Haemophilia: factoring in new therapies. Review uri icon

Overview

abstract

  • Haemophilia is an inherited bleeding disorder in which the haemostatic defect results from deficiency of coagulation factor VIII (FVIII) in haemophilia A or factor IX (FIX) in haemophilia B. Traditional treatments for haemophilia have largely worked by directly replacing the missing coagulation factor, but face challenges due to the short half-life of FVIII and FIX, the need for frequent intravenous access and development of neutralising antibodies to coagulation factors (inhibitors). Recent advances in haemophilia therapy have worked to eliminate these challenges. Half-life extension of factor concentrates has lengthened the time needed between infusions, enhancing quality of life. Subcutaneous administration of therapeutics utilising alternative mechanisms to overcome inhibitors have expanded the options to prevent bleeding. Finally, initial successes with gene therapy offer a cautious hope for durable cure. In the present review, we will discuss currently available treatments, as well as highlight therapeutics in various stages of clinical development for the treatment of haemophilia A and B. In this review, we present therapies that are currently clinically available and highlight therapeutics that are in various stages of clinical development for the treatment of haemophilia A and B.

publication date

  • July 28, 2021

Research

keywords

  • Hemophilia A
  • Hemophilia B

Identity

Scopus Document Identifier

  • 85111379511

Digital Object Identifier (DOI)

  • 10.1111/bjh.17580

PubMed ID

  • 34322873

Additional Document Info

volume

  • 194

issue

  • 5