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

ABSTRACT

Introduction

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.

Areas covered

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.

Expert Opinion

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.

KEYWORDS:

• Hemophilia B
• FIX concentrates
• replacement therapy
• extended half-life
• prophylaxis

Article highlights

• Hemophilia B is a rare inherited bleeding disorder characterized by deficiency of the coagulation FIX.

• In hemophilia B patients, the clinical severity of bleeding tendency is related to the degree of FIX defect.

• The mainstay of treatment of hemophilia B is replacement therapy with FIX concentrates.

• Several FIX concentrates are currently available, from plasma-derived to recombinant FIX products with standard (SHL) or extended half-life (EHL).

• EHL recombinant FIX products currently represent the best therapeutic approach for hemophilia B, improving patients’ adherence to prophylaxis regimens and, ultimately, their quality of life.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

A reviewer on this manuscript has disclosed that they have served as PI, consultant and advisory board member for CSL, Novo Nordisk, and Sobi. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Supplemental data

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14656566.2023.2196012.

Additional information

Funding

This paper was not funded.