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.