Concizumab Subcutaneous Prophylaxis Improves Health-Related Quality-Of-Life Measures In Patients With Congenital Hemophilia With Inhibitors: Phase 2 Trial Results

BLOOD(2019)

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摘要
Introduction Concizumab is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody in clinical development for the subcutaneous prophylactic treatment of hemophilia patients. We present exploratory analyses assessing the impact of concizumab prophylaxis on health-related quality-of-life (HRQoL) in hemophilia A and B with inhibitor (HAwI/HBwI) patients compared with recombinant activated factor VII (rFVIIa) on-demand treatment in a phase 2 trial (NCT03196284; explorer4). HRQoL is impaired in patients with hemophilia relative to the general population and this impairment is more pronounced among patients with inhibitors, including HBwI patients for whom treatment options are particularly limited.
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