Intelligence Analysis of Treatments for Hemophilia B
10.3870/j.issn.1004-0781.2024.07.013
- VernacularTitle:血友病B治疗药物情报分析
- Author:
Weiwei ZHANG
1
;
Chunying LI
;
Ying LIU
Author Information
1. 北京大学医学图书馆,北京 100191
- Keywords:
Hemophilia B;
Biological products;
Intelligence analysis
- From:
Herald of Medicine
2024;43(7):1096-1102
- CountryChina
- Language:Chinese
-
Abstract:
Hemophilia B(HB)is a recessive congenital bleeding disorder with X-linked inheritance.The treatments for HB have made tremendous progress during past decades.Treatment paradigm shifting from prothrombin complex to plasma-derived coagulation factor IX(FIX)significantly improved the safety of patients.Bioengineered recombinant FIXs(rFIXs)with extended half-life compared to rFIXs ameliorat patient's compliance and clinical outcomes.Non-factor replacement therapies could reduce the burden of treatment through subcutaneous injection and avoid the risk of developing inhibitors to FIX.Meanwhile,these molecules could provide effective prophylaxis in the presence or absence of inhibitor.Genetically modified adeno-associated virus engineered to deliver FIX gene to the liver has heralded a new era of HB treatment.A comprehensive intelligence tracking of the treatments for HB,including currently marketed pharmaceuticals,pipelines of molecules under development and related research results is the focus of this paper.
