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Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A
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Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A
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Journal Article
Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A
2016
Overview
Emicizumab is a humanized bispecific antibody that mimics the cofactor function of factor VIII. In a dose-escalation study in Japanese persons with hemophilia A, including those with factor VIII inhibitors, emicizumab markedly reduced the number of bleeding episodes.
Hemophilia A is a serious bleeding disorder caused by a deficiency of clotting factor VIII. Approximately 50% of patients have severe hemophilia A,
1
defined as less than 1% residual factor VIII activity (<1 IU per deciliter).
2
Such patients have severe bleeding from early childhood, and without appropriate treatment, recurrent bleeding into joints can lead to irreversible hemoarthropathy.
3
,
4
Standard treatment for hemophilia A includes regular prophylaxis and episodic treatment with recombinant or plasma-derived factor VIII. The goals of prophylaxis with factor VIII are to increase factor VIII activity to at least a moderate level (1 to 5 IU per deciliter) . . .
Publisher
Massachusetts Medical Society
Subject
/ Adult
/ Antibodies, Bispecific - blood
/ Antibodies, Bispecific - pharmacology
/ Antibodies, Bispecific - therapeutic use
/ Antibodies, Monoclonal, Humanized - blood
/ Antibodies, Monoclonal, Humanized - pharmacology
/ Antibodies, Monoclonal, Humanized - therapeutic use
/ Bleeding
/ Child
/ Children
/ Clotting
/ Dose-Response Relationship, Drug
/ Factor VIII - antagonists & inhibitors
/ Factor VIII - therapeutic use
/ Hemophilia A - complications
/ Hemorrhage - prevention & control
/ Humans
/ Injections, Subcutaneous - adverse effects
/ Male
/ Mimicry
/ Patients
