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

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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Peer Support/Outreach/Integration Models
Author(s):
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)

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Collaboration/Team Models
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Collaboration/Team Models
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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.
On My Own, A Pilot Transition Program for Teens

On My Own, A Pilot Transition Program for Teens

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Collaboration/Team Models
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Biomedical/Coagulation Research
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Biomedical/Coagulation Research
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Quality of Life/Outcomes Research
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Quality of Life/Outcomes Research
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Quality of Life/Outcomes Research
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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

Year: 2020
Grants:
Bleeding Disorders Conference
Tags:
Clinical Research/Clinical Trials
Author(s):
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.