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Physical activity in individuals with haemophilia and experience with recombinant factor VIII Fc fusion protein and recombinant factor IX Fc fusion protein for the treatment of active patients: a literature review and case reports

机译:血友病患者的身体活动以及重组因子VIII Fc融合蛋白和重组因子IX Fc融合蛋白在活跃患者中的治疗经验:文献复习和病例报告

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摘要

The World Federation of Hemophilia and the National Hemophilia Foundation encourage people with haemophilia (PWH) to participate in routine physical activity. The benefits of physical activity for PWH include improvements in joint, bone, and muscle health. Accordingly, a number of studies suggest that levels of physical activity among PWH are similar to those of their healthy peers, especially among individuals who began prophylaxis at an early age (≤3 years). Importantly, several studies found either no increased risk or only a transient increase in risk of bleeding with more intensive physical activity compared with less intensive physical activity. Data on optimal prophylaxis regimens for PWH who participate in physical/sporting activities; however, remain sparse. Long-acting recombinant factor VIII Fc fusion protein (rFVIIIFc) and recombinant factor IX Fc fusion protein (rFIXFc) demonstrated efficacy for the prevention and treatment of bleeding episodes in Phase 3 clinical trials of participants with haemophilia A and B, respectively, with most individuals able to maintain or increase their physical activities. This manuscript reviews the current literature that describes physical activity in PWH. Additionally, case studies are presented to provide supplemental information to clinicians illustrating the use of rFVIIIFc and rFIXFc in physically active patients with haemophilia A and B, respectively. These case reports demonstrate that it is possible for patients to be physically active and maintain good control of their haemophilia with extended interval prophylactic dosing using rFVIIIFc or rFIXFc.
机译:世界血友病联合会和国家血友病基金会鼓励血友病患者参加常规体育锻炼。体育锻炼对PWH的好处包括关节,骨骼和肌肉健康的改善。因此,许多研究表明,PWH中的体育锻炼水平与其健康同龄人相似,尤其是在早于年龄(≤3岁)开始进行预防的个体中。重要的是,几项研究发现,与强度较低的体育活动相比,强度较高的体育活动不会增加出血风险,或者只是暂时增加出血风险。参加体育/体育活动的PWH最佳预防方案的数据;但是,保持稀疏状态。长效重组凝血因子VIII Fc融合蛋白(rFVIIIFc)和重组凝血因子IX Fc融合蛋白(rFIXFc)在分别针对大多数人的A和B型血友病患者的3期临床试验中证明了预防和治疗出血发作的功效能够维持或增加他们的体育活动。该手稿回顾了描述PWH中身体活动的最新文献。此外,还提供了一些案例研究,以向临床医生提供补充信息,以说明rFVIIIFc和rFIXFc在分别患有A和B型血友病的体力活动患者中的使用。这些病例报告表明,使用rFVIIIFc或rFIXFc进行间隔延长的预防性给药,可使患者保持身体活动并保持对血友病的良好控制。

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