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Review
. 2016 May;141 Suppl 2(Suppl 2):S28-30.
doi: 10.1016/S0049-3848(16)30359-0.

Targeting TFPI for hemophilia treatment

Julie A Peterson  1 , Susan A Maroney  1 , Alan E Mast  2
Affiliations
Review

Targeting TFPI for hemophilia treatment

Julie A Peterson et al. Thromb Res. 2016 May.

Abstract

Hemophilia is a severe bleeding disorder treated by infusion of the missing blood coagulation protein, factor VIII or factor IX. The discovery and characterization of the anticoagulant protein tissue factor pathway inhibitor (TFPI) led to the realization that inhibition of TFPI activity could restore functional hemostasis through the extrinsic blood coagulation pathway in a manner that does not require the activity of factors VIII or IX. There are currently several therapeutic agents that inhibit TFPI in development for treatment of hemophilia. A comprehensive understanding of TFPI structure, biochemistry, and cellular expression is necessary to understand how it modulates bleeding in hemophilia and the physiological impact of therapeutic agents targeting TFPI.

Keywords: Hemophilia; TFPI; blood coagulation; factor IX; factor VIII; prothrombinase.

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