重组人凝血因子VIII在A型血友病患者中的有效性和安全性：真实世界AHEAD国际研究长达7年的随访
The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study.
作者信息
Ozelo Margareth C, Hermans Cedric, Carcao Manuel, Guillet Benoît, Gu Joan, Guerra Randy, Tang Leilei, Khair Kate
机构信息
Hemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil.
St-Luc University Hospital, Université catholique de Louvain, Louvain, Belgium.
出版信息
Ther Adv Hematol. 2024 Feb 15;15:20406207231218624. doi: 10.1177/20406207231218624. eCollection 2024.
BACKGROUND
Real-world data assessing treatment outcomes in patients with hemophilia A in routine clinical practice are limited.
OBJECTIVE
To evaluate the effectiveness and safety of octocog alfa in patients with moderate/severe hemophilia A receiving treatment in clinical practice.
DESIGN
The international Antihemophilic Factor Hemophilia A Outcome Database study is an observational, noninterventional, prospective, multicenter study.
METHODS
This planned interim data read-out was conducted following 7 years of observation of patients receiving octocog alfa (cut-off, 30 June 2020). The primary endpoint was joint health status, assessed by the Gilbert Score. Secondary endpoints included annualized bleeding rates (ABRs), Hemophilia Joint Health Score (HJHS), health-related quality of life, consumption, and safety. This analysis stratified data by hemophilia severity at baseline [moderate, factor VIII (FVIII) 1-5%; severe, FVIII <1%].
RESULTS
Of the 711 patients in this analysis, 582 (82%) were receiving prophylaxis with octocog alfa at enrollment, and 498 (70%) had severe disease. Median Gilbert Scores were higher with on-demand therapy prophylaxis and scores were comparable in moderate and severe disease. In patients receiving prophylaxis, there was an improvement in HJHS Global Gait Score over 7 years of follow-up overall and in patients with severe disease. ABRs and annualized joint bleeding rates were low across all 7 years. An ABR of zero was reported in 34-56% of prophylaxis patients 20-40% in the on-demand group. ABRs were similar in severe and moderate disease. In total, 13/702 (1.9%) patients experienced 18 treatment-related adverse events.
CONCLUSION
These data demonstrate the long-term effectiveness and safety of octocog alfa in patients with moderate and severe hemophilia A, especially in those receiving prophylaxis. The high number of patients receiving on-demand treatment experiencing zero bleeds could be due to selection bias within the study, with patients with less severe disease more likely to be receiving on-demand treatment.
TRIAL REGISTRATION
ClinicalTrials.gov: NCT02078427.
背景
在常规临床实践中评估甲型血友病患者治疗结果的真实世界数据有限。
目的
评估奥曲泊帕在临床实践中接受治疗的中重度甲型血友病患者中的有效性和安全性。
设计
国际抗血友病因子甲型血友病结果数据库研究是一项观察性、非干预性、前瞻性、多中心研究。
方法
在对接受奥曲泊帕治疗的患者进行7年观察后（截止日期为2020年6月30日）进行了本次计划中的中期数据解读。主要终点是关节健康状况，通过吉尔伯特评分进行评估。次要终点包括年化出血率（ABR）、血友病关节健康评分（HJHS）、健康相关生活质量、消耗量和安全性。该分析根据基线时的血友病严重程度对数据进行分层[中度，因子VIII（FVIII）1-5%；重度，FVIII<1%]。
结果
在本次分析的711名患者中，582名（82%）在入组时接受奥曲泊帕预防治疗，498名（70%）患有严重疾病。按需治疗组的中位吉尔伯特评分高于预防治疗组，中度和重度疾病患者的评分相当。在接受预防治疗的患者中，总体上以及重度疾病患者在7年随访期间HJHS全球步态评分有所改善。在所有7年中，ABR和年化关节出血率均较低。在34-56%的预防治疗患者中报告ABR为零，按需治疗组为20-40%。重度和中度疾病患者的ABR相似。共有13/702（1.9%）名患者发生了18起与治疗相关的不良事件。
结论
这些数据证明了奥曲泊帕在中重度甲型血友病患者中的长期有效性和安全性，尤其是在接受预防治疗的患者中。大量接受按需治疗的患者出血为零，这可能是由于研究中的选择偏倚，病情较轻的患者更有可能接受按需治疗。
试验注册
ClinicalTrials.gov：NCT02078427。
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