Medical Program, Faculty of Medicine, Universitas Airlangga, Surabaya, Indonesia.
Department of Physiology and Pharmacology, SUNY Downstate Health Sciences University, Brooklyn, New York, USA.
F1000Res. 2021 Oct 15;10:1049. doi: 10.12688/f1000research.73884.3. eCollection 2021.
Patients with severe hemophilia often present with painful joint and soft tissue bleeding which may restrict them from their daily activities. The current standard of care still relies on a regular prophylactic factor VIII (FVIII), which has a high daily treatment burden. Recently, rurioctocog alfa pegol, a third-generation recombinant FVIII with a modification in its polyethylene glycol (PEG) component, has been developed. Several trials have studied this synthetic drug as bleeding prophylaxis in severe hemophilia A. This study aims to evaluate the efficacy, safety, and immunogenicity of rurioctocog alfa pegol for previously treated patients with severe hemophilia A. : This study was conducted in conformity with the PRISMA guidelines. Data were retrieved from PubMed, Scopus, Cochrane Library, Wiley Online Library, and CINAHL (via EBSCOhost). Study qualities were assessed using the Methodological Index for Non-Randomized Studies (MINORS) and Modified Jadad scales. Four studies involving 517 previously treated severe hemophilia A patients were included in this study. The pooled mean of total annualized bleeding rate (ABR) and hemostatic efficacy was 2.59 (95% CI = 2.04-3.14) and 92% (95% CI = 85%-97%), respectively. Only 30 (2.3%) non-serious and one (1.4%) serious adverse events were considered related to rurioctocog alfa pegol treatment. At the end of the studies, no development of FVIII inhibitory antibodies was observed. None of the developed binding antibodies to FVIII, PEG-FVIII, or PEG was correlated to the treatment efficacy and safety. Despite the limited availability of direct comparison studies, our analyses indicate that rurioctocog alfa pegol could serve as a safe and effective alternative for bleeding prophylaxis in previously treated hemophilia A patients. Moreover, it appears to have low immunogenicity, which further increases the safety profile of the drug in such clinical conditions.
患有严重血友病的患者常出现疼痛性关节和软组织出血，这可能限制他们的日常活动。目前的标准治疗方法仍依赖于定期预防性使用第八因子（FVIII），这会带来很高的日常治疗负担。最近，第三代重组 FVIII 药物 rurioctocog alfa pegol 被开发出来，其聚乙二醇（PEG）成分经过修饰。多项试验研究了这种合成药物作为严重血友病 A 出血预防的疗效。本研究旨在评估 rurioctocog alfa pegol 对既往接受过治疗的严重血友病 A 患者的疗效、安全性和免疫原性。：本研究符合 PRISMA 指南。数据从 PubMed、Scopus、Cochrane 图书馆、Wiley Online Library 和 CINAHL（通过 EBSCOhost）检索。使用非随机研究方法学指数（MINORS）和改良 Jadad 量表评估研究质量。 本研究共纳入了 4 项涉及 517 例既往接受过治疗的严重血友病 A 患者的研究。总年化出血率（ABR）和止血疗效的汇总平均值分别为 2.59（95% CI = 2.04-3.14）和 92%（95% CI = 85%-97%）。仅 30 例（2.3%）非严重不良事件和 1 例（1.4%）严重不良事件被认为与 rurioctocog alfa pegol 治疗相关。在研究结束时，未观察到 FVIII 抑制性抗体的产生。未发现针对 FVIII、PEG-FVIII 或 PEG 的结合抗体与治疗疗效和安全性相关。 尽管直接比较研究的可获得性有限，但我们的分析表明，rurioctocog alfa pegol 可作为既往接受过治疗的血友病 A 患者出血预防的安全有效替代药物。此外，它似乎具有低免疫原性，这进一步增加了该药物在这种临床情况下的安全性。
