Department of Hematology, Bayer AG, Basel, Switzerland.
Hematology Department, Hospital Universitario La Paz, Madrid, Spain.
BMJ Open. 2021 Sep 2;11(9):e044997. doi: 10.1136/bmjopen-2020-044997.
Haemophilia A is a rare bleeding disorder caused by defects in coagulation factor VIII (FVIII). Damoctocog alfa pegol (BAY 94-9027, Jivi, Bayer, Germany) is a site-specifically PEGylated, extended-half-life, recombinant FVIII, approved for use in previously treated patients (PTPs) aged ≥12 years with haemophilia A. However, a real-world evidence regarding routine clinical use of damoctocog alfa pegol is limited.
HEM-POWR is a multinational, multicentre, non-interventional, prospective, postmarketing cohort study evaluating the effectiveness and safety of real-world treatment with damoctocog alfa pegol. Estimated enrolment is ≥200 PTPs with haemophilia A, receiving damoctocog alfa pegol (on-demand, prophylaxis or intermittent prophylaxis (as per local label)), observed for 36 months. Primary outcomes are total bleeding events and annualised bleeding rate; secondary outcomes include long-term safety, joint health, pharmacokinetics, patient-reported outcomes (PROs) from validated questionnaires and perioperative haemostasis. Where applicable, reasons for switching to damoctocog alfa pegol, choice of treatment regimen and dose will also be captured. Exploratory and descriptive statistical analyses will be performed, and will be stratified by parameters including, but not limited to, prophylaxis regimen and haemophilia severity. Patients can record bleeds and consumption in electronic (e) Diaries, ePROs, and can access non-promotional study information (videos explaining study procedures) via an online patient portal. Optionally, patients can enrol in the LIFE-ACTIVE substudy designed to investigate the relationship between activity (measured by the ActiGraph CP Insight watch) and effectiveness parameters collected from HEM-POWR.
Study approval was obtained by local independent ethics committees and authorities in participating study centres across Europe, the Americas and Asia. Informed consent from patients or their legal representative is a requirement for participation. The study results will be submitted for publication in a peer-reviewed scientific journal and presented at scientific conferences.
NCT03932201, EUPAS26416.
V.1.2, 27 September 2019.
A 型血友病是一种罕见的出血性疾病，由凝血因子 VIII（FVIII）缺陷引起。达莫洛科戈阿尔法 Pegol（BAY 94-9027、Jivi、拜耳，德国）是一种定点 PEG 化、半衰期延长的重组 FVIII，批准用于年龄≥12 岁的既往治疗患者（PTP）与 A 型血友病。然而，关于达莫洛科戈阿尔法 Pegol 的常规临床应用的真实世界证据有限。
HEM-POWR 是一项多中心、多国、非干预性、前瞻性、上市后队列研究，旨在评估达莫洛科戈阿尔法 Pegol 在真实世界环境中的有效性和安全性。预计≥200 例 A 型血友病 PTP 将接受达莫洛科戈阿尔法 Pegol（按需、预防或间歇性预防（根据当地标签））治疗，并观察 36 个月。主要结局是总出血事件和年化出血率；次要结局包括长期安全性、关节健康、药代动力学、来自验证问卷的患者报告结果（PROs）和围手术期止血。在适用的情况下，还将记录转换为达莫洛科戈阿尔法 Pegol 的原因、治疗方案和剂量的选择。将进行探索性和描述性统计分析，并按包括但不限于预防方案和血友病严重程度等参数进行分层。患者可以通过电子（e）日记、ePROs 记录出血和消耗量，并通过在线患者门户访问非促销研究信息（解释研究程序的视频）。患者可以选择参加 LIFE-ACTIVE 子研究，旨在调查 HEM-POWR 收集的活动（通过 ActiGraph CP Insight 手表测量）和有效性参数之间的关系。
研究已获得欧洲、美洲和亚洲参与研究中心的当地独立伦理委员会和当局的批准。患者或其法定代表的知情同意是参与的要求。研究结果将提交同行评议的科学期刊发表，并在科学会议上展示。
NCT03932201，EUPAS26416。
V.1.2，2019 年 9 月 27 日。
