rIX-FP用于血友病B治疗的临床综合评价

A comprehensive clinical evaluation of rIX-FP for the treatment of hemophilia B

摘要

目的: 开展albutrepenonacog alfa(rIX-FP)治疗血友病B的临床综合评价。方法: 基于指南、文献及公开数据,按PICOS原则,从有效性、安全性、适宜性、创新性和经济性5个维度,将rIX-FP与血源性凝血因子IX(coagulation factor IX,FIX)、标准半衰期重组FIX(standard half-life recombinant factor IX,SHL rFIX)和延长半衰期重组FIX(extend half-life recombinant factor IX,EHL rFIX)进行比较分析。结果: 评价结果显示,有效性方面,rIX-FP在降低年化出血率、提高零出血患者比例及改善关节健康方面,相较于其他FIX制剂具有潜在优势,儿童亚组趋势一致。其安全性与其他重组FIX相当,未发现不可管理的安全风险。适宜性方面,提供5种规格,剂量选择灵活,无须特殊储存。创新性方面,依托白蛋白融合技术延长半衰期,减少给药频次,提升谷浓度。经济性方面,未在国内上市,国际数据显示长期治疗(≥3年)相较于SHL rFIX及rFIXFc具有成本优势。结论: 基于现有证据,rIX-FP在血友病B治疗中展现出一定的临床优势,但仍需开展头对头比较研究及本土化药物经济性评价。

Abstract

Objective: To conduct a comprehensive clinical evaluation of albutrepenonacog alfa (rIX-FP) for the treatment of hemophilia B. Methods: Based on guidelines, literature, and publicly available data, and following the PICOS principle, rIX-FP was compared with plasma-derived FIX, standard half-life recombinant FIX (SHL rFIX), and extended half-life recombinant FIX (EHL rFIX) across five dimensions: efficacy, safety, suitability, innovation, and economy. Results: The evaluation results indicate that rIX-FP has potential advantages over other FIX preparations in terms of reducing the annualized bleeding rate, increasing the proportion of patients with zero bleeding events, and improving joint health, with consistent trends observed in the pediatric subgroup. The safety profile of rIX-FP is comparable to that of other recombinant FIX preparations, with no unmanageable safety risks observed. Regarding suitability, rIX-FP is available in five dosage specifications, offers flexible dosing options, and requires no special storage conditions. In terms of innovation, it utilizes albumin fusion technology to extend the half-life, thereby reducing dosing frequency and increasing trough levels. As for economy, although not yet marketed in China, international data suggest that long-term treatment (≥3 years) with rIX-FP has a cost advantage compared to SHL rFIX and rFIXFc. Conclusion: Based on available evidence, rIX-FP demonstrates certain clinical advantages in the treatment of hemophilia B. However, head-to-head comparative studies and local pharmacoeconomic evaluations are still warranted.    

关键词

血友病B / albutrepenonacog alfa / 延长半衰期 / 凝血因子IX / 临床综合评价

Key words

hemophilia B / albutrepenonacog alfa / extended half-life / coagulation factor IX / clinical comprehensive evaluation

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基金资助

国家重点研发计划资助项目(2023YFC2706100);新发突发与重大传染病防控国家科技重大专项资助项目(2025ZD01904903)