Franchini Massimo, Zaffanello Marco, Focosi Daniele
Department of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantua, Italy.
Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, University of Verona, Verona, Italy.
Expert Opin Pharmacother. 2023 Apr;24(6):729-736. doi: 10.1080/14656566.2023.2196012. Epub 2023 Mar 29.
Therapy for hemophilia B is aimed at replacing the congenital deficiency of coagulation factor IX (FIX). For replacement therapy, several FIX concentrates derived from donated human plasma or engineered by recombinant DNA technology are currently commercially available. The use of these products is well established and permit patients a relatively normal life. To further improve treatment efficacy, recombinant FIX products with a prolonged half-life have been developed, allowing relaxed prophylactic dosing and reducing treatment burden.
In this review, we explore the current FIX replacement options for hemophilia B patients by analyzing the outcomes of their main clinical trials. We cover advances in the FIX molecules with extended half-life (EHL). Published literature on products for replacement of hemophilia B was retrieved using PubMed with no temporal limits.
The recent introduction of recombinant EHL FIX products has represented a major advance in the therapeutic management of hemophilia B patients, permitting both a reduction of treatment burden and improving patients' compliance to prophylaxis and, ultimately, quality of life.
B型血友病的治疗旨在补充先天性凝血因子IX（FIX）缺乏。对于替代疗法，目前有几种源自捐赠人血浆或通过重组DNA技术制造的FIX浓缩物可供商业使用。这些产品的使用已得到充分确立，使患者能够过上相对正常的生活。为了进一步提高治疗效果，已开发出半衰期延长的重组FIX产品，从而允许放宽预防性给药并减轻治疗负担。
在本综述中，我们通过分析主要临床试验的结果，探讨了目前B型血友病患者的FIX替代选择。我们涵盖了半衰期延长（EHL）的FIX分子的进展。使用PubMed检索了关于B型血友病替代产品的已发表文献，无时间限制。
最近引入的重组EHL FIX产品代表了B型血友病患者治疗管理方面的一项重大进展，既减轻了治疗负担，又提高了患者对预防治疗的依从性，并最终改善了生活质量。
