pharmacokinetic (PK) model has only been published for patients ≥12 years of age. AIM: Externally evaluate the predictive performance of the published rFIX-Fc population PK model for patients of all ages and develop a model that describes rFIX-Fc PK using real world data. METHODS: We collected prospective and retrospective data from patients with haemophilia B (FIX activity level ≤5 IU/dL) treated with rFIX-Fc and included in the OPTI-CLOT TARGET study (NTR7523) or United Kingdom (UK)-EHL Outcome Registry (NCT02938156)...
August 11, 2023: British Journal of Clinical Pharmacology