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

Real-world treatment patterns, health outcomes, and healthcare resource use among persons with hemophilia A

Real-world treatment patterns, health outcomes, and healthcare resource use among persons with hemophilia A

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Brandon T Suehs, PharmD, PhD, Humana Healthcare Research, Inc.; Daniel Cornett, PharmD, BCPS, Humana, Inc.; Eleanor O Caplan, PharmD, PhD, Humana Healthcare Research, Inc.; Rahul Khairnar, MS, PhD, Genentech, Inc.; Marina Sehman, PharmD, CSP, Humana, Inc.; Anisha. M Patel, PhD, Genentech, Inc.; Rich DeClue, PhD, MPH, Humana Healthcare Research, Inc.; Tiffany Fair Shaffer, RN, BSN, Humana Healthcare Research, Inc.
The World Federation of Hemophilia World Bleeding Disorders Registry: A Two-year Update

The World Federation of Hemophilia World Bleeding Disorders Registry: A Two-year Update

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Barbara Konkle, MD, Bloodworks Northwest; Cedric Hermans, MD Ph.D., St-Luc University Hospital; Donna Coffin, M.Sc., World Federation of Hemophilia; Declan Noone, M.Sc., European Haemophilia Consortium; Emily Ayoub, Ph.D., World Federation of Hemophilia; Ellia Tootoonchian, M.Sc., World Federation of Hemophilia; Glenn Pierce, MD, World Federation of Hemophilia; Alfonso Iorio, MD PhD FRCPC, McMaster University; Jamie O'Hara, M.Sc., HCD Economics; Mayss Naccache, M.Sc., World Federation of Hemophilia; Saliou Diop, MD, Cheikh Anta Diop University; Toong Youttananukorn, Ph.D., World Federation of Hemophilia; Vanessa Byams, DrPH, Centers for Disease Control and Prevention
Progress Update on the Development of Etranacogene Dezaparvovec (AMT-061) in Severe or Moderately Severe Hemophilia B

Progress Update on the Development of Etranacogene Dezaparvovec (AMT-061) in Severe or Moderately Severe Hemophilia B

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Annette von Drygalski, University of California; Eileen K Sawyer, uniQure biopharma B.V.; Esteban Gomez, Phoenix Children's Hospital; Frank W.G. Leebeek, Erasmus University Medical Center; Giancarlo Castaman, Azienda Ospedaliera Universitaria Careggi; Adam Giermasz, University of California Davis; Karina Meijer, University Medical Center Groningen; Susan Lattimore, Oregon Health & Science University; Michiel Coppens, Academic Medical Center; Wolfgang Miesbach, university Hospital Frankfurt; Nigel S Key, University of North Carolina; Peter Kampmann, Rigshospitalet; Robert Gut, uniQure biopharma B.V; Roger Schutgens, University Medical Center; Michael Recht, Oregon Health & Science University; Robert Klamroth, Vivantes Klinikum; Steven W Pipe, University of Michigan
An analysis of fatalities in persons with congenital hemophilia A (PwcHA) reported in the FDA Adverse Event Reporting System (FAERS) database

An analysis of fatalities in persons with congenital hemophilia A (PwcHA) reported in the FDA Adverse Event Reporting System (FAERS) database

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Aijing Shang, MD, PhD, F. Hoffmann-La Roche Ltd; Tiffany Chang, MD, MAS, Genentech, Inc.; Christian De Ford, PharmD, PhD, F. Hoffmann-La Roche Ltd; Fabian Sanabria, MD, F. Hoffmann-La Roche Ltd; Richard H. Ko, MD, MHS, MS, Genentech, Inc.; Peter Kuebler, PharmD, Genentech, Inc.
Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design

Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Annemieke J. Willemze, MD, Sanofi; Annette von Drygalski, MD, PharmD, University of California San Diego; Barbara A. Konkle, MD, Bloodworks Northwest; Bent Winding, MD, Sobi AB; John Pasi, MB, ChB, PhD, Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry; Marek Demissie, MD, PhD, Sanofi; Roshni Kulkarni, MD, Michigan State University; Stacey Poloskey, MD, Sanofi; Stella Lin, PhD, Sanofi; Suresh Katragadda, PhD, Sanofi; Toshko Lissitchkov, MD, Specialized Hospital for Active Treatment of Hematological Diseases
Characteristics of persons with hemophilia A treated with emicizumab with or without factor VIII inhibitors

Characteristics of persons with hemophilia A treated with emicizumab with or without factor VIII inhibitors

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Ibrahim Abbass, PhD, Genentech, Inc.; Arash Mahajerin, MD, MSc, CHOC Children’s Hospital; Rahul Khairnar, MS, PhD, Genentech, Inc.; Craig S. Meyer, PhD, MPH, MS, Genentech, Inc.; Anisha M. Patel, PhD, Genentech, Inc.; Karina Raimundo, MS, Genentech, Inc.; Erru Yang, MS, Genentech, Inc.
Non-severe hemophilia is not benign? - Insights from the PROBE Study

Non-severe hemophilia is not benign? - Insights from the PROBE Study

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Randall Curtis, MBA, Factor VIII Computing; Chatree Chai-Adisaksopha, MD, Department of Medicine, McMaster University; Department of Internal Medicine, Chiang Mai University; Neil Frick, MS, National Hemophilia Foundation; Michael Nichol, Ph.D., University of Southern California, Sol Price School of Public Policy; Declan Noone, MEng, Health Decisions Consultants; European Haemophilia Consortium; Federico Germini, MD, Department of Medicine, McMaster University; Department of Health Research Methods, Evidence, and Impact, McMaster University; Brian O'Mahony, Irish Haemophilia Society; Trinity College Dublin; David Page, Canadian Hemophilia Society; Jeff Stonebraker, Ph.D., North Carolina State University, Poole College of Management; Alfonso Iorio, MD Ph.D. FRCP, Department of Health Research Methods, Evidence, and Impact; McMaster-Bayer Endowed Research Chair in Clinical Epidemiology of Congenital bleeding Disorders, Department of Medicine, McMaster; Mark Skinner, JD, Institute for Policy Advancement Ltd.; Department of Health Research Methods, Evidence, and Impact, McMaster University
The Moti-VIII Study – Factors for Empowering Mobility and Well-being in Hemophilia A

The Moti-VIII Study – Factors for Empowering Mobility and Well-being in Hemophilia A

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Anna Biberger, BSc, Kantar; Christina Ashburner, BSc, Centre for Inherited Blood Disorders; Ceri Hirst, PhD, Bayer; Jessica Charlet, BSc, MSc, PhD, Bayer; Spencer Dunn, MSW, Center for Inherited Blood Disorders; Sharonne Herbert, MD, CHOC
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.