关键词: albumin-fusion proteins albutrepenonacog-alfa extended half-life concentrates hemophilia B recombinant FIX

Mesh : Adult Child Factor IX / therapeutic use Half-Life Hemophilia B / drug therapy Humans Quality of Life Recombinant Fusion Proteins / adverse effects Serum Albumin, Human

来  源:   DOI:10.2147/DDDT.S236788   PDF(Pubmed)

Abstract:
Albutrepenonacog-alfa (Idelvion®, CSL Behring) is a recombinant fusion protein in which the recombinant FIX (rFIX) links a recombinant human albumin, extending the half-life of rFIX even beyond 100 hours. In 2016, this drug was approved worldwide for the treatment of pediatric and adult persons with hemophilia B (PWH-B). Its efficacy and safety were described in the PROLONG-9FP program and subsequently confirmed in the real-world practice, even if to date there are not many manuscripts that extensively and completely deal with the use of albutrepenonacog-alfa in daily practice, also evaluating its impact on the quality of life of patients treated with this drug; this review therefore aims to analyze all the publications currently available regarding the real-world use of this extended half-life concentrate, also noting which topics need further study and research.
摘要:
Albtrepenonacog-alfa(Idelvion®,CSLBehring)是一种重组融合蛋白,其中重组FIX(rFIX)连接重组人白蛋白,延长rFIX的半衰期甚至超过100小时。2016年,该药物在全球范围内被批准用于治疗儿童和成人血友病B(PWH-B)。其有效性和安全性在PROLONG-9FP计划中进行了描述,随后在现实世界的实践中得到了证实。即使到目前为止,在日常实践中广泛而全面地处理abulpenronacog-alfa的手稿并不多,还评估其对使用该药物治疗的患者的生活质量的影响;因此,这篇综述旨在分析目前可用的关于这种半衰期延长浓缩物的真实世界使用的所有出版物,还指出哪些主题需要进一步研究和研究。
公众号