What Symptoms of Hemophilia Most Impact Quality of Life – A Quantitative Survey of People Living with or Caring for Someone with Hemophilia A
Objectives: To better understand what symptoms beyond bleeds are experienced, as well as the depth of impact of these symptoms and how they uniquely impact people living with hemophilia on…
Efficacy, safety and pharmacokinetics of emicizumab (ACE910) prophylaxis in persons with hemophilia A with inhibitors: randomized, multicenter, open-label, phase 3 study (HAVEN 1)
Objectives: Emicizumab, a bispecific humanized monoclonal antibody in development to address unmet medical needs in persons with hemophilia A with inhibitors (PwHAwI), bridges FIXa and FX to replace the function of missing FVIIIa…
Efficacy, safety and pharmacokinetics of once-weekly prophylactic emicizumab (ACE910) in pediatric persons (<12 years) with hemophilia A with inhibitors: interim analysis of single-arm, multicenter, open-label, phase 3 study (HAVEN 2)
Objectives: Emicizumab, a novel bispecific humanized monoclonal antibody promotes coagulation by bridging FIXa and FX to replace the function of missing activated FVIII, and has potential to address unmet medical needs in…
Living with hemophilia B: examining quality of life and associated characteristics in the Hemophilia Utilization Group Studies (HUGS Vb) cohort
Objective: To identify sociodemographic, clinical and treatment characteristics associated with the quality of life (QoL) of individuals with hemophilia B (HB) using longitudinal data in the HUGS Vb cohort. Methods:…
Qualitative findings from bleeding disorders camp
Camping programs for individuals with chronic illness are increasingly common. Unfortunately, few studies have been conducted to empirically evaluate whether camping programs are meeting their intended goals or having the positive outcomes that are…
Staying on TRAQ: Determining transition readiness from pediatric to adult care in adolescents and young adults with hemophilia
Advancements in medical care for the hemophilia patients has created the need for an active and intentional process of transition from pediatric oriented health care to adult oriented health care. Instruments to measure transition “readiness” have…
My Life, Our Future: Development of the World’s Largest Genetic Research Repository for Hemophilia
Objective: My Life, Our Future (MLOF) seeks to advance understanding of hemophilia by developing the MLOF Research Repository, a resource for use in scientific study. To strengthen the database and improve clinical care for…
A Multicenter, Retrospective Data Collection Study on the Compassionate Use of a Plasma-Derived Factor X Concentrate to Treat Patients with Hereditary Factor X Deficiency
Objective: Report results of an open-label international study that collected retrospective data on compassionate use of high-purity plasma-derived FX concentrate (pdFX) in subjects with hereditary factor X (FX) deficiency (FXD…
Pharmacokinetics, Efficacy, and Safety of High-Purity Factor X for Prophylactic Treatment of Hereditary Factor X Deficiency
Discrepant Hemophilia A: Single Institution Experience