Rush University Medical Center, Hemophilia and Thrombophilia Center, 1653 West Congress Parkway, Chicago, IL 60612-3833, USA.
Expert Opin Biol Ther. 2011 Oct;11(10):1361-8. doi: 10.1517/14712598.2011.603304. Epub 2011 Jul 22.
Hemophilia B, the deficiency or complete absence of coagulation factor IX (FIX), affects an estimated 80,000 people throughout the world. Some of these individuals are managed with prophylaxis, which involves the intravenous infusion of FIX concentrate two to three times weekly to prevent bleeding. Because FIX prophylaxis remains underutilized, patients with hemophilia B are at risk for bleeding that may be severe and potentially life- or limb-threatening, and they may experience arthropathy resulting from recurrent hemarthroses.
This review focuses on recent advances in therapeutic protein fusion technology as they apply to FIX deficiency. The National Library of Medicine Medline database was searched for articles containing the term 'Fc fusion proteins'.
Genetically engineered recombinant FIX fused to the Fc portion of immunoglobulin significantly extends FIX half-life, thereby decreasing the frequency of prophylactic infusions. This in turn may increase the adoption of, and adherence to, prophylaxis, leading to better outcomes for hemophilia B patients.
血友病 B 是由凝血因子 IX（FIX）缺乏或完全缺失引起的，全球估计有 8 万人受到影响。其中一些人通过预防治疗来管理，即每周通过静脉输注 FIX 浓缩物两到三次以预防出血。由于 FIX 预防治疗的使用率仍然较低，血友病 B 患者存在出血风险，这种出血可能很严重，甚至可能危及生命或肢体，并且他们可能会因反复关节内出血而出现关节病。
本篇综述重点介绍了治疗性蛋白融合技术的最新进展及其在 FIX 缺乏症中的应用。美国国立医学图书馆的 Medline 数据库检索了包含术语“Fc 融合蛋白”的文章。
通过基因工程将重组 FIX 与免疫球蛋白的 Fc 部分融合，可显著延长 FIX 的半衰期，从而减少预防性输注的频率。这反过来又可能增加预防治疗的采用率和患者的依从性，从而改善血友病 B 患者的预后。
