Laboratory testing for non-factor therapy in hemophilia: progress and challenges from traditional to innovative approaches
10.3760/cma.j.cn114452-20250425-00254
- VernacularTitle:血友病非因子治疗的实验室检测：从传统到创新的进展与挑战
- Author:
Jing DAI
1
;
Xiaoli MA
1
;
Yu LIU
1
;
Xuefeng WANG
1
Author Information
1. 上海交通大学医学院附属瑞金医院检验科，上海 200025
- Publication Type:Journal Article
- Keywords:
Hemophilia A;
Hemophilia B;
Hemostasis and thrombosis determination;
Non-factor drugs
- From:
Chinese Journal of Laboratory Medicine
2025;48(8):953-961
- CountryChina
- Language:Chinese
-
Abstract:
Hemophilia A and hemophilia B are hereditary coagulation bleeding disorders. Traditional treatment primarily relies on factor replacement therapy using coagulation factor Ⅷ(FⅧ) or coagulation factor Ⅸ (FⅨ)products. Although conventional therapies can alleviate bleeding symptoms to some extent, they also have certain limitations, such as the need for frequent infusions, the risk of infection, and the potential development of inhibitors in some patients. Currently, treatment strategies for hemophilia are gradually shifting toward non-factor therapies, providing the appearence of novel non-factor drugs. However, these novel therapies not only interfere with traditional coagulation assays but may also fail to comprehensively evaluate their efficacy and safety through conventional coagulation tests. Therefore, there is an urgent need to develop and optimize new laboratory testing methods to ensure accurate assessment of patients′ responses to non-factor therapies and the hemostatic capacity of the drugs themselves. Although some studies have explored the coagulation factor equivalence of non-factor agents, such equivalence cannot fully reflect the actual hemostatic effect in patients after treatment and is therefore unsuitable as a prognostic indicator. Compared with assessing coagulation factor equivalence, total coagulation assays, such as the thrombin generation assay (TGA), can more accurately evaluate efficacy and safety. TGA can take into account multiple factors in the coagulation process, providing a more comprehensive assessment of coagulation function. Furthermore, combining TGA with patient symptoms for comprehensive analysis can enhance its prognostic predictive ability, offering more reliable support for clinical decision-making.
