Van Thillo Quentin, Hermans Cédric
Haemophilia Centre, Department of Cardiovascular Sciences, University Hospitals Leuven, Leuven, Belgium.
Thrombosis and Haemostasis Unit, Division of Haematology, Cliniques universitaires Saint-Luc, Brussels, Belgium.
Res Pract Thromb Haemost. 2025 Mar 6;9(2):102723. doi: 10.1016/j.rpth.2025.102723. eCollection 2025 Feb.
Gene therapy will very likely change the treatment paradigm of hemophilia B in the coming years. For the majority of patients, adjunctive exogenous factor (F)IX clotting factor concentrate will continue to be needed in case of surgery or bleeding. However, there is insufficient evidence on the optimal FIX product to be used in these circumstances, given the differences in body distribution between the currently available products. Unknown factors include the behavior of FIX Padua in the extravascular space and its contribution to hemostasis. Other issues are the potential importance of the presence of cross-reactive material and the discrepancies between different assays in measuring FIX activity. In conclusion, even after gene therapy, the differences between different FIX products remain relevant for optimal bleeding and perioperative management. Thus, real-world data on the use of exogenous FIX after gene therapy are needed to determine the preferred exogenous FIX concentrate.
基因疗法很可能在未来几年改变乙型血友病的治疗模式。对于大多数患者来说，在手术或出血情况下仍将继续需要辅助性外源性凝血因子IX浓缩物。然而，鉴于目前可用产品在体内分布上的差异，关于在这些情况下使用的最佳FIX产品的证据不足。未知因素包括FIX Padua在血管外空间的行为及其对止血的贡献。其他问题是交叉反应物质存在的潜在重要性以及不同检测方法在测量FIX活性方面的差异。总之，即使在基因治疗后，不同FIX产品之间的差异对于最佳出血和围手术期管理仍然具有相关性。因此，需要基因治疗后使用外源性FIX的真实世界数据来确定首选的外源性FIX浓缩物。
