Anti-Gravity Treadmill Use in an Adult with Severe Hemophilic Arthropathy: A Case Report.

Anti-Gravity Treadmill Use in an Adult with Severe Hemophilic Arthropathy: A Case Report.

Year: 2020
Grants:
Bleeding Disorders Conference
Orthopedic and Physical Therapy
Author(s):
Janet Tankersley, Doctor of Philosophy in Pediatric Science and Doctor of Physical Therapy, Augusta University

Objective:

The objective of this case report was to determine the effects of anti-gravity treadmill use on walking endurance, mobility, and quality of life in an adult with severe hemophilic arthropathy and limited options for exercise.

Methods:

The participant in this case report was a 53-year-old male with a diagnosis of hemophilia A: severe, hypertension, HIV+ (undetectable viral load), obesity (BMI: 36.5) and diabetes mellitus type 2.  Pre-test video analysis of gait was recorded, functional mobility and fall risk was assessed with the Timed Up and Go (TUG), aerobic capacity and endurance was measured by the 6 Minute Walk Test (6MWT), disease impairment severity was measured by the Hemophilia Joint Health Score (HJHS), participation was measured by the Functional Independence Score in Hemophilia (FISH) and the Hemophilia Activities List (HAL) and quality of life was measured by the PROMIS Global Health Score. Intervention included four sessions of walking using the AlterG® anti-gravity treadmill™ with self-selected parameters for speed and duration after body weight percentage was reduced to 40% to achieve pain free walking. Post-test measures of all outcome measures and video gait analysis were recorded.

Summary:

The participant improved in all six outcome measures post-treatment. Distance covered during the 6MWT increased by 120 meters post-treatment (44% increase), the HJHS improved 4 points (6.6%), FISH improved 5 points (17.3%), HAL improved 9 points (5.3%) and the PROMIS Global Health Score improved 14 points (30.4%). The TUG improved from 15 seconds to 13 seconds, placing our participant below the 14 second cut off for high-fall risk. He reported a preference for this intervention over other modes of exercise because upright activity-induced back spasms and ankle pain were eliminated.

Gait and posture analyses revealed improvements in cadence, speed, alignment, and step length while walking in the AlterG® compared to land walking. Duration of exercise increased each session, peak speeds were reached sooner each session, and participant's ability to assume full weight bearing during cool down was achieved sooner each session. Participant reported both short-term and long-term reduction of anxiety and depressive symptoms post-treatment (62% increase in PROMIS sub-category of mental health).

Conclusion:

The results of this case report show the potential benefits of anti-gravity aerobic exercise for adults with severe hemophilic arthropathy. Therapeutic interventions incorporating reduction in body weight while walking, such as, therapeutic pool, body-weight support harness, lowering BMI, and AlterG® could be incorporated in physical therapy intervention plans for patients with severe hemophilic arthropathy to allow aerobic fitness training while minimizing stress on joints. To conclude, anti-gravity treadmill training using the AlterG® can be a safe, effective intervention for improvement of walking endurance, mobility, and quality of life in an adult with severe hemophilic arthropathy.

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

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
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.
Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design

Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design

Year: 2020
Grants:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
Author(s):
Annemieke J. Willemze, MD, Sanofi; Annette von Drygalski, MD, PharmD, University of California San Diego; Barbara A. Konkle, MD, Bloodworks Northwest; Bent Winding, MD, Sobi AB; John Pasi, MB, ChB, PhD, Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry; Marek Demissie, MD, PhD, Sanofi; Roshni Kulkarni, MD, Michigan State University; Stacey Poloskey, MD, Sanofi; Stella Lin, PhD, Sanofi; Suresh Katragadda, PhD, Sanofi; Toshko Lissitchkov, MD, Specialized Hospital for Active Treatment of Hematological Diseases
Characteristics of persons with hemophilia A treated with emicizumab with or without factor VIII inhibitors

Characteristics of persons with hemophilia A treated with emicizumab with or without factor VIII inhibitors

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Ibrahim Abbass, PhD, Genentech, Inc.; Arash Mahajerin, MD, MSc, CHOC Children’s Hospital; Rahul Khairnar, MS, PhD, Genentech, Inc.; Craig S. Meyer, PhD, MPH, MS, Genentech, Inc.; Anisha M. Patel, PhD, Genentech, Inc.; Karina Raimundo, MS, Genentech, Inc.; Erru Yang, MS, Genentech, Inc.
Non-severe hemophilia is not benign? - Insights from the PROBE Study

Non-severe hemophilia is not benign? - Insights from the PROBE Study

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Randall Curtis, MBA, Factor VIII Computing; Chatree Chai-Adisaksopha, MD, Department of Medicine, McMaster University; Department of Internal Medicine, Chiang Mai University; Neil Frick, MS, National Hemophilia Foundation; Michael Nichol, Ph.D., University of Southern California, Sol Price School of Public Policy; Declan Noone, MEng, Health Decisions Consultants; European Haemophilia Consortium; Federico Germini, MD, Department of Medicine, McMaster University; Department of Health Research Methods, Evidence, and Impact, McMaster University; Brian O'Mahony, Irish Haemophilia Society; Trinity College Dublin; David Page, Canadian Hemophilia Society; Jeff Stonebraker, Ph.D., North Carolina State University, Poole College of Management; 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; Mark Skinner, JD, Institute for Policy Advancement Ltd.; Department of Health Research Methods, Evidence, and Impact, McMaster University
Redefining Treatment Satisfaction and Its Impact on Treatment Adherence and Value for Persons with Hemophilia: Findings from the HemACTIVE Study

Redefining Treatment Satisfaction and Its Impact on Treatment Adherence and Value for Persons with Hemophilia: Findings from the HemACTIVE Study

Year: 2020
Grants:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
Author(s):
Adolfo Llinas, MD, Fundación Santa Fe de Bogotá & Universidad de los Andes; Jamie O'Hara, MSc, HCD Economics; Mark Skinner, JD, Institute for Policy Advancement, Ltd.
Fear of pain in people with hemophilia and their families – a pilot study

Fear of pain in people with hemophilia and their families – a pilot study

Year: 2020
Grants:
Bleeding Disorders Conference
Psychosocial Issues
Author(s):
Gaby Golan, PhD, National Hemophilia Center
Pain is one of the most threatening feelings, and people will usually do whatever they can to avoid it. People with hemophilia (PWH) suffer from acute pain from the early days of their life - during blood tests and factor IV infusions. Later, they start to suffer from chronic pain due to target joint damages, inner bleedings, hematuria and so on. The families of PWH are also exposed to the PWH’s pain, and usually are also involved in medical procedures that the PWH undergo and they even cause the pain when they infuse their children the IV factors. The present study aimed to find out the amount of fear of pain in PWH and their relatives. Method: The fear of pain Questioner - FPQ-III: The Fear of Pain - was given to 24 PWH with severe hemophilia, most of them type A, and 39 family members. PWH’s range of age was 10-94 with an average age of 32. Results: Analysis of the questioner revealed that PWH presented significantly less fear of pain compared to their family members. We find the same results even when we compared the PWH’s fear of pain to those of students, hospitalized patients and even of chronic pain patients (according to publication of other studies). Conclusions: When we try to understand the surprising results of the study, which means that PWH fear of pain less than the other populations, we may go in two directions: First, it might be that PWH are getting used to the pain or to the fear of pain, or developed emotional coping mechanisms due to the fact that they are exposed to pain almost from day one of their lives. The second way of understanding this may be that the PWH learned to hide their feelings and they use a denial mechanism, meaning that they are denying their fear of pain feelings.     
The Moti-VIII Study – Factors for Empowering Mobility and Well-being in Hemophilia A

The Moti-VIII Study – Factors for Empowering Mobility and Well-being in Hemophilia A

Year: 2020
Grants:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
Author(s):
Anna Biberger, BSc, Kantar; Christina Ashburner, BSc, Centre for Inherited Blood Disorders; Ceri Hirst, PhD, Bayer; Jessica Charlet, BSc, MSc, PhD, Bayer; Spencer Dunn, MSW, Center for Inherited Blood Disorders; Sharonne Herbert, MD, CHOC
Developing My Bleeding Disorders Community (MyBDC): A community-powered registry to provide a 360-view of living with a bleeding disorder

Developing My Bleeding Disorders Community (MyBDC): A community-powered registry to provide a 360-view of living with a bleeding disorder

Year: 2020
Grants:
N/A
Collaboration/Team Models
Author(s):
Michelle L. Witkop; Maria E. Santaella

Knowing the importance of including the consumers voice in the future of research, the National Hemophilia Foundation (NHF) partnered with the software healthcare analytics company; ArborMetrix, to develop a cloud-based platform; NHF’s Community Voices in Research (CVR), formerly known as MyBDC.

Through CVR, NHF will be able to send out longitudinal surveys and follow participants for a minimum of 5 years.
 

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A US payer database algorithm to identify clinical profiles of hemophilia B for burden of illness assessment

A US payer database algorithm to identify clinical profiles of hemophilia B for burden of illness assessment

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Arielle G. Bensimon, PhD, Analysis Group, Inc.; Eileen K. Sawyer, PhD, uniQure, Inc.; Eric Q. Wu, PhD, Analysis Group, Inc.; Iryna Bocharova, BA, Analysis Group; Nanxin (Nick) Li, PhD, uniQure, Inc.; Tyler W. Buckner, MD, MSc, Hemophilia and Thrombosis Center, University of Colorado School of Medicine
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

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

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