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
The relationship between self-reported physical activity, treatment regimen, mental health and pain in persons with hemophilia enrolled in Community Voices in Research

The relationship between self-reported physical activity, treatment regimen, mental health and pain in persons with hemophilia enrolled in Community Voices in Research

Year: 2020
Grants:
N/A
Collaboration/Team Models
Author(s):
Michelle L. Witkop DNP; Maria Santaella, MSN, BC,; Cindy Nichols, PhD

The objective of this study is to assess the relationship between self-reported physical activity, treatment regimen, mental health, and pain in persons with hemophilia (PWH) enrolled in CVR.
 

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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.
Treatments and Clinical Outcomes of Bleeding Related to Pregnancy, Surgery, or Spontaneous or Traumatic Bleeds in Women and Girls With Factor VIII and IX Deficiency: Results From a Retrospective Chart Review

Treatments and Clinical Outcomes of Bleeding Related to Pregnancy, Surgery, or Spontaneous or Traumatic Bleeds in Women and Girls With Factor VIII and IX Deficiency: Results From a Retrospective Chart Review

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Women's Research
RESEARCHERS:
Ateefa Chaudhury, MD, Center For Inherited Blood Disorders; Elisa Tsao, PhD, Sanofi; Justyna Tymoszczuk, MS, Sanofi; Mariana Oviedo Ovando, PhD, ICON plc; Nisha Jain, MD, Sanofi; Robert Sidonio, MD, Emory University School of Medicine and Children’s Healthcare of Atlanta; Roshni Kulkarni, MD, Michigan State University
Final Results of PUPs A-LONG Study: Evaluating Safety and Efficacy of rFVIIIFc in Previously Untreated Patients With Hemophilia A

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

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Amy Dunn, MD, Nationwide Children's Hospital; Bent Winding, MD, Swedish Orphan Biovitrum AB; Christoph Königs, MD, University Hospital Frankfurt; Deepthi Jayawardene, MS, Sanofi; Beatrice Nolan, MD, Our Lady's Children's Hospital; Manuel Carcao, MD, The Hospital for Sick Children; Margareth C. Ozelo, MD, PhD, University of Campinas; Michele Schiavulli, MD, A.O.R.N. Santobono-Pausilipon; Raina Liesner, MD, Great Ormond Street Hospital; Roshni Kulkarni, MD, Michigan State University; Simon A. Brown, MBBS, MD, Queensland Children’s Hospital; Sriya Gunawardena, MD, Sanofi; Sutirtha Mukhopadhyay, MBBS, Sanofi
Hanny Al-Samkari

Hanny Al-Samkari

Year:
-
Grants:
NBDF-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.
Michael H. White

Michael H. White

Year:
-
Grants:
NBDF-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.
EMS Education

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

Year:
-
Grants:
Nursing Research and Practice Grant
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.

Identification, Characterization and Therapeutic Targeting of Key Molecular Markers and Pathways Implicated in the Development of Hemophilic Arthropathy

Identification, Characterization and Therapeutic Targeting of Key Molecular Markers and Pathways Implicated in the Development of Hemophilic Arthropathy

Year:
-
Grants:
Nicholas Cirelli Family
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilic Arthropathy
Pain
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Esther Cooke

Dr. Esther Cooke received her Ph.D. from the Leeds Institute of Cardiovascular and Metabolic Medicine at the University of Leeds, U.K., where she studied the role of fibrinogen phosphorylation in thrombosis. Dr. Cooke is currently a postdoctoral fellow in the laboratory of Dr. Annette von Drygalski, at the University of California San Diego, and in collaboration with the laboratory of Dr. Laurent Mosnier at the Scripps Research Institute. Dr. Cook's JGP Fellowship project will focus on pathological mechanisms associated with joint bleeding, re-bleeding, and the development of hemophilic arthropathy. Dr. Cooke will perform comprehensive gene expression analyses to explore key molecular markers and pathways that drive soft tissue inflammation and vascular changes in joints after bleeding. In this way, she hopes to identify new therapeutic targets and develop novel treatment strategies to down-regulate these processes, thereby reducing re-bleeding tendency and slowing the progression of hemophilic arthropathy.