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Control of bleeding in patients with hemophilia requires the regular intravenous infusion of clotting factor concentrates, which is painful and expensive. An alternative approach is to enhance hemostasis by decreasing the levels of natural anticoagulants such as antithrombin.
To examine the safety and effectiveness of fitusiran, an RNA interference agent that specifically targets antithrombin, investigators conducted a dose-escalation study in 25 patients, 18 with hemophilia A and 7 with hemophilia B. Excluded were patients with thrombophilic disorders, liver dysfunction, or inhibitors. Fitusiran was given subcutaneously either weekly or monthly, and follow-up was for up to 112 days.
The maximum reduction of antithrombin was 89% from baseline…