Department of Haematology, University Hospital of Wales and School of Medicine Cardiff University, Cardiff, UK.
Br J Haematol. 2015 Jun;169(6):768-76. doi: 10.1111/bjh.13360. Epub 2015 Mar 7.
Treatment of congenital haemophilia with factor VIII and IX concentrates often requires frequent infusions. This has obvious implications in establishing effective administration strategies and, in turn, adherence. To overcome these issues, three main technologies--polyethylene-glycol, Fc-neonatal IgG1 and albumin fusion products--have emerged into various stages of clinical development. Published data indicates an approximately 1·5- and fivefold increase in half-life of factor VIII and IX, respectively, compared to standard recombinant concentrates. Studies into efficacy and safety are starting to be published. Monitoring and optimal use of these new concentrates remains unknown. Weekly factor IX prophylaxis appears to be a feasible prophylactic regimen in haemophilia B patients. Weekly longer-acting FVIII is unlikely to provide adequate prophylaxis in most patients with haemophilia A but may reduce the frequency of infusions. Ongoing clinical trials and real life experience will help shape how these products can be used in practice and their cost effectiveness. The drive for convenience however should not overshadow the ultimate goal of prophylaxis, namely, preventing bleeding and arthropathy.
治疗先天性血友病患者时，常需要使用凝血因子 VIII 和 IX 浓缩物进行频繁输注。这对建立有效的管理策略产生了明显的影响，进而影响患者的治疗依从性。为了解决这些问题，聚乙二醇化、Fc-新生儿 IgG1 和白蛋白融合产品等三种主要技术已经进入了不同的临床开发阶段。已发表的数据表明，与标准重组浓缩物相比，VIII 因子和 IX 因子的半衰期分别延长了约 1.5 倍和 5 倍。关于这些新的浓缩物的疗效和安全性的研究也开始发表。这些新的浓缩物的监测和最佳使用方法仍不清楚。每周一次的凝血因子 IX 预防性治疗似乎是乙型血友病患者可行的预防性治疗方案。每周使用作用时间更长的 FVIII 不太可能为大多数甲型血友病患者提供充分的预防作用，但可能会减少输注的频率。正在进行的临床试验和实际经验将有助于确定如何在实践中使用这些产品及其成本效益。然而，便利性的诉求不应掩盖预防的最终目标，即预防出血和关节病。
