Klamroth Robert, Hampton Kingsley, Saulyte Trakymienė Sonata, Korsholm Lars, Carcao Manuel
Department of Internal Medicine, Hemophilia Treatment Centre, Vivantes Klinikum im Friedrichshain, Berlin, Germany.
Department of Cardiovascular Science, University of Sheffield, Sheffield, UK.
Patient Prefer Adherence. 2021 Nov 4;15:2443-2454. doi: 10.2147/PPA.S326282. eCollection 2021.
To illustrate the benefits of the extended half-life (EHL) recombinant factor VIII product N8-GP (Esperoct, turoctocog alfa pegol) by describing individual cases of patients with severe hemophilia A treated with N8-GP in the pathfinder clinical trial program.
This manuscript presents selected patient cases from the pivotal pathfinder clinical trial program, which included a number of clinical studies in adults (pathfinder 2 and 3) and children (pathfinder 5); overall results published previously. Clinical data and outcomes described in this manuscript are more detailed and derived from several interesting patient cases (five adults from pathfinder 2 and two children from pathfinder 5), who received N8-GP as prophylaxis (PPX) for their severe hemophilia A. Three of the five adults described here also underwent multiple major surgeries (for which they moved from pathfinder 2 into pathfinder 3 and later returned to pathfinder 2). New analyses on pediatric joint health from pathfinder 5 are also summarized here. Outcomes assessed included bleeding complications, improvements in quality of life, intraoperative hemostatic response, blood loss during surgery, number of blood transfusions, and annualized bleeding rates. For the pediatric patients, target joint resolution, adverse events, and annualized joint bleeding rate were also assessed, all by the treating physician.
Considerable improvements in treatment adherence, bleeding rates, and overall physical activity levels were demonstrated in two adult cases from the pathfinder 2 trial. N8-GP demonstrated good or excellent hemostatic coverage in three adult patients undergoing multiple major surgeries. The benefits of N8-GP for joint health and in support of children and adolescents with evolving active lifestyles were reported for several pediatric cases.
These patient cases highlight the benefits of EHL products, such as N8-GP, for patients with severe hemophilia A. They include more challenging scenarios relating to improvements in previously poor adherence to PPX, children with active sporting lifestyles, and patients requiring multiple major surgeries.
通过描述在探索者临床试验项目中接受N8-GP（Esperoct，聚乙二醇化重组凝血因子VIII）治疗的重度甲型血友病患者的个体病例，来说明延长半衰期（EHL）重组凝血因子VIII产品N8-GP的益处。
本手稿介绍了关键探索者临床试验项目中的部分患者病例，该项目包括多项针对成人（探索者2和3）和儿童（探索者5）的临床研究；此前已发表总体结果。本手稿中描述的临床数据和结果更详细，源自几个有趣的患者病例（探索者2中的5名成人和探索者5中的2名儿童），他们因重度甲型血友病接受N8-GP预防治疗（PPX）。此处描述的5名成人中有3人还接受了多次大手术（为此他们从探索者2转入探索者3，后来又回到探索者2）。此处还总结了探索者5中关于儿童关节健康的新分析。评估的结果包括出血并发症、生活质量改善、术中止血反应、手术期间失血、输血次数和年化出血率。对于儿科患者，治疗医生还评估了目标关节恢复情况、不良事件和年化关节出血率。
探索者2试验中的2例成人病例显示，在治疗依从性、出血率和总体身体活动水平方面有显著改善。N8-GP在3例接受多次大手术的成人患者中显示出良好或优异的止血覆盖效果。在几个儿科病例中，报告了N8-GP对关节健康的益处以及对积极生活方式不断变化的儿童和青少年的支持。
这些患者病例突出了EHL产品（如N8-GP）对重度甲型血友病患者的益处。这些益处包括与改善先前较差的PPX依从性、积极运动生活方式的儿童以及需要多次大手术的患者相关的更具挑战性的情况。
