Expert opinion paper on the treatment of hemophilia B with albutrepenonacog alfa

Abstract

Introduction: Current guidelines recommend prophylactic treatment of hemophilia B with the missing coagulation factor IX, either with standard half-life or extended half-life products. Extended half-life products have half-lives three to six times longer than the former, allowing a reduction in the number of weekly injections and therefore, potentially impacting on treatment adherence and quality of life. Albutrepenonacog alfa is an extended half-life fusion protein of coagulation factor IX with recombinant human albumin, indicated for both on-demand and prophylactic treatment for bleeding in patients with hemophilia B of all ages. Areas covered: The authors review the clinical and pharmacokinetic characteristics of albutrepenonacog alfa, as well as the available information regarding trough levels and real-world evidence. Given the availability of other factor IX products in the market, indirect comparisons of clinical and pharmacokinetic characteristics are presented. Expert opinion: The authors exhibit their expert opinion on which patient profiles are candidates for prophylactic treatment with albutrepenonacog alfa, and on the management of patients in terms of dosing, regimens of administration and protocols for switching the treatment.

Prophylaxis in children with haemophilia in an evolving treatment landscape

“…PEGylated products are not licensed in Europe for children younger than 12 years, so at present rFIXFc or rIX–FP can be given in this age range. We should bear in mind, however, that the pharmacokinetics of each EHL FIX molecule differ significantly due to the differences in extravascular distribution, which impact, for example, terminal half‐life 36,37 . This difference may have potential clinical importance when choosing the respective derivate, measuring its effect and discussing with parents and/or care givers.…”

Albumin-Fusion Recombinant FIX in the Management of People with Hemophilia B: An Evidence-Based Review

“…There are several abstracts presented at the World Congresses in recent years have described real-world data, as highlighted by Álvarez Román et al 17 in their review. In the first, dated 2018, Escobar et al 18 reported the data on 145 PWH-B treated with different rFIX concentrates and obtained from the US healthcare providers (hematologists and Hemophilia Treatment Centers).…”