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

Characterization of the Early Immune Response to Factor VIII

Characterization of the Early Immune Response to Factor VIII

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research
RESEARCHERS:
Glaivy Batsuli, MD, Emory University; Wallace Baldwin, MS, Emory University; Jasmine Ito, BS, Emory University; John Healey, BS, Emory University; Shannon Meeks, MD, Emory University; Seema Patel, PhD, Emory University
Overview of the clinical development of fitusiran

Overview of the clinical development of fitusiran

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Baisong Mei, MD, PhD, Sanofi; Naghmana Bajwa, MD, Sanofi; Fadi Shammas, MD, Sanofi; Salim Kichou, MD, Sanofi; Shauna Andersson, M.D., PhD, Sanofi
A Novel Approach for Rare Bleeding Disorders: Shielded Living Therapeutics

A Novel Approach for Rare Bleeding Disorders: Shielded Living Therapeutics

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research
RESEARCHERS:
Christine Carroll, BS, Sigilon Therapeutics; David Peritt, PhD, Sigilon Therapeutics; Devyn Smith, PhD, Sigilon Therapeutics; Deya Corzo, MD, Sigilon Therapeutics; Guillaume Carmona, PhD, Sigilon Therapeutics; Jared Sewell, PhD, Sigilon Therapeutics; Janet Huang, MS, Sigilon Therapeutics; Kevin Lai, PhD, Sigilon Therapeutics; Lauren Barney, PhD, Sigilon Therapeutics; Michaela Toland, BS, Sigilon Therapeutics; Michele McAuliffe, BS, Sigilon Therapeutics; Susan Drapeau, PhD, Sigilon Therapeutics; Rogerio Vivaldi, MD, MBA, Sigilon Therapeutics; Verna Zhao, BS, Sigilon Therapeutics
Analysis of Bleeding and Treatment Patterns in Children and Adolescents before and after Von Willebrand Disease Diagnosis Using Data from a US Medical Claims Database

Analysis of Bleeding and Treatment Patterns in Children and Adolescents before and after Von Willebrand Disease Diagnosis Using Data from a US Medical Claims Database

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Abioloa Oladapo, PhD, Takeda Pharmaceuticals, USA; Imrran Halari, MS, Charles River Associates; Jonathan Roberts, MD, Bleeding and Clotting Disorders Institute; Lynn Malec, MD, MSc, Blood Research Institute, Versiti; Robert Sidonio, MD, Emory University and Children's Healthcare of Atlanta; Sarah Hale, PhD, Takeda Pharmaceuticals, USA
Development of the WFH-ISTH Gene Therapy Registry

Development of the WFH-ISTH Gene Therapy Registry

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Barbara Konkle, MD, Bloodworks NW, Washington Center for Bleeding Disorders; Brian O’Mahony, NA, Irish Haemophilia Society; Cary Clark, NA, International Society on Thrombosis and Haemostasis Inc; Crystal Watson, NA, American Thrombosis and Hemostasis Network; Donna Coffin, MSc, World Federation of Hemophilia; Flora Peyvandi, MD, PhD, IRCCS Maggiore Hospital Milan and University of Milan; Lindsey A. George, MD, The Children's Hospital of Philadelphia; Glenn F. Pierce, MD, PhD, World Federation of Hemophilia; Alfonso Iorio, MD, McMaster University; Johnny Mahlangu, MD, Haemophilia Comprehensive Care Centre, University of the Witwatersrand, NHLS; Mayss Naccache, MSc, World Federation of Hemophilia; Mark W. Skinner, JD, Institute for Policy Advancement Ltd.; Steven W. Pipe, MD, University of Michigan, Pediatrics
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