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Emicizumab (ACE910) is a bispecific antibody that binds to activated factor IX and factor X, substituting for factor VIII. In a prior study, emicizumab improved the clotting of factor VIII–depleted plasma and was well-tolerated by healthy persons (NEJM JW Oncol Hematol Jul 2016 and Blood 2016; 127:1633).
To determine whether emicizumab can prevent hemophilic bleeding, Japanese investigators conducted an open-label, nonrandomized, dose-escalation trial in 18 severe hemophiliacs who received 12 weekly subcutaneous injections of 0.3, 1.0, or 3.0 mg/kg. Eleven patients had factor VIII inhibitors.
Median annualized bleeding rates declined regardless of the dose administered, and no bleeding episodes occurred in 13 patients (72%), including 8 of th…