bleeding disorders and heamophillia.

1. WHF Poster 2020
Developing My Bleeding Disorders Community (MyBDC): A community-powered
registry to provide a 360-view of living with a bleeding disorder
Log In: https://summit2020-wfh.ipostersessions.com/Default.aspx?s=login
Email: mwitkop@hemophilia.org
Password: MUg9E57c
Poster ID: 317

2. Layout Choices

3. #1 Content Box: Introduction
• Introduction and Objective: 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.
Image: Table: What it is vs. what it is not

4. What is it?
A community-powered registry providing a 360 degree view
of living with a bleeding disorder from those affected and
their unaffected family members
Gathers the consumer’s perspectives
complementing research efforts in the community
Tool to encourage participants to become
active partners in their healthcare
Helps identify the community’s research,
educational and advocacy priorities .

5. What it’s NOT
MyBDC is NOT:
• Research
• HTCs and ATHN drive research
• NHF’s goal is to add the patient’s perspective
• Designed to compete with already established research initiatives in the
community
• Secret
• HTCs and researchers will have access to insights to further their projects
• Rely on HTCs to recruit or enroll participants
• NHF welcomes your support and assistance disseminating information

6. #2-3 Content Box: Timeline for CVR
4/2017 MASAC guidance to hear more of the patient’s voice
10/2017 Strategic plan development for NHFs Research Department
Goal: Improving the Lives of People with Bleeding Disorders by Closing the
Informational Gap through the Generation of Knowledge and Evidence
Objective 1: Advance the Care, Treatment and Support of Advocacy for Persons with
Bleeding Disorders through Transformational Research
Objective 2: Support the Attraction and Retention of a Highly-Engaged Provider
Workforce to Sustain the Interdisciplinary Model of Care
Objective 3: Enhance the Impact of Research through Effective Communication with
Internal and External Audiences
Objective 4: Develop and Operationalize a Patient-Driven Registry to Complement and
Integrate with Existing Databases, thus Increasing the Impact of Research on
Inheritable Bleeding Disorders

7. #2-3 Content Box: Timeline for CVR
1/2018 First visit with Center for Disease Control and Prevention (CDC)
2/2018 Staff Integration with Strategic Plan
4/2018 Call for RFPs and Interviews
6/2018 Charter Stakeholder Meeting
6-10/2018 Focus Group Meetings in conjunction with Inhibitor Summits/BDC
(consumer/provider)
10/2018 Contracted with ArborMetrix to host the platform
1/2019 First meeting with ArborMetrix/NHF staff- weekly meetings there after
1-4/2019 Development of skeleton of MyBDC with NHF/ArborMetrix staff and HTC provider
representative
2/2019 Stakeholder Update via webinar
3/2019 Exempted by Quorum Internal Review Board (IRB) (now Advarra)
4/2019 First enrollment at NHF’s Washington Days – Enrollment continues throughout
4-12/2019 Multiple NHF Chapter in-person events/presentations
4-7/2019 Developed Baseline survey and marketing tools

8. #2-3 Content Box: Timeline for CVR
5/2019 2nd meeting with CDC to review Baseline survey – Multiple informational Chapter
webinars offered
6/2019 First of multiple Virtual Advisory Panels
6-8/2019 Teen track focus groups
8/2019 Baseline survey released for affected individuals
9/2019 Hot Topic Survey on vaping released
10/2019 Personalized Dashboard (PD) released at Anaheim BDC along with embedded
educational resources
11/2019 Incorporated Guias and NYLI to assist with minority and young population recruitment
1-3/2020 Developed Family Baseline Survey, Pain Survey- # of resources and reports available on
PD increased
3-6/2020 Pandemic strikes the USA
4/2020 Family Baseline released, Pain Survey released, COVID Hot Topics developed and
released. Applied for NIH Certificate of Confidence (CofC)
5/2020 Name changed to NHF’s Community Voices in Research (CVR) – CofC approved
6/2020 Enrollment statistics _________________________________________

9. #4 Content Box: Materials and Methods
• Materials and Methods: Exempted by a centralized IRB, surveys are sent to
voluntarily enrolled participants via email and are completed utilizing
smartphones, tablets, or computers. Types of surveys include: enrollment,
baseline, annual, sub-surveys of interest, and hot topics. Participants may
opt-in/out of any survey. Identified through chapter and NHF meetings or
social media, eligible persons include anyone with an inherited bleeding
disorder and their immediate unaffected family members. All results are
reported out in a de-identified, aggregated manner. Each participant will
have a personalized dashboard that allows them to see the results and
benchmark themselves against all participants and have access to
resources and education.
• Images: screen shots of dashboard – link to video tour of dashboard with
audio

10. #5 Content Box: Patient Perspective
Enrollment – Link to
domains covered by
enrollment survey
Baseline Survey
Completed (once) –
Link to domains covered
by baseline survey
Eligible to access PDD
Eligible for VAP
CORE Survey
(yearly)
Sub Surveys Hot Topics
Question of the Day/Month

11. Links: Domains covered by Enrollment Survey
Affected Not Affected
Enrollment  Age
 Birth Sex
 Gender Identify
 Sexual Orientation
 Ethnicity
 Race
 Primary/Secondary Bleeding Disorders
 Severity
 Type
 Relationship to family member with a
bleeding disorder
 Primary/Secondary Language
 Birth Country
 Country you live in now
 Same

12. Link: Domains covered by Baseline Survey
Affected Not Affected - Answered specifically thinking about the
unaffected person
Baseline  Demographics
 Family Associations
 Diagnosis History
 Hx Bleeding Symptoms
 Co-Morbidities
 Inhibitor Status
 Treatment Regimen
 Gene Therapy
 Bleeding History
 Joint
 Life threatening
 Reproductive
 Sexual Activity Limitations
 Provider Information
 HTC vs non-HTC
 NHF and other organizations participation
 Pain
 Intensity
 Experience
 Impact
 Pain Treatment
 Prescription
 Non-prescription
 Medical Marijuana Use
 Mental Health
 Passing of Others
 Physical Activity
 Camp Experience
 Demographics
 Family Associations
 Co-Morbidities
 Reproductive
 Sexual Activity Limitations
 Smoking Status
 Medical Procedures
 Hepatitis Status
 Menstrual Cycle
 Chapter Activity
 Pain
 Intensity
 Experience
 Impact
 Pain Treatment
 Prescription
 Non-prescription
 Medical Marijuana Use
 Mental Health
 Passing of Others
 Social Support
 Physical Activity
 Dental History

13. #5 Content Box: Results
• Results: Enrollment began in late March 2019.
• By June 2020, 593 persons were enrolled (60% affected, 40% unaffected
family members).
• The baseline survey was sent out to both those affected with an inherited
bleeding disorder (8/2019) and their family members (3/2020). As of June,
2020, 37% of affected persons have completed this survey and 19% of family
members.
• 40% have completed a pain survey that was sent out to 115 affected persons
who identified as having chronic pain in their baseline survey.
• 35% completed a COVID survey sent out to everyone enrolled in CVR.
• Use slide show option to present survey data (next 8 slides)

14. 141
423
455
488
505
552
581 583 593
140
190
240
290
340
390
440
490
540
590
593
Total
Enrollment
Launched
3/2019
366
Females
221
Males
Enrollment
6
Other
6/1/2020
Gender Identity

15. Demographics and Self-Identified Race and Ethnicity
35
60%
(n=355
)
Have a bleeding disorder
35
40%
(n=238
)
Is a family member
6/1/2020
19
49
445
41
33
20
6
4
3
0 100 200 300 400 500
Unknown
I prefer not to answer
White/Caucasian
Black/African American
Asian
American Indian/Alaska…
South Asian
Native Hawaiian/Other…
Middle Eastern
Race –select all that apply
142
427
24
Hispanic/Latino
Yes No Prefer not to answer

16. 35
41%
Completio
n Rate
Invitations
Sent 326
Baseline Survey for Affected Individuals
56
35
49
38 38
56 63 65
70
78
80 99
117
122
130
133
0
20
40
60
80
100
120
140
160
180
200
Oct 2019 Nov 2019 Dec 2019 Jan 2020 Feb 2020 Mar 2020 Apr 2020 May 2020
133
Total
Affected
Baseline
Surveys
Completed
Since
10/2019
65
in progress
198 total
61%
6/1/2020

17. 75
30
12
4
3
3
2
2
0 20 40 60 80
Hemophilia A (57%)
Von Willebrand Disease (23%)
Hemophilia B (9%)
Other (3%)
Platelet Disorder (2%)
Factor VII deficiency (2%)
Factor XIII deficiency (2%)
Factor X deficiency (2%)
Primary Bleeding Diagnosis
(N = 131)
4
3
2
2
1
1
1
0 5 10 15 20
Platelet Disorder (29%)
Von Willebrand Disease (21%)
Other (14%)
Hemophilia B (14%)
Factor V Deficiency (7%)
Factor I deficiency (7%)
Ehler-Danlos Syndrome (7%)
Secondary Bleeding Diagnosis
(N = 14)
Self-Reported Bleeding Diagnoses
(N)
6/1/2020

18. 11%
11%
11%
12%
12%
13%
13%
13%
13%
19%
21%
22%
22%
23%
23%
27%
29%
40%
0% 5% 10% 15% 20% 25% 30% 35% 40% 45%
ADD-ADHD
IBS
Kidney Stones
Ulcers
Thyroid Disease
Hypercholesterolemia
Eczema-Psoriasis
Blood in Urine
Head Injury
Asthma
Hepatitis C
Osteoarthritis
Anxiety
Hypertension
Migraine
Hemarthropathy
Depression
Chronic Pain
Most Frequently Observed (n=198)
Most Frequently Reported Co-Morbidities
6/1/2020
26%
46%
67%
0% 20% 40% 60% 80%
PMS
Dymennorrhagia
Hypermenorrhagia
Women (N=90)
Women (N=90)

19. Care as Identified by the Participant
N = 141
Includes partial responses
6/1/2020
HTC vs. no-HTC
Yes (N=86) N (N=48) Other (N=7)

20. What we’ve learned so far…
44% of women experience pain
most days or everyday
That number jumps to 66%
when we look at men
44%
66%
0% 20% 40% 60%
Men
Women
50%
40%
Overall 47% of participants reports having
difficulty maintaining employment
because of their bleeding disorder
18% of participants are using
medical marijuana for symptom
management
65%
43%
96%
0% 20% 40% 60% 80% 100%
Anxiety
Depression
Pain
Yes No
40% of all women respondents
vs 50% of all men respondents
N = 131
6/1/2020

21. Women with bleeding disorders
(N=64) reported a history of:
81%
48%
73%
0% 20% 40% 60% 80%100%
Pregnancy
Miscarriage
Bleeding before or
after childbirth
Yes No
49%
44%
7%
Yes No I don't know
Family history of a bleeding disorder
at time of diagnosis
N = 131
0 20 40 60 80
Get help when sick in bed
Get love and attention
Have people who care
what happens to me
Get invited to do things
with others
Social Support (N)
Much less than I would like
Less than I would like
Some, but would like more
Almost as much as I would like
As much as I would like
What we’ve learned so far…
6/1/2020

22. #6 Content Box: Conclusions
• Conclusions: There is great initial interest in CVR, a community-
powered registry, designed to promote the community’s voice in
research. The de-identified, aggregated results will be available to
researchers promoting a better understanding of what it is like to live
with an inherited bleeding disorder. The personalized dashboard was
launched in November 2019.
• Image: participants’ quotes on next slide

23. As both a person with a bleeding disorder and as a researcher,
I am extremely curious and excited by what is possible with MyBDC!
It is immediately clear to me the value it will bring to not only those who
study, but to those who are studied (a rare dual feat!).
A huge opportunity exists to push our understanding and to leverage those
insights into substantive advancements for the bleeding disorder community.
Participant’s Quotes