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
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
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
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
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
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
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
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.
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
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
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
NHF Builds a Community-Driven National Research Blueprint for Inherited Bleeding Disorders

NHF Builds a Community-Driven National Research Blueprint for Inherited Bleeding Disorders

Year: 2020
Grants:
N/A
Collaboration/Team Models
Author(s):
Michelle Witkop, DNP, National Hemophilia Foundation; Michael Recht, MD, PhD, American Thrombosis and Hemostasis Network;
Donna DiMichele, MD, Consulting, LLC; Leonard Valentino, MD, National Hemophilia Foundation
  • The inherited bleeding disorders (IBD) community has witnessed significant advances in recent years thanks to novel therapeutic advances and technologies and improved diagnostic proficiency.

  • Yet important gaps persistent, particularly for those with rare disorders and underserved populations, including women with IBD.

  • A new initiative led by the National Hemophilia Foundation (NHF) and shaped by the voices of the patient community is underway to address this gap.

  • Our goal: to design and implement a national research blueprint that outlines actionable strategies to address the most important needs within the community and opportunities to accelerate progress through coordinated collaboration.
     

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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
Michael H. White

Michael H. White

Year:
-
Grants:
NHF-Takeda Clinical Fellowship
Author(s):
Michael H. White
Dr. Michael H. White is a pediatric hematology and oncology fellow at Emory University/CHOA. He earned his undergraduate degree in biology from Harding University and completed his medical degree at the University of Texas Southwestern Medical School. He went on to complete his general pediatrics residency training at Vanderbilt University in Nashville, TN. He is currently pursuing a Master of Science degree in Clinical Research at Emory University and has received a TL1 research training grant (also known as a "Linked Training Award"), through the National Institutes of Health for his focus on multidisciplinary clinical and translational science. Dr. White is also the Chair-Elect of the Hemostasis and Thrombosis Research Society (HTRS) Fellows Network and serves as a board member for the International Health Care Foundation. As an NHF-Shire clinical fellow, Dr. White will receive specialized clinical training in the Comprehensive Bleeding Disorders Clinic and Women and Girls' Bleeding Clinic at CHOA under the mentorship of Drs. Robert Sidonio and Shannon Meeks. In addition to his clinical focus treating patients with disorders of hemostasis and thrombosis, Dr. White will pursue his clinical research characterizing the management and outcomes of heavy menstrual bleeding in adolescents with bleeding disorders and in those who are taking anticoagulant or antiplatelet medications.
Hanny Al-Samkari

Hanny Al-Samkari

Year:
-
Grants:
NHF-Takeda Clinical Fellowship
Author(s):
Hanny Al-Samkari
While an NHF-Shire Clinical Fellow, Dr. Hanny Al-Samkari will acquire a clinical and research expertise in hemophilia and rare bleeding disorders under the mentorship of Dr. Stacy Croteau at Boston Children's Hospital and Drs. Eric Grabowski and Larissa Bornikova at Massachusetts General Hospital (MGH). Dr. Al-Samkari received his medical degree from Washington University in St. Louis, where he was elected to AOA (Alpha Omega Alpha is a national medical honor society). He completed his residency in internal medicine at the University of Pennsylvania, where he served as Chief Medical Resident. He completed his fellowship in hematology and medical oncology at the Dana-Farber Cancer Institute and Massachusetts General Hospital (MGH) combined program. During this fellowship, he strengthened his passion for classical hematology, and his research was focused on novel indications for thrombopoietin receptor agonists. Dr. Al-Samkari will lead the Hereditary Hemorrhagic Telangiectasia (HHT) clinic at MGH under the mentorship of Dr. David Kuter. He is also a dedicated medical educator, having worked as a member of the American Society of Hematology's Benign Hematology Curriculum Initiative, and he has continued to be active in teaching medical students, residents and physician assistants.
EMS Education

EMS Provider Education Regarding Bleeding Disorders and the Treatment Required in an Emergency

Year:
-
Grants:
Nursing Excellence Fellowship
Author(s):
Lydia Johnson

People with bleeding disorders are often connected to and with various community members such as local and national foundations/associations, their home health company, nurses , pharmaceutical representatives, etc. While people are often encouraged to wear medic alert ID, they do not know anyone in the EMS system who may be caring for them in case of an emergency. Through this project, EMS providers will receive education regarding bleeding disorders and the treatment required in an emergency. This project will seek to connect the HTC's with the EMS systems locally and regionally to coordinate the education with the EMS professionals to provide care.