Farrugia A
Manager Blood and Tissue Services, Therapeutic Goods Administration, Australian Commonwealth Department of Health and Aged Care, Adviser on Blood Safety, World Federation of Hemophilia, Australia.
Haemophilia. 2002 May;8(3):236-43. doi: 10.1046/j.1365-2516.2002.00596.x.
The past decade has seen a consolidation of the safety measures built into the manufacture of coagulation factor concentrates for people with haemophilia. The scientific developments of the 1980s have been fully reflected in the manufacturing principles and the regulatory control of concentrate production, so that the safety of concentrate therapy now exceeds the safety of normal blood transfusion. A clear understanding of the epidemiology of the transfusion-transmitted viruses allows the selection of donors with a satisfactory safety profile. Source material for plasma-derived concentrates is now screened with sensitive tests that detect viral infection in donors at a very early phase in the viral life cycle. This decreases the potential viral load to levels that are easily eliminated by well-accredited viral inactivation procedures. These measures have ensured a high level of assurance regarding the safety of products from the traditional transfusion-transmitted infections. However, testing for some transfusion-transmitted viruses does not yet form part of mainstream blood screening; alternative strategies based upon the particular needs of pooled plasma product recipients may be more feasible. Whereas the risk of emerging pathogens has to be kept constantly under review, four such viruses identified over the 1990s have proven to be of little relevance to plasma product recipients and the need for measures specifically directed against them is debatable. The risk of variant Creuzfeldt-Jakob Disease (vCJD) is a special case of an emerging infection that is insufficiently well characterized to allow a conclusive assessment of its role in the safety of concentrates; however, data regarding the capacity of concentrate manufacturing methods to clear the putative agent are encouraging. The relative uncertainty surrounding vCJD has caused the influential regulatory authorities of North America to take a precautionary approach regarding the selection of blood donors. This is having an effect on the supply of factor VIII concentrates and has possibly affected the rate at which developed countries have switched to recombinant products. The safety of recombinant products continues to be supported through patient monitoring, and the new generation of plasma protein-free products will further enhance the role of these products as the treatment of choice with people with haemophilia. However, their cost-effectiveness relative to the current generation of plasma-derived products makes it unlikely that they will be accessible by developing countries. The dependence of most of the world's population of people with haemophilia on a safe and sufficient blood supply will therefore continue into the foreseeable future, and with it the need to maintain constant vigilance on blood safety matters.
在过去十年中，血友病患者凝血因子浓缩物生产过程中的安全措施得到了巩固。20世纪80年代的科学发展已充分体现在浓缩物生产的制造原则和监管控制中，因此目前浓缩物疗法的安全性已超过正常输血的安全性。对输血传播病毒流行病学的清晰了解有助于选择具有令人满意安全状况的献血者。现在，用于血浆源性浓缩物的原料通过灵敏检测进行筛查，这些检测能在病毒生命周期的非常早期阶段检测出献血者的病毒感染。这将潜在病毒载量降低到通过公认的病毒灭活程序易于消除的水平。这些措施确保了产品在传统输血传播感染方面的高度安全性。然而，对某些输血传播病毒的检测尚未成为主流血液筛查的一部分；基于混合血浆产品接受者的特殊需求的替代策略可能更可行。虽然必须不断审查新出现病原体的风险，但在20世纪90年代确定的四种此类病毒已证明与血浆产品接受者关系不大，针对它们采取专门措施的必要性存在争议。变异型克雅氏病（vCJD）的风险是一种新出现感染的特殊情况，其特征尚不充分，无法对其在浓缩物安全性中的作用进行确定性评估；然而，关于浓缩物生产方法清除假定病原体能力的数据令人鼓舞。围绕vCJD的相对不确定性导致北美有影响力的监管当局在献血者选择方面采取预防措施。这正在影响凝血因子VIII浓缩物的供应，并可能影响发达国家转向重组产品的速度。重组产品的安全性通过患者监测继续得到保障，新一代无血浆蛋白产品将进一步增强这些产品作为血友病患者首选治疗方法的作用。然而，相对于当前一代血浆源性产品，它们的成本效益使得发展中国家不太可能获得。因此，在可预见的未来，世界上大多数血友病患者对安全充足血液供应的依赖仍将持续，随之而来的是需要对血液安全问题保持持续警惕。
