Research Presented at Our Conference

Updated results from a dose-escalation study in adults with severe or moderate-severe hemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy: up to 1.5 years follow-up

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Wolfgang Miesbach, Karina Meijer, Michel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Giancarlo Castaman, Erhard Seifried, Joachim Schwaeble, Halvard Bonig, Christian Meyer, Federica Cattaneo, Eileen Sawyer, Frank Leebeek

Lessons Learned in the Assessment of Functional Status in US Adults With Hemophilia in the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study: Importance of More Formalized Assessment of Function in the Comprehensive Care Setting

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Quality of Life/Outcomes Research

RESEARCHERS:

Michelle Witkop, Grace Hernandez, Michael Recht, Michael Wang, Kimberly Baumann, David Cooper, Christine Kempton

A Feasibility and Usability Study of a Nursing Orchestrated, Customized 3 Dimensional Virtual Reality Environment in Children with Hemophilia Undergoing Routine Intravenous Procedures

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Charmaine Biega, Jeremy Patterson, Alice Grishchenko, Juan Lima, Rob Strouse, Pam Widener, Amy Dunn

Estimating the prevalence of symptomatic, undiagnosed von Willebrand disease: analysis of medical insurance claims data

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Robert Sidonio, Ayesha Zia, Dana Fallaize

Ethics of Compensated Plasma Donation

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Law/Ethics/Health Policy

RESEARCHERS:

James Crone, Mark Skinner

Efficacy, safety and pharmacokinetics of emicizumab (ACE910) prophylaxis in persons with hemophilia A with inhibitors: randomized, multicenter, open-label, phase 3 study (HAVEN 1)

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Johannes Oldenburg, Johnny Mahlangu, Benjamin Kim, Christophe Schmitt, Michael Callaghan, Guy Young, Elena Santagostino, Rebecca Kruse-Jarres, Claude Negrier, Craig Kessler, Nancy Valente, Elina Asikanius, Gallia Levy, Jerzy Windyga, Midori Shima

Depression levels in patients with Hemophilia and von Willebrand

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Psychosocial Issues

RESEARCHERS:

Ana Paola Abreu Bastar, Valeria Escobar Ruiz, María Fernanda Domínguez Ballesteros, Leydi Lizbhet Morales de la Cruz, María Laura Giselle Torres Chablé

Qualitative findings from bleeding disorders camp

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Psychosocial Issues

RESEARCHERS:

Sunnye Mayes, Osman Khan

Hereditary factor X (FX) deficiency in women and girls: treatment with a high purity plasma-derived factor X concentrate

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Roshni Kulkarni, Andra James, Miranda Norton, Amy Shapiro

Management of Hemophilia Carriers Around The Time of Their Delivery: Phenotypic Variation Requiring Customization of Management

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Women's Research

RESEARCHERS:

Marian Girgis, Philip Kuriakose, Laura Gusba

Centralized Inhibitor Testing in the United States: Laboratory Methods Used for the Community Counts Registry for Bleeding Disorders Surveillance

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Inhibitors

RESEARCHERS:

Amanda B. Payne, Dorothy Ellingsen, Jennifer Driggers, Brian Boylan, Fiona Bethea, Vanessa R. Byams, J. Michael Soucie, Connie H. Miller, Christopher J. Bean

Chronicles of Caring: Nursing Stories from the Heart of Hemophilia

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Quality of Life/Outcomes Research

RESEARCHERS:

Angela Lambing, Jessica Walker, Jennifer Harris, Judith Kauffman, Joan Osip, Linda Peacock

Real-World Pharmacy Dispensation and Expenditures Associated with Standard and Extended Half-Life Recombinant Factor VIII Products in Hemophilia A

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

New Products

RESEARCHERS:

Patrick F. Fogarty, José Alvir, Amit Chhabra, Dean Spurden, Margaret McDonald, Bartholomew J. Tortella, Andreas M. Pleil

Factors influencing uptake of evaluation among hemophilia carriers and potential carriers

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Women's Research

RESEARCHERS:

Elizabeth Parks, Lauren Lichten, Michelle Alabek

Real-World Specialty Pharmacy Dispensation and Expenditures Associated with Prophylactic Regimens Using Standard and Extended Half-Life Recombinant Factor IX Products in Severe Hemophilia B

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

New Products

RESEARCHERS:

Patrick F. Fogarty, José Alvir, Amit Chhabra, Dean Spurden, Margaret McDonald, Bartholomew J. Tortella, Andreas M. Pleil

Patient Reported Outcomes, Burdens and Experiences (PROBE) Study Data Visualization and Analysis

AWARDED/PRESENTED: 2017

GRANT/PROGRAM:

Bleeding Disorders Conference

Quality of Life/Outcomes Research

RESEARCHERS:

Alexandra Pastarnak, Randall Curtis, Neil Frick, Alfonso Iorio, Michael Nichol, Declan Noone, Brian O'Mahony, David Page, Jeffrey Stonebraker, Mark Skinner