Franchini Massimo
Carlo Poma Hosp - Dept Transfus Med and Hematol, Mantova, Italy.
Expert Rev Hematol. 2014 Oct;7(5):573-81. doi: 10.1586/17474086.2014.947955. Epub 2014 Aug 12.
Hemophilia B is a rare hereditary hemorrhagic disorder characterized by deficiency of the clotting factor IX (FIX). Hemophilia B patients experience mild-to-severe bleeding complications according to the degree of FIX defect. The mainstay of treatment of hemophilia B consists of replacement therapy and nowadays several plasma-derived and recombinant FIX products are commercially available. This article reviews the current management of hemophilia B patients analyzing the results of the most important clinical trials. In addition, it will focus on the more recent advances in the production of new FIX molecules aimed at the improvement of the clinical management of such patients.
血友病B是一种罕见的遗传性出血性疾病,其特征是凝血因子IX(FIX)缺乏。根据FIX缺陷程度,血友病B患者会出现轻度至重度出血并发症。血友病B的主要治疗方法是替代疗法,如今有几种血浆源性和重组FIX产品可供商业使用。本文回顾了血友病B患者的当前管理情况,分析了最重要的临床试验结果。此外,还将重点关注旨在改善此类患者临床管理的新型FIX分子生产方面的最新进展。