Extravascular factor IX after gene therapy in hemophilia B, does it matter?

B型血友病基因治疗后血管外因子IX是否重要?

阅读:1

Abstract

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.

特别声明

1、本页面内容包含部分的内容是基于公开信息的合理引用;引用内容仅为补充信息,不代表本站立场。

2、若认为本页面引用内容涉及侵权,请及时与本站联系,我们将第一时间处理。

3、其他媒体/个人如需使用本页面原创内容,需注明“来源:[生知库]”并获得授权;使用引用内容的,需自行联系原作者获得许可。

4、投稿及合作请联系:info@biocloudy.com。