Pharmaceutical Medicine Group, Faculty of Life Sciences and Medicine, King's College London, 150 Stamford Street, London, SE1 9NH, England.
EBD London Ltd., 147 Sulgrave Road, London, W6 7PX, England.
Pharmaceut Med. 2021 Sep;35(5):281-286. doi: 10.1007/s40290-021-00400-0. Epub 2021 Sep 7.
Traditional approaches to blood regulation emphasise the precautionary principle and pursue zero-risk for viral transmission; these traditional approaches have usually followed tragedy, such as the HIV and hepatitis C infections that followed the use of factor VIII concentrates. However, a much more haphazard haemovigilance system operates for general adverse events. Such imprecise assessment of hazards prevents sound benefit-risk assessment, and for blood products this is further confounded by the fact that their efficacy has attracted little systematic study. The ongoing COVID-19 pandemic has now prompted the proposal of a convalescent plasma (CP) blood product. Clearly, mere freedom from infectious agents will not suffice in assessing CP, and an objective measure of efficacy, so as to permit formal benefit-risk analysis, is essential. This is both a scientific and an ethical demand, as has been the case for other experimental COVID-19 treatments. With special reference to COVID-19 CP, the well-recognized adverse events of transfusion-associated lung injury (TRALI) and transfusion-associated circulatory overload (TACO) will be important. Furthermore, not only efficacy but also product quality attributes (e.g., antibody titre) will have to be defined. Both of these are outside the traditional regulatory philosophy for blood products and are needed to truly assess the benefit-risk of this putative therapeutic product.
传统的血液调控方法强调预防原则,并追求零病毒传播风险;这些传统方法通常是在发生悲剧后才采取的,例如在使用 VIII 因子浓缩物后出现的 HIV 和丙型肝炎感染。然而,对于一般不良事件,通常采用一种更加随意的血液监测系统。这种对危害的不准确评估妨碍了对收益风险的合理评估,对于血液制品来说,由于其疗效很少受到系统研究,情况更是如此。目前正在进行的 COVID-19 大流行促使人们提出了恢复期血浆(CP)血液制品的建议。显然,仅从没有传染性病原体方面评估 CP 是不够的,必须要有一个客观的疗效衡量标准,以便进行正式的收益风险分析。这既是科学上的要求,也是伦理上的要求,其他 COVID-19 实验性治疗也是如此。特别是对于 COVID-19 CP,众所周知的输血相关肺损伤(TRALI)和输血相关循环超负荷(TACO)等不良反应将很重要。此外,不仅需要确定疗效,还需要确定产品质量属性(例如抗体滴度)。这两者都超出了血液制品的传统监管理念,是真正评估这种潜在治疗产品收益风险所必需的。