背景:需要源血浆收集来满足对血浆衍生的医药产品的日益增长的需求。自1992年以来,美国血浆收集目标体积一直由FDA发布的基于标准重量的列线图(STAN)指导。在这项研究中,我们对基于美国的大规模真实世界数据(RWD)进行了分析,以确认之前在上市前随机对照环境中研究的新引入的个性化列线图(PERS)的安全性和体积增益.
方法:进行了非劣效性(NI)分析,以比较新的列线图的显著低血压不良事件(AE)发生率与大型历史标准列线图AE数据集。此外,在PERS和STAN之后,对收集的平均目标量和捐献者返回率进行了比较.
结果:414,957名捐献者共4,816,784次捐赠(PERS)导致0.0998%(95%CI[0.0970,0.1027])的显著低血压不良事件发生率。NI分析表明新技术具有很强的非劣效性(Δ=-0.0082%,95%CI[-0.0113,-0.0050],预设NI边距=0.1080)。平均血浆收集目标体积增加66.39mL(8.49%;p<0.0001)。连续的每周捐赠者回报率在两个列线图之间是一致的(PERS:73.6%,95%CI[69.6%-76.7%];STAN:74.1%,95%CI[66.1%-77.2%])。
结论:该分析在大规模的现实世界数据集中证实了一种新型的关键安全参数和收集益处,启用技术的列线图。所述列线图可以通过在保持供体安全性和回报率的同时,平均每次捐献提供大约8.5%的更多血浆来帮助满足对血浆衍生疗法的日益增长的需求。
Source plasma collections are needed to satisfy the growing demand for plasma-derived medicinal products. The US plasma collection target volume has been guided by a standard weight-based FDA-issued nomogram (STAN) since 1992. In this research, large-scale US-based real-world data (RWD) were analyzed to confirm the safety and volume gains of a newly introduced personalized nomogram (PERS) that was previously studied in a premarket randomized controlled environment.
A non-inferiority (NI) analysis was conducted to compare the novel nomogram\'s significant hypotensive adverse event (AE) incidence rate with large historical standard nomogram AE datasets. Additionally, the average target volumes and donor return rates were compared for collections following PERS and STAN.
A total of 4,816,784 donations (PERS) by 414,957 donors resulted in a rate of 0.0998% (95% CI [0.0970, 0.1027]) significant hypotensive AEs. NI analysis suggested strong non-inferiority of the new technology (Δ = -0.0082%, 95% CI [-0.0113, -0.0050], prespecified NI margin = 0.1080). Average plasma collection target volumes increased by 66.39 mL (8.49%; p < .0001). Consecutive weekly donor return rates were consistent between the two nomograms (PERS: 73.6%, 95% CI [69.6%-76.7%]; STAN: 74.1%, 95% CI [66.1%-77.2%]).
This analysis confirms in a large-scale real-world dataset the key safety parameter and collection benefit of a novel, technology-enabled nomogram. The nomogram may help meet the growing demand for plasma-derived therapies by providing approximately 8.5% more plasma per donation on average while maintaining donor safety and return rates.