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Hamostaseologie 2021; 41(04): 247-256
DOI: 10.1055/a-1424-7900
Review Article

Nonfactor Therapies: New Approaches to Prophylactic Treatment of Haemophilia

Pratima Chowdary
1   Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, United Kingdom
› Author Affiliations Funding This work was funded by the Royal Free Charity TF 35.
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Abstract

For several decades, the treatment of haemophilia has relied on factor replacement therapy, which restores haemostasis by replacing the missing coagulation factor. In recent years, novel alternative therapies for the treatment of haemophilia in patients with and without inhibitors have been developed. These emergent therapies promote haemostasis by mimicking coagulation factors or inhibiting natural anticoagulants. They provide a less invasive route of administration (i.e. subcutaneous) and some offer reduced frequency of dosing (i.e. every 2 weeks, monthly) compared with the majority of factor replacement therapies, and thus have the potential to simplify treatment, increase adherence and subsequently improve outcomes for patients. Their introduction has transformed the care of haemophilia patients with inhibitors to factor VIII, with similar expectation for haemophilia B patients with inhibitors. However, these therapies also come with several new challenges including their limitation to prophylactic treatment, the observed increased incidence of thrombosis, or their impact on the natural history of the disease and potential disruption of existing treatment guidelines like the use of immune tolerance induction. Moreover, questions remain regarding the long-term impact of non-replacement therapies on joint health as well as the optimal strategy to manage breakthrough bleeds in patients with inhibitors.

Keywords

coagulation - tissue factor pathway inhibitor - monoclonal antibody - RNA interference - activated protein C inhibitor


Publication History

Received: 25 November 2020

Accepted: 10 March 2021

Article published online:
29 June 2021

© 2021. Thieme. All rights reserved.

Georg Thieme Verlag KG
Rüdigerstraße 14, 70469 Stuttgart, Germany

 

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