Margareth C Ozelo, Cedric Hermans, Manuel Carcao, Benoît Guillet, Joan Gu, Randy Guerra, Leilei Tang, Kate Khair
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引用次数: 0
Abstract
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 post hoc 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 versus 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 versus 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.
octocog alfa 对 A 型血友病患者的有效性和安全性：真实世界 AHEAD 国际研究长达 7 年的随访。
背景：在常规临床实践中，评估血友病 A 患者治疗效果的真实世界数据非常有限：评估血友病 A 患者在常规临床实践中治疗效果的真实世界数据非常有限：评估在临床实践中接受治疗的中度/重度血友病 A 患者使用辛可格α的有效性和安全性：国际抗血友病因子血友病 A 型结果数据库研究是一项观察性、非介入性、前瞻性、多中心研究：在对接受辛可克α治疗的患者进行 7 年观察后（截止日期为 2020 年 6 月 30 日），计划进行中期数据读取。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。这项事后分析根据基线时血友病的严重程度对数据进行了分层[中度，因子 VIII (FVIII) 1-5%；重度，因子 VIII 结果]：在参与分析的 711 名患者中，582 人（82%）在入组时接受了辛可格α的预防治疗，498 人（70%）病情严重。按需治疗与预防治疗的吉尔伯特评分中位数更高，中度和重度患者的评分相当。在接受预防性治疗的患者中，经过7年的随访，HJHS总体步态评分总体上有所改善，重症患者的步态评分也有所改善。所有 7 年的 ABR 和年化关节出血率都很低。34-56%的预防性治疗患者的ABR为零，而按需治疗组为20-40%。重度和中度患者的 ABR 相似。13/702（1.9%）名患者共发生了18次与治疗相关的不良事件：这些数据证明了辛可格α对中度和重度 A 型血友病患者的长期有效性和安全性，尤其是对接受预防性治疗的患者。接受按需治疗的患者中出现零出血的人数较多，这可能是由于研究中存在选择偏差，病情较轻的患者更有可能接受按需治疗：试验注册：ClinicalTrials.gov：试验注册：ClinicalTrials.gov：NCT02078427。
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Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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