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
Summary of thrombotic or thrombotic microangiopathy events in persons with hemophilia A taking emicizumab

Summary of thrombotic or thrombotic microangiopathy events in persons with hemophilia A taking emicizumab

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Kirsten Balogh, NP, MPH, Genentech, Inc.; Tiffany Chang, MD, MAS, Genentech, Inc.; Fabian Sanabria, MD, F. Hoffmann-La Roche Ltd; Katya Moreno, MD, F. Hoffmann-La Roche Ltd; Richard H. Ko, MD, Genentech, Inc.; Peter Kuebler, PharmD, Genentech, Inc.; Lucy Lee, PhD, Genentech, Inc.; Eunice Tzeng, PhD, Genentech, Inc.
Assessing and Responding to the Oral Health Care Needs of Adults in a Bleeding Disorders Population

Assessing and Responding to the Oral Health Care Needs of Adults in a Bleeding Disorders Population

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Joanna Larson, FNP-C, University of Texas Health Science Center at Houston; Megan Ullman, MPH, University of Texas Health Science Center at Houston; Michael M. Chan, DDS, University of Texas Health Science Center at Houston; Miguel Escobar, MD, University of Texas Health Science Center at Houston
Longitudinal trends of patient-focused programs in the bleeding disorders community from 2013-20: a retrospective analysis of Hemophilia Alliance Foundation grants

Longitudinal trends of patient-focused programs in the bleeding disorders community from 2013-20: a retrospective analysis of Hemophilia Alliance Foundation grants

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models
RESEARCHERS:
Amy Marquez, MS, Fairview Health Services; Anjali Sharathkumar, MBBS, MD, MS, University of Iowa; Audra Ames, PhD, Hemophilia Alliance Foundation; Brenda Riske, MS, MBA, MPA, Hemophilia Alliance Foundation; Crystal Sallans, LCSW, Texas Children’s Hematology Center; Donald Akers, JD, Hemophilia Alliance Foundation; Grant Hiura, MPH, Columbia University Irving Medical Center; Joseph Pugliese, BS, Hemophilia Alliance Foundation; Laurel Pennick, MSSW, LCSW, Arizona Hemophilia and Thrombosis Center; Michael Craciunoiu, EdM, National Hemophilia Foundation; Maria Manahan, MS, Hemophilia Alliance Foundation; Susan Karp, RN, MS, Hemophilia Alliance Foundation; Stephanie Raymond, BS, Cascade Hemophilia Consortium
Unmet Needs in Women with Severe Von Willebrand Disease

Unmet Needs in Women with Severe Von Willebrand Disease

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Women's Research
RESEARCHERS:
Beverly Schaefer, MD, WNY BloodCare, University at Buffalo, Roswell Park Cancer Institute; Jeanette Cesta, BS, VWD Connect Foundation; Shaveta Malik, MD, University at Buffalo, WNY BloodCare
Empowering Nurse Practitioners to Shorten Time to Diagnosis of von Willebrand Disease

Empowering Nurse Practitioners to Shorten Time to Diagnosis of von Willebrand Disease

Year: 2020
Grants:
N/A
Collaboration/Team Models
Author(s):
Michelle L. Witkop, DNP, FNP-BC & Maria E. Santaella, RN-BC, MSN, CPHON

Von Willebrand disease (VWD) is the most common inherited bleeding disorder known in humans, but there are numerous barriers to accurate and timely diagnosis. In January 2021, new evidence-based guidelines were released by the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) (James et al., 2021). The guidelines were designed to support patients, clinicians, and other health care professionals in promptly, accurately, and efficiently diagnosing VWD. Key recommendations in the guidelines include the use of bleeding-assessment tools when unusual bleeding is present and/or VWD is suspected; diagnostic assays; and the role of genetic vs. phenotypic testing for some types of VWD. Nurse practitioners can aid in identifying potential symptoms of VWD to help shorten the time from onset of symptoms to diagnosis.
 

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