Recombinant factor IX Fc for the treatment of hemophilia B

Rolf Ljung, Davide Matino, Amy D Shapiro

Research output: Contribution to journalReview articlepeer-review

Abstract

Current hemophilia B treatment guidelines recommend routine prophylaxis with factor IX (FIX) replacement products, tailored to maintain plasma activity at levels that will prevent bleeds. However, plasma FIX activity may not be the primary determinant or best indicator of hemostatic efficacy due to its extravascular distribution. FIX replacement therapy has evolved to include extended half-life (EHL) products that provide effective bleed protection when administered at intervals of 7 days or longer. rFIXFc is a recombinant fusion protein with an extended circulation time. rFIXFc has a biodistribution profile consistent with distribution into extravascular space, where it may support hemostasis at sites of vessel injury independent of circulating plasma activity levels. The safety and efficacy of rFIXFc prophylaxis is well established in adults, adolescents and children including previously untreated patients with hemophilia B, with substantial evidence from clinical trials and real-world clinical practice. This review describes the pharmacokinetic characteristics of rFIXFc, summarizes available safety and efficacy data, and evaluates the use of rFIXFc in special populations. Current hemophilia B treatment challenges, including target FIX plasma levels, perioperative use, and management of patients with comorbidities, are discussed together with the potential role of EHL products in the future treatment landscape of hemophilia B.

Original languageEnglish
JournalEuropean Journal of Haematology
DOIs
Publication statusE-pub ahead of print - 2024 Feb 18

Subject classification (UKÄ)

  • Hematology

Fingerprint

Dive into the research topics of 'Recombinant factor IX Fc for the treatment of hemophilia B'. Together they form a unique fingerprint.

Cite this