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

A US payer database algorithm to identify clinical profiles of hemophilia B for burden of illness assessment

A US payer database algorithm to identify clinical profiles of hemophilia B for burden of illness assessment

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Arielle G. Bensimon, PhD, Analysis Group, Inc.; Eileen K. Sawyer, PhD, uniQure, Inc.; Eric Q. Wu, PhD, Analysis Group, Inc.; Iryna Bocharova, BA, Analysis Group; Nanxin (Nick) Li, PhD, uniQure, Inc.; Tyler W. Buckner, MD, MSc, Hemophilia and Thrombosis Center, University of Colorado School of Medicine
Behavior and cognition in children and young adults with hemophilia A or B: an update on developmental outcome

Behavior and cognition in children and young adults with hemophilia A or B: an update on developmental outcome

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Cathy Buranahirun, PsyD, Keck School of Medicine, University of Southern California/Children’s Hospital Los Angeles; Christine Mrakotsky, PhD, Boston Children’s Hospital/Harvard Medical School; Cara Hannemann, PsyD, Indiana Hemophilia and Thrombosis Center; David L. Cooper, MD, MBA, Novo Nordisk Inc.; Greta N. Wilkening, PsyD, PhD, Children’s Hospital Colorado; Kevin A. Shapiro, MD, PhD, Cortica Healthcare; Karin S. Walsh, PsyD, Children’s National Hospital; Milan Geybels, PhD, Novo Nordisk A/S; Madhvi Rajpurkar, MD, Carman and Ann Adams Department of Pediatrics, Children’s Hospital of Michigan/Wayne State University; Pamela Ventola, PhD, Cogstate; Stacy E. Croteau, MD, MMS, Boston Children’s Hospital/Harvard Medical School
A Review of Current Patient Reported Outcome Measures Used to Assess Mental Health in People with Hemophilia

A Review of Current Patient Reported Outcome Measures Used to Assess Mental Health in People with Hemophilia

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Elizabeth Clearfield, MHS, Center for Medical Technology Policy; Ellen M. Janssen, PhD, Center for Medical Technology Policy; Mark W. Skinner, JD, Institute for Policy Advancement, Ltd.; Michelle Witkop, DNP, FNP-BC, National Hemophilia Foundation; Hsing-Yuan (Susan) Chang, MD, MPH, Center for Medical Technology Policy; Susan Reed, MA, Center for Medical Technology Policy
Supporting patient voice to inform healthcare decision-making: a discrete choice experiment on disability paradox in hemophilia

Supporting patient voice to inform healthcare decision-making: a discrete choice experiment on disability paradox in hemophilia

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Antony Martin, BSc MSc PhD, HCD Economics; Brendan Mulhern, BSc MSc, University of Technology Sydney; Brian O'Mahony, N/A, Irish Haemophilia Society; Diane Nugent, MD, CHOC Children's Hospital; Eileen Sawyer, PhD, uniQure Inc; George Morgan, BSc MSc, HCD Economics; Jamie O'Hara, BSc MSc, HCD Economics; Mark Skinner, JD, Institute for Policy Advancement, Ltd.; Michelle Witkop, DNP, National Hemophilia Federation; Nick Li, PhD, uniQure Inc; Tyler Buckner, MD, MSc, University of Colorado School of Medicine
An ECHO’d Practice: Utilizing Tele-Mentoring for Enhanced Data Quality Across One Hemophilia Treatment Center Region

An ECHO’d Practice: Utilizing Tele-Mentoring for Enhanced Data Quality Across One Hemophilia Treatment Center Region

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Collaboration/Team Models
RESEARCHERS:
Christina Ashburner, B.A., Center for Inherited Blood Disorders; Judith Baker, DrPH, MHSA, Center for Inherited Blood Disorders; Lisa Preciado, N/A, Hemostasis and Thrombosis Center of Nevada; Nicole Crook, RN, Center for Inherited Blood Disorders; Rajalakshmi Ganapathy, B.A., Center for Inherited Blood Disorders
Characterization and management of patients with mild or moderate hereditary factor X deficiency: a retrospective chart review

Characterization and management of patients with mild or moderate hereditary factor X deficiency: a retrospective chart review

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Cynthia Sabo, NP, Children's Hospital of Michigan, Detroit; Meera Chitlur, MD, Wayne State University School of Medicine and Children's Hospital of Michigan; Suchitra Acharya, MD, Cohen Children's Medical Center, Northwell Health, Zucker School of Medicine at Hofstra/Northwell
A single administration of AAV5-hFIX in newborn, juvenile and adult mice leads to stable hFIX expression up to 18 months after dosing

A single administration of AAV5-hFIX in newborn, juvenile and adult mice leads to stable hFIX expression up to 18 months after dosing

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research
RESEARCHERS:
Jaap Twisk, uniQure Biopharma B.V.; Liesbeth Heijink, uniQure Biopharma B.V.; Lisa Spronck, uniQure Biopharma B.V.; Martin de Haan, uniQure Biopharma B.V.; Richard van Logtenstein, uniQure Biopharma B.V.; Sander van Deventer, uniQure Biopharma B.V.; Valerie Ferreira, uniQure Biopharma B.V.
Treatments and Clinical Outcomes of Bleeding Related to Pregnancy, Surgery, or Spontaneous or Traumatic Bleeds in Women and Girls With Factor VIII and IX Deficiency: Results From a Retrospective Chart Review

Treatments and Clinical Outcomes of Bleeding Related to Pregnancy, Surgery, or Spontaneous or Traumatic Bleeds in Women and Girls With Factor VIII and IX Deficiency: Results From a Retrospective Chart Review

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Women's Research
RESEARCHERS:
Ateefa Chaudhury, MD, Center For Inherited Blood Disorders; Elisa Tsao, PhD, Sanofi; Justyna Tymoszczuk, MS, Sanofi; Mariana Oviedo Ovando, PhD, ICON plc; Nisha Jain, MD, Sanofi; Robert Sidonio, MD, Emory University School of Medicine and Children’s Healthcare of Atlanta; Roshni Kulkarni, MD, Michigan State University
Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients With Hemophilia A

Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients With Hemophilia A

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Amy Dunn, MD, Nationwide Children's Hospital; Bent Winding, MD, Swedish Orphan Biovitrum AB; Christoph Königs, MD, University Hospital Frankfurt; Deepthi Jayawardene, MS, Sanofi; Beatrice Nolan, MD, Our Lady's Children's Hospital; Manuel Carcao, MD, The Hospital for Sick Children; Margareth C. Ozelo, MD, PhD, University of Campinas; Michele Schiavulli, MD, A.O.R.N. Santobono-Pausilipon; Raina Liesner, MD, Great Ormond Street Hospital; Roshni Kulkarni, MD, Michigan State University; Simon A. Brown, MBBS, MD, Queensland Children’s Hospital; Sriya Gunawardena, MD, Sanofi; Sutirtha Mukhopadhyay, MBBS, Sanofi
Summary of thrombotic or thrombotic microangiopathy events in persons with hemophilia A taking emicizumab

Summary of thrombotic or thrombotic microangiopathy events in persons with hemophilia A taking emicizumab

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Kirsten Balogh, NP, MPH, Genentech, Inc.; Tiffany Chang, MD, MAS, Genentech, Inc.; Fabian Sanabria, MD, F. Hoffmann-La Roche Ltd; Katya Moreno, MD, F. Hoffmann-La Roche Ltd; Richard H. Ko, MD, Genentech, Inc.; Peter Kuebler, PharmD, Genentech, Inc.; Lucy Lee, PhD, Genentech, Inc.; Eunice Tzeng, PhD, Genentech, Inc.
Assessing and Responding to the Oral Health Care Needs of Adults in a Bleeding Disorders Population

Assessing and Responding to the Oral Health Care Needs of Adults in a Bleeding Disorders Population

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Joanna Larson, FNP-C, University of Texas Health Science Center at Houston; Megan Ullman, MPH, University of Texas Health Science Center at Houston; Michael M. Chan, DDS, University of Texas Health Science Center at Houston; Miguel Escobar, MD, University of Texas Health Science Center at Houston
Longitudinal trends of patient-focused programs in the bleeding disorders community from 2013-20: a retrospective analysis of Hemophilia Alliance Foundation grants

Longitudinal trends of patient-focused programs in the bleeding disorders community from 2013-20: a retrospective analysis of Hemophilia Alliance Foundation grants

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models
RESEARCHERS:
Amy Marquez, MS, Fairview Health Services; Anjali Sharathkumar, MBBS, MD, MS, University of Iowa; Audra Ames, PhD, Hemophilia Alliance Foundation; Brenda Riske, MS, MBA, MPA, Hemophilia Alliance Foundation; Crystal Sallans, LCSW, Texas Children’s Hematology Center; Donald Akers, JD, Hemophilia Alliance Foundation; Grant Hiura, MPH, Columbia University Irving Medical Center; Joseph Pugliese, BS, Hemophilia Alliance Foundation; Laurel Pennick, MSSW, LCSW, Arizona Hemophilia and Thrombosis Center; Michael Craciunoiu, EdM, National Hemophilia Foundation; Maria Manahan, MS, Hemophilia Alliance Foundation; Susan Karp, RN, MS, Hemophilia Alliance Foundation; Stephanie Raymond, BS, Cascade Hemophilia Consortium
Unmet Needs in Women with Severe Von Willebrand Disease

Unmet Needs in Women with Severe Von Willebrand Disease

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Women's Research
RESEARCHERS:
Beverly Schaefer, MD, WNY BloodCare, University at Buffalo, Roswell Park Cancer Institute; Jeanette Cesta, BS, VWD Connect Foundation; Shaveta Malik, MD, University at Buffalo, WNY BloodCare