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.

摘要

简介：目前的指南建议使用标准半衰期或延长半衰期产品对缺少凝血因子 IX 的血友病 B 进行预防性治疗。延长半衰期产品的半衰期是前者的三到六倍，从而可以减少每周注射次数，因此可能会影响治疗依从性和生活质量。Albutrepenonacog alfa 是一种延长半衰期的凝血因子 IX 与重组人白蛋白的融合蛋白，适用于所有年龄段的 B 型血友病患者的按需和预防性出血治疗。

涵盖的领域：作者回顾了 albutrepenonacog alfa 的临床和药代动力学特征，以及有关谷浓度和真实世界证据的可用信息。鉴于市场上其他因子 IX 产品的可用性，提出了临床和药代动力学特征的间接比较。

专家意见：作者就哪些患者资料适合使用 albutrepenonacog alfa 进行预防性治疗，以及在剂量、给药方案和转换治疗方案方面对患者进行管理，发表了他们的专家意见。