Low Annualized Bleeding Rates in Phase 3 Studies of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects with Target Joints at Baseline
Introduction and Objectives: The phase 3 A-LONG and Kids A-LONG studies demonstrated the safety and efficacy of rFVIIIFc for the control and prevention of bleeding episodes in subjects with severe hemophilia A. This subgroup…
A Novel Anti-Tissue Factor Pathway Inhibitor Antibody, BAY 1093884, Restores Hemostasis in Induced Hemophilia A Rabbits With Reduced Thrombogenic Potential
Background: BAY 1093884 is a fully human monoclonal antibody against tissue factor pathway inhibitor developed as a bypass agent for hemophilia patients with inhibitors. It restores thrombin burst, leading to stable clot…
Leading an Active Lifestyle with Hemophilia B: Estimating the Bleeding Risk with Different FIX Treatment Regimens
Development of a novel patient-centered outcome measure in hemophilia using Goal Attainment Scaling
Background and Objective: Standard outcome measures in hemophilia, such as the annual bleeding rate, have inherent limitations in both clinical practice and in research, including lack of both sensitivity and personalization.…
Initial Observations From the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study: Patient-Reported Outcome Assessments in US Adults With Hemophilia
Objective: To assess pain and functional impairment through 5 patient-reported outcome (PRO) instruments in non-bleeding adults with hemophilia. Methods: Adult men with hemophilia (mild-severe) with history…
Pharmacokinetics, safety, and efficacy of pdFX, a new high-purity factor X concentrate: a phase 3 study in patients with moderate or severe hereditary factor X deficiency
Objective: To assess the pharmacokinetics (PK), safety, and efficacy of the first high- purity, plasma-derived factor X concentrate (pdFX) for on-demand treatment of bleeding episodes in a prospective, open-label, multicenter,…
Patient Preferences for FVIII and BAX 855: Results from the BAX 855 Pivotal Trial
Objective: BAX 855 is an extended half-life recombinant Factor VIII based on ADVATE. It was developed as an option to further personalize care and improve outcomes in people with hemophilia A. As a new therapy, it is important to…
Efficacy and safety of pdFX, a new high-purity factor X concentrate, in patients with mild to severe hereditary factor X deficiency undergoing surgery
Objective: To assess the safety and efficacy of treatment with the first high-purity plasma-derived factor X concentrate (pdFX) in 2 prospective, multicenter, open-label, nonrandomized phase 3 studies in subjects with hereditary…
My Life, Our Future: A “Genetics Day” to Facilitate Efficient Enrollment
Hemophilia B Patients Who Switch From rFIX to Extended Half-Life rFIX-Fc: A Retrospective Analysis of Cost using US Specialty Pharmacy Dispensing Data
Background: Until recently, the hemophilia B community has only had standard Factor IX (FIX-Fc) products as treatment choices. With the availability of additional standard rFIX products and extended half-life (EHL) rFIX-Fc, there…