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Haemophilia: factoring in new therapies
Author(s) -
Fassel Hannah,
McGuinn Catherine
Publication year - 2021
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.17580
Subject(s) - haemophilia , medicine , haemophilia b , haemophilia a , factor ix , clotting factor , coagulation , intensive care medicine , coagulopathy , immunology , pediatrics , surgery
Summary 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.