B. Pollio
Abstract
In 2007 Manco Johnson's pivotal randomized trials established that primary prophylaxis is the standard of care for people with hemophilia. In just 15 years, hemophilia has seen an extraordinary development of increasingly innovative molecules. By the end of 2021, an extraordinary therapeutic arsenal of at least a dozen factors designed to reduce immunogenicity and modify clearance in order to increase protection and shorten dosing intervals is available for treatment of hemophilia.
The non-factor replacement therapies are novel approaches restoring generation of thrombin by means of monoclonal antibodies mimicking FVIII, antithrombin interference RNA therapy and monoclonal antibodies directed against the tissue factor pathway inhibitor (anti-TFPI).
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