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A型血友病患者换用八因子(BAY 81-8973)的疗效和安全性:全球真实世界研究 TAURUS 的最终结果。

Efficacy and safety in patients with haemophilia A switching to octocog alfa (BAY 81-8973): Final results of the global real-world study, TAURUS.

机构信息

Hematology, University Hospital, Policlinico Umberto I, Rome, Italy.

East Carolina University, Greenville, North Carolina, USA.

出版信息

Eur J Haematol. 2023 Jan;110(1):77-87. doi: 10.1111/ejh.13876. Epub 2022 Oct 17.

Abstract

OBJECTIVES

To report the final results of the 2-year TAURUS study, assessing weekly prophylaxis dosing regimens of octocog alfa (Kovaltry®/BAY 81-8973) used in standard clinical practice in patients with moderate-to-severe haemophilia A.

METHODS

TAURUS (NCT02830477) is a phase 4, multinational, prospective, non-interventional, single-arm study in patients of any age with moderate or severe haemophilia A (≤5% factor [F]VIII activity). TAURUS was designed to primarily investigate weekly prophylaxis dosing regimens used in standard clinical practice. Annualised bleeding rates (ABRs), treatment satisfaction and adherence, and safety were also assessed.

RESULTS

Of 302 patients included in the full analysis set, 84.4% (n = 255) maintained their octocog alfa prophylaxis baseline regimen throughout the study, with a majority of patients (76.5%, n = 231) on two times or three times weekly regimens at the end of the observation period (≥1-≤2 years). ABRs, treatment satisfaction, and adherence remained stable during the observation period. Octocog alfa was well tolerated and there were no new or unexpected adverse events.

CONCLUSIONS

These data show that a smooth transition is observed when switching to octocog alfa from a previous FVIII treatment, with no safety issues and stable bleeding rates in a real-world setting of patients with moderate-to-severe haemophilia A.

摘要

目的

报告 2 年 TAURUS 研究的最终结果,评估在中度至重度血友病 A 患者的标准临床实践中使用的八聚体人凝血因子 VIII (奥塔库阿尔法)(Kovaltry®/BAY 81-8973)每周预防治疗方案。

方法

TAURUS(NCT02830477)是一项多中心、前瞻性、非干预性、单臂的 4 期研究,纳入了任何年龄的中重度血友病 A(≤5% FVIII 活性)患者。TAURUS 主要旨在研究标准临床实践中使用的每周预防治疗方案。还评估了年化出血率(ABR)、治疗满意度和依从性以及安全性。

结果

在全分析集的 302 名患者中,84.4%(n=255)在整个研究期间维持其八聚体人凝血因子 VIII 预防治疗的基线方案,在观察期结束时(≥1-≤2 年),大多数患者(76.5%,n=231)采用每周两次或三次方案。在观察期间,ABR、治疗满意度和依从性保持稳定。八聚体人凝血因子 VIII 耐受性良好,没有新的或意外的不良事件。

结论

这些数据表明,在中度至重度血友病 A 患者的真实世界环境中,从之前的 FVIII 治疗转换为八聚体人凝血因子 VIII 后,可实现平稳过渡,没有安全性问题,且出血率稳定。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/55bd/10092432/9969089697de/EJH-110-77-g003.jpg

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