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

Adding the patient's voice to a hemophilia-specific goal menu to facilitate Goal Attainment Scaling: a qualitative study

Adding the patient's voice to a hemophilia-specific goal menu to facilitate Goal Attainment Scaling: a qualitative study

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Quality of Life/Outcomes Research
RESEARCHERS:
Amanda Simpson, BBA, DGI Clinical; Jonathan Roberts, MD, Bleeding and Clotting Disorders Institute; Justin Stanley, BS, DGI Clinical; Kari Knox, BS, DGI Clinical; Kenneth Rockwood, MD, DGI Clinical; Michael Denne, PharmD, Takeda Pharmaceuticals, USA
The Impact of Novel Hemophilia Treatment Products on Inhibitor Testing for the Community Counts Registry for Bleeding Disorders Surveillance

The Impact of Novel Hemophilia Treatment Products on Inhibitor Testing for the Community Counts Registry for Bleeding Disorders Surveillance

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Inhibitors
RESEARCHERS:
Amanda B. Payne, PhD, MPH, Centers for Disease Control and Prevention; Connie H. Miller, PhD, Centers for Disease Control and Prevention; Christopher J. Bean, PhD, Centers for Disease Control and Prevention; Jennifer Driggers, BS, Centers for Disease Control and Prevention; Karon Abe, PhD, Centers for Disease Control and Prevention; Brian Boylan, MS, Centers for Disease Control and Prevention; Glenn P. Niemeyer, PhD, Centers for Disease Control and Prevention; Vanessa R. Byams, DrPH, MPH, Centers for Disease Control and Prevention
Utilization of Telehealth for Home Infusion Teaching and Support in the COVID Era

Utilization of Telehealth for Home Infusion Teaching and Support in the COVID Era

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Peer Support/Outreach/Integration Models
RESEARCHERS:
Amy Pegman, RN, University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital; Caitlin Tucker, RN,BSN, University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital; Ryan Summers, RN, BSN, University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital; Sanjay Ahuja, MD, MSc MBA, University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital; Susan Hunter, RN,BSN, University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital
A contemporary framework for understanding mortality in people with congenital hemophilia A (PwcHA)

A contemporary framework for understanding mortality in people with congenital hemophilia A (PwcHA)

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Clinical Research/Clinical Trials
RESEARCHERS:
Tiffany Chang, MD, MAS, Genentech, Inc.; Charles R.M. Hay, MbChB, MD, FRCP, FRCPath, UK National Haemophilia Database and Manchester Royal Infirmary; Fabian Sanabria, MD, F. Hoffmann-La Roche Ltd; Flora Peyvandi, MD, PhD, IRCCS Ca’ Granda Foundation, Ospedale Maggiore Policlinico; Glenn F. Pierce, MD, PhD, World Federation of Hemophilia; Johnny N. Mahlangu, BSc, MBBCh, University of the Witwatersrand and NHLS; Richard H. Ko, MD, MHS, MS, Genentech, Inc.; Peter Kuebler, PharmD, Genentech, Inc.; Rebecca Kruse-Jarres, MD, University of Washington and Washington Center for Bleeding Disorders, Bloodworks Northwest; Steven W. Pipe, MD, University of Michigan
The Need for Comprehensive Care for Persons with Chronic Platelet Disorders

The Need for Comprehensive Care for Persons with Chronic Platelet Disorders

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Collaboration/Team Models
RESEARCHERS:
Chanel J Unzicker, RN, BSN, Bleeding and Clotting Disorders Institute; Kristin Ansteatt, FNP-BC, Bleeding and Clotting Disorders Institute; Marsha L Hurn, MSW, LSW, Bleeding and Clotting Disorders Institute; Michael D Tarantino, MD, Bleeding and Clotting Disorders Institute
Final Results of PUPs B-LONG Study: Evaluating Safety and Efficacy of rFIXFc in Previously Untreated Patients With Hemophilia B

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

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Clinical Research/Clinical Trials
RESEARCHERS:
Anna Klukowska, MD, MedIcal University of Warsaw; Antoine Rauch, MD, PhD, Lille University Hospital Center (CHU) Lille; Amy Shapiro, MD, Indiana Hemophilia and Thrombosis Center; Bent Winding, MD, Swedish Orphan Biovitrum AB; Deepthi Jayawardene, MD, Sanofi; Beatrice Nolan, MD, Our Lady's Children's Hospital; Julie Curtin, MBBS, PhD, The Children's Hospital at Westmead; Kathelijn Fischer, MD, PhD, University Medical Center Utrecht; Margaret Ragni, MD, MPH, University of Pittsburgh Medical Center; Michael Recht, MD, PhD, Oregon Health and Science University; Raina Liesner, MD, Great Ormond Street Hospital; Sriya Gunawardene, MD, Sanofi; Stacey Poloskey, MD, Sanofi
3 apps in 1: MyCBDR, myWAPPS and myPROBE

3 apps in 1: MyCBDR, myWAPPS and myPROBE

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Collaboration/Team Models
RESEARCHERS:
David Page, Canadian Hemophilia Society; Chatree Chai-Adisaksopha, MD, Department of Medicine, McMaster University; Department of Internal Medicine, Chiang Mai University; Randall Curtis, MBA, Factor VIII Computing; Neil Frick, MS, National Hemophilia Foundation; 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; Michael Nichol, Ph.D., University of Southern California, Sol Price School of Public Policy; Declan Noone, MEng, Health Decisions Consultants; European Haemophilia Consortium; Brian O'Mahony, Irish Haemophilia Society; Trinity College Dublin; Jeff Stonebraker, Ph.D., North Carolina State University, Poole College of Management; Federico Germini, MD, Department of Medicine, McMaster University; Department of Health Research Methods, Evidence, and Impact, McMaster University; Mark Skinner, JD, Institute for Policy Advancement Ltd.; McMaster University Department of Health Research Methods, Evidence, and Impact, McMaster University
Bone and joint health markers in persons with hemophilia A treated with emicizumab in the HAVEN 3 clinical trial

Bone and joint health markers in persons with hemophilia A treated with emicizumab in the HAVEN 3 clinical trial

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Clinical Research/Clinical Trials
RESEARCHERS:
Joanne I. Adamkewicz, PhD, Genentech, Inc.; Anna Kiialainen, PhD, F. Hoffmann-La Roche Ltd; Tiffany Chang, MD, MAS, Genentech, Inc.; Christine L. Kempton, MD, MSc, Emory University School of Medicine; Giancarlo Castaman, MD, Careggi University Hospital; Markus Niggli, PhD, F. Hoffmann-La Roche Ltd; Ido Paz-Priel, MD, Genentech, Inc.
Retrospective review of Hemophilia patients before and after treatment with Emicizumab

Retrospective review of Hemophilia patients before and after treatment with Emicizumab

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Quality of Life/Outcomes Research
RESEARCHERS:
Aisha Hussain, PharmD, CVS Health; Amy Thompson, PharmD, MBA, CVS Health; Anna A Divney, DrPH, MPH, CVS Health; Elisea Avalos-Reyes, PhD, CVS Health; Natalie Watkins, PharmD, CVS Health; Nishta Patel, PharmD, CVS Health
On My Own, A Pilot Transition Program for Teens

On My Own, A Pilot Transition Program for Teens

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
RESEARCH CATGEORY:
Collaboration/Team Models
RESEARCHERS:
Amanda Fridley, Pharm D, Cincinnati Children's Hospital Medical Center; Anna Worpenberg, MSW, LSW, Cincinnati Children's Hospital Medical Center; Helen Lamping, Associates, Tri-State Bleeding Disorder Foundation; Julie Doyle, BSN, RN, Cincinnati Children's Hospital Medical Center; Julie Hendrickson, BSN, RN, CPN, Cincinnati Children's Hospital Medical Center; Lisa Littner, MPH, MSW, LISW-S, CHES, Cincinnati Children's Hospital Medical Center; Mary Busam, Pharm D, Cincinnati Children's Hospital Medical Center; Molly Mays, MPT, Cincinnati Children's Hospital Medical Center; Shanna Korn, BA, Cincinnati Children's Hospital Medical Center
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
RESEARCH CATGEORY:
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
RESEARCH CATGEORY:
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
RESEARCH CATGEORY:
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
RESEARCH CATGEORY:
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
RESEARCH CATGEORY:
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
RESEARCH CATGEORY:
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.