NBDF showcases a wide array of findings from the latest in bleeding disorders research at our annual Bleeding Disorders Conference.

Understanding and finding symptomatic undiagnosed women
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models

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
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Factor X deficiency consumer education program’s inaugural year
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models

Ultrasound-mediated Therapeutic Gene Transfer for Hemophilia
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research

Do Hemophilia Treatment Centers Want Or Need A Regional Ethics Committee?
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Law/Ethics/Health Policy

Change in cost and units consumed by people with factor VIII and factor IX deficiency after switching from a standard half-life product to an extended half-life product
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Long-term clinical outcomes of rFVIIIFc prophylaxis in adults 50 years of age or older with severe hemophilia A
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Achievement of therapeutic levels of factor VIII activity following gene transfer with valoctocogene roxaparvovec (BMN 270): Long-term efficacy and safety results in patients with severe hemophilia A
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Annual Bleed Rates Compared Before and After Changing to Extended Half Life Products in Home Infusion Patients with severe hemophilia
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Long-term Benefit of BAY 81-8973 Prophylaxis in Children With Severe Hemophilia A: Interim Analysis of the LEOPOLD Kids Extension Study
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Impact of hemophilia on employment - Insights from the PROBE Study
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

Empowering the Future of Hemophilia Through Swimming (Poster Abstract)
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Psychosocial Issues

Effects of Factor VIII Prophylaxis on Vascular Remodeling and Synovial Gene Expression Changes Associated with Hemarthrosis in FVIII-Deficient Mice
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research

Efficacy of on-demand treatment of bleeding episodes in hemophilia B patients with extended half-life N9-GP in pivotal trials: an in-depth analysis of treatment
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Retrospective review of unplanned hospitalizations and perceived pain in children and adults with a diagnosis of factor ten deficiency receiving home infusions of commercially available factor ten
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
New Products

Persons With Hemophilia Reinforce Their Desire to be More Active: US Findings From an International Patient Survey
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

The Relationship of Hope and Self-Compassion with Quality of Life among Individuals with Bleeding Disorders
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Psychosocial Issues

Inhibitor Teams: building stronger connections and deeper learning
AWARDED/PRESENTED: 2018
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models

Efficacy, safety, and pharmacokinetics of a high-purity plasma-derived factor X (pdFX) concentrate in the prophylaxis of bleeding episodes in children <12 years with moderate to severe congenital factor X deficiency (FXD)
AWARDED/PRESENTED: 2017
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia A and B with and Without Inhibitors: Interim Results from a Phase 2 Extension Study
AWARDED/PRESENTED: 2017
GRANT/PROGRAM:
Bleeding Disorders Conference
New Products