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Br J Haematol. 2018 Apr;181(2):161-172. doi: 10.1111/bjh.15084. Epub 2018 Jan 23.

Recent advances in developing specific therapies for haemophilia.

Author information

1
Katherine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, UK.

Abstract

Haemophilia therapy has undergone very rapid evolution in the last 10 years. The major limitation of current replacement therapy is the short half-life of factors VIII and IX. These half-lives have been extended by the addition of various moieties, allowing less frequent infusion regimens. Entirely novel approaches have also entered the clinic, including a bispecific antibody that mimics factor VIII and strategies that rebalance the haemostatic mechanism by reducing antithrombin through inhibition of synthesis. These two treatments are available by subcutaneous injection at infrequent intervals and both can be used in patients with neutralising antibodies (inhibitors). Finally, a cure may be on the horizon with preliminary evidence of success for gene therapy in haemophilia B and A.

KEYWORDS:

bleeding disorders; factor IX; factor VIII; gene therapy; haemophilia; therapy

PMID:
29359795
DOI:
10.1111/bjh.15084
[Indexed for MEDLINE]

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