Current factor IX replacement options for hemophilia B and the challenges ahead.

Massimo Franchini, Marco Zaffanello, Daniele Focosi

Expert opinion on pharmacotherapy 2023 Apr

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Therapy for hemophilia B is aimed at replacing the congenital deficiency of coagulation factor IX (FIX). For replacement therapy, several FIX concentrates derived from donated human plasma or engineered by recombinant DNA technology are currently commercially available. The use of these products is well established and permit patients a relatively normal life. To further improve treatment efficacy, recombinant FIX products with a prolonged half-life have been developed, allowing relaxed prophylactic dosing and reducing treatment burden. In this review, we explore the current FIX replacement options for hemophilia B patients by analyzing the outcomes of their main clinical trials. We cover advances in the FIX molecules with extended half-life (EHL). Published literature on products for replacement of hemophilia B was retrieved using PubMed with no temporal limits. The recent introduction of recombinant EHL FIX products has represented a major advance in the therapeutic management of hemophilia B patients, permitting both a reduction of treatment burden and improving patients' compliance to prophylaxis and, ultimately, quality of life.

Citation

Massimo Franchini, Marco Zaffanello, Daniele Focosi. Current factor IX replacement options for hemophilia B and the challenges ahead. Expert opinion on pharmacotherapy. 2023 Apr;24(6):729-736