Transcript CAF 2015 Patient and Parent Study

2015 Patient and Caregiver Study
Full Report
May 2015
Prepared by:



Objectives and methodology
Summary of findings
Detailed findings
Information Sources
Treatment Centers
Adherence
Quality of Life
Clinical Trials
About CAF
Profile of Group Participants106
3
5
12
22
25
36
51
72
2

To determine barriers and facilitators of compliance with treatment for
thalassemia; what has or would be helpful in supporting patient
adherence to treatment as outlined by his or her physician

To identify awareness and interest in clinical trials; reasons why
patients have/ would participate or have not/ would not participate if
given the opportunity

To examine patient views of his or her quality of life, what impacts
this, and conditions under which this could be improved

To obtain perceptions of CAF; likes, dislikes, and what is or would be
most valuable from CAF
3
14
102
participants
groups

(67 patients, 35 parents)
All participants stated that they:
 Are a person with thalassemia, or are a
caregiver for a person with thalassemia
 Would be able to complete the 90
minute focus group and the 10 to 15
minute online survey
…most of these and
others not in a group
completed an online
survey…
68
patients
105
respondents
in total in the
online survey
37
parents
4

Issues or barriers to compliance with prescribed treatment of
thalassemia included logistics/ work, school or family
obligations, pain or inability to function (as required by
employer or educational institution), dislike taste of
medication, issues with insurance/ cost, or lack of time due to
other priorities.

What has or would be helpful in being more compliant or
adherent to prescribed treatment included support from family/
friends/ patients/ doctors/ CAF, oral chelators, and greater
level of knowledge/ better education on available options and
resources.
5

As the study included patients in their 40’s and 50’s, some
had participated in clinical trials going back to the 1970’s on
Desferal; others had participated in clinical trials on L1,
Exjade and/or bone marrow over the past 10 or 20 years.
Many had heard of clinical trials for gene therapy and a
minority had heard of treatment of thalassemia with the
protein Luspateracept.

Decisions to participate or interest in participating in any
clinical trial was driven most by an interest in benefitting all
thalassemia patients and/or promising signs of the treatment,
possibility of a cure, easy or acceptable logistics, minimal
risk, and to identify a preferable treatment over existing
options.
6

Decisions to not participate or lack of interest in participating in
any clinical trial was driven most by the assumed risk, absence of
data/ information on risks and outcomes from a given treatment or
a risk-benefit ratio that was or is unacceptable.

Most patients’ and parents’ (rating for their child) quality of life
rating was a “4” or higher on a 5 point scale where 1 is not good
and 5 is very good. In terms of what has a positive impact on their
quality of life, respondents mentioned family/ friends, oral
medications/ transfusions (and staying compliant), career, exercise/
diet, and normal activities which include any number of things
(cooking, travel, hobbies, etc.). What was cited as having a
negative impact included pain, logistics with treatment, missing
normal activities, fatigue/ lack of energy, etc.
7

Most respondents indicated a significant level of familiarity
with and some degree of involvement in CAF (events,
donations, helping other patients, etc.). Over 1/3 or patients
and nearly half of parents also noted that their level of
engagement with CAF has increased from what it was five
years ago.

Impressions of CAF were very positive, with the majority of
patients and parents rating CAF as “Excellent”. Many
commented on how CAF has been supportive (in numerous
ways), cares about patients “like family members”, and goes
above and beyond when it comes to serving as an advocate for
thalassemia patients. The few dislikes that have surfaced have
been regarding holding events in other parts of the United
States.
8

What patients and parents found most valuable from CAF
included support/ encouragement/ sense of community,
information/ expertise/ being kept up to date, and advocacy.
Lack of CAF presence/ conferences in regions outside the
northeast U.S. was cited as an area valued least by some
respondents.

What would be even more valuable according to some group
participants included more networking opportunities/ a forum
for patients/ parents (online, in-person), CAF presence/
conferences in other parts of U.S., training/ education/
network for hematologists/ medical community, and (more)
information/ support on clinical trials.
9
Number of respondents
Parents
68
37
Percent reporting transfusions every 2 to
4 weeks
93%
92%
Adherence rating (5 = fully adherent)
Are or have participated in a clinical trial
4.4
63%
4.8
19%
Have opted not to participate
32%
5%
Rating of interest in gene therapy (5 =
definitely would participate)
4.0
4.6
Rating of typical pain (1 = no pain)
2.5
1.7
Rating of quality of life (5 = very good)
4.2
4.5
Reporting that quality of life has
improved in last five years
46%
57%
Yes, more support could improve quality
of life
54%
43%
Have contacted CAF
79%
95%
Overall impression of CAF (5 = excellent)
4.6
4.8
Web Survey
Patients
10
Focus Groups and Web
Survey
Patient Groups
CAF, their hematologist, websites (including pharma company
site), and Facebook groups were popular sources of information
for patients and parents.
Where do you look for reliable information about
thalassemia?
# Mentions
CAF (website and newsletter)
26
Doctors/Hematologists/Clinics
24
Online websites (including the drug
manufacturers website)
19
Facebook groups
14
Other patients
Conferences
Mobile apps (like ThalPal)
6
6
4
(67 patients, 35 parents)
Parent Groups

Number of mentions cited above
13
Patient Groups
Parent Groups
hematologist
endocrinologist
On Facebook, we have a support group for parents
who've adopted kids with Thalassemia…Sometimes
it's that I have a specific situation that's come up
with my daughter and I can ask how other people's,
if they've ever encountered the same experience or
they understand how it should be handled. And
sometimes it's just seeing what other people are
experiencing. Even if it's not what I'm experiencing
at the time. --Parent
14
Patient Groups
Monthly
Quarterly
Yearly
Parent Groups
Many study participants
didn’t have set type(s) of
information that they sought
regularly. Rather, patient
and parent questions
evolved with learning and
current circumstances.
Those who did cite specific
types of inquiries or
searches mentioned clinical
trials, gene therapy, side
How often?
effects, older patients, and
As needed
standards of care.
Daily
# Mentions
11
9
7
4
4
15
New drug developments
Clinical trials
How other patients are handling
treatments
Chelation therapy
What’s working for other thal patients
About possible cures
Recommended regimen of tests and
check-ups
Patient Groups
Long-term effects of thalassemia and
the treatments
Gene therapy
Bone marrow transplants
Fertility and pregnancy
About tests
Drug interactions
The effects that Exjade can have on
the liver
Cause of death of thalassemia
Stem cell research
patients
Symptoms
Diet and supplements
More information than what is
The biology of thalassemia
currently available
Insurance information
Anything CAF provides is good
Muscle deterioration
Adoption
The standard formula for transfusion amount based upon hemoglobin counts
of children
Parent Groups
The "Guide to Living with Thalassemia" which
is the comprehensive workbook…They've
streamlined it. It's an excellent resource that
I've given to family and friends and my child's
school nurse and things like that. It's a great
reference. --Parent
16
Patient Groups
Patients
Parents
How do you prefer to discover or receive this
information?
30
19
18
17
9
10
12
1
Conference
9
6
Regular Mail
5
2
Thal Centers
5
0
Other Patients
3
0
0
3
In Person
Mobile app
Online/ Website/ Social Media
Hematologist/ Doctor/ Nurse
Email
(67 patients, 35 parents)
Number of mentions cited above
Parent Groups
Most patients and parents prefer to access information about
thalassemia online, via email or through their doctor.
Like Facebook push
notifications, it would
be so nice to have
information pushed
out. --Parent
17
What is lacking in the information available
about thalassemia?
Clinical trial information/ updates
Hard copies of information about Thalassemia (studies,
patient care, newsletters, etc)
Studies on long term effects/complications of the disease
(older patients)
Information for female patients on puberty,
pregnancy/fertility
Information on Insurance Issues for patients (causes them
to have to choose between therapies)
Info on chelation therapies
Doctors familiar with the disease
# Mentions
Patient Groups
Parent Groups
A range of items surfaced when respondents were asked
what was lacking in information available about
thalassemia...
8
7
5
5
5
5
5
(67 patients, 35 parents)
Number of mentions cited above
18
Patient Groups
 “I'll share a big one. One of the ones is in the standards of care or any
publication is the standard formula for transfusion amount based upon
hemoglobin counts of children. It's like missing in all the literature so when
you're trying to get your child transfused with an adequate amount of blood
you're, there's no material that says this is the amount to use. I think that's the
biggest one I find.” – Parent
 “(One of the doctors) had a theory about some kind of mitochondrial study that
would help thalassemics build up their hemoglobin and then 2 years later,
never heard anything about it. So I asked (another doctor), I said, "what's
happening with that?" She's like, it just died in the water, nobody picked up the
study to fund it. So I think what people need to know every year is like okay,
what happened with that study or how far are we getting with gene therapy.” Patient
Parent Groups
Some patient and parent comments on what is lacking included...
19
Patient Groups
Parent Groups
 “What’s interesting is information on patients that are probably our age like
patients who are 30 and older, patients who are now starting families who
are kind of think of their kids of their own. That information is very difficult
to find and if you’re lucky to have another person on your transfusion group
that has gone through those days but otherwise it’s not really good
information about those kind of things.” - Patient
 “Because they're not moderated (some online groups/ communities on FB)
there's some honestly crappy information that's thrown out there on all of
them. There've been several times that I've gone to my hematologist saying, I
feel like I need to be the moderator of these groups because people are
saying things that are totally not true. It's a really, really tricky thing these
online communities. They're an amazing source of support, but they're alsosomeone said yesterday that patients with thal intermedia can't become iron
overloaded. Completely and utterly false.” - Patient
20
Patient Groups
Parent Groups
 “The research that I've been looking at- I have a daughter - and I'm just looking
at if she'll have to transfuse more frequently when she starts hitting puberty,
because of her cycle. I don't really see much of a difference of that, or if
anybody explaining how that works. Or even when she decides to get pregnant
when she's older. I know people who have been thought that, and they say that
they just stop chelating, but as far as the transfusions, I think they stop that as
well from what I hear.” - Parent
 “How to steer your doctors in the correct direction. Your local doctors are very
often not giving you the highest quality of care available.” – Patient
 “...(would prefer) having a little bit more access to some of the more research
oriented, the raw data, the pure science stuff, like PubMed to log in and read
the research reports or to be able to get the same access that researchers have. I
think patients should be able to access the same information their doctors are
accessing about the research studies and the trials that are going on if they're
interested.” – Patient
21
Parents
Which major treatment centers come to mind
for persons with thalassemia? (unaided)
29
22
Oakland Childrens
26
14
Philadelphia Childrens/ CHOP
22
5
16
17
16
4
Cornell
14
3
LA Childrens
7
6
Chicago Childrens
4
3
3
15
Texas Childrens
Atlanta Childrens (Children's Healthcare of
Atlanta/ CHOA)
New York Presbyterian Childrens
Boston Childrens
Web Survey
Patients
(68 patients, 37 parents)
Number of mentions cited above
23
Patients
(p/c)
Parents
(p/c)
Which major treatment centers have you used? (p)
Which are you using now? (c, # bold)
14/3
12/7
6/7
5/5
11/6
-
6/2
4/3
Boston Childrens
7/8
2/1
Cornell
6/2
-
LA Childrens
2/1
-
Chicago Childrens
1/0
-
3/1
5/4
Texas Childrens
Atlanta Childrens (Children's Healthcare of Atlanta/
CHOA)
Oakland Childrens
Philadelphia Childrens/ CHOP
Web Survey
New York Presbyterian Childrens
(68 patients, 37 parents)
Number of mentions cited above
24
To determine barriers and facilitators of compliance with treatment
for thalassemia; what has or would be helpful in supporting patient
adherence to treatment as outlined by his or her physician.
Most respondents indicate that they are fully adherent to treatment as prescribed
by his or her physician. Not surprisingly, parents as the primary caregivers to
their children (in some cases very young) indicate higher levels of compliance.
Average adherence rating
now is
2%
4.4
7%
2%
3% 3%
"4"
31%
4.8
"5" Fully adherent
14%
"3"
59%
Patients (68)
Web Survey
Average adherence rating
now is
"2"
"1" Not at all adherent
81%
Parents (37)
26
When asked about their level of adherence five years ago, and as a child,
respondents lower their rating. Of note are parents indicating “not at all
adherent” for their child. In these cases, the child was not getting treatment
prior to adoption.
Average adherence rating then is
9%
19%
"5" Fully adherent
48%
16%
3.7
4.0
6%
"4"
14%
"3"
21%
"2"
"1" Not at all adherent
Patients (68)
60%
Web Survey
Average adherence rating
then is
3%
5%
Parents (37)
27
Those who indicated that they (or their child – in some cases 18+ years old)
were not fully adherent in treatment of thalassemia were asked where they are
not adherent/ compliant. Most indicated following exact chelation therapy as
prescribed.
 “Chelation therapy was always a challenge especially through school and
college days. Managing chelation regimen (today) to the fullest is a challenge.
Especially with combination therapy of Exjade and Ferriprox.” - Patient
 “He takes the Exjade every day but not he is not compliant with the directions
for Desferal.” - Parent
 “My 18 year old has been having a problem with compliance for a few years
now. He does not always take his medication and when he was on Exjade he
was not always finishing it.” – Parent
 “I skip a day or two of chelation.” - Patient
Web Survey
 Chelation medication as prescribed (18)
28
 Appointments/ exams/ tests (6)
 Diet/ supplements (3)
 “I don't adhere to a low iron diet.” - Patient
 “I’m supposed to be on calcium supplementation but choose to get it in my diet
instead.” – Patient
 “He doesn’t like taking the recommended micronutrients/ vitamins.” - Parent
Web Survey
 “Certain yearly exams or tests are not always done. Such as audiology or bone
density.” - Patient
 “Sometimes I do not get all the testing necessary such as Liver Ultrasound
annually.” – Patient
 “Maybe once or twice a month. I miss appointments.” – Patient
29
Some of the more frequently mentioned reasons for not being fully adherent
included:
 “Time issues. I'm a college student, and often I won't have time to go to the
doctor appointments and I'll forget to take my Exjade because something else is
making me preoccupied (like an upcoming exam).” - Patient
 “I already go to many doctor's appointments. Having me take more time off for
some of these other tests seem like a nuisance. It would be great if a treatment
center or location would do all the tests we need in one spot.” – Patient
 “Work or being too busy.” – Patient
 “I suspect he might have skipped medication when he
studied late and fell asleep.’ - Parent
Web Survey
 Logistics/ work/ not enough time (9)
30
 “I get lumps and welts on my stomach from the desferal and it's painful. “Patient
 “For L1, the side effects were really harsh - stomach pain/vomiting, drowsiness,
made it really difficult to function at a high level as might be required of a law
student who is taking classes and working”. - Patient
 “The injection is painful. Injection sites have become sore, scarred, and
indurated.” – Patient
 Don’t like the taste (4)
 “He hates the taste and texture of ex-jade. Also doesn't like having to do
something every day.” – Parent
 “For the exjade it was the taste. It was hard for him to take no matter what he
mixed it with.” – Parent
 “I get lazy, I don't like taste - its chalky, also waste of time mixing it.” – Patient
Web Survey
 Painful/ side effects (4)
31
 Forget to take or bring meds with me (4)
 “A change in my routine will make it difficult to remember a set of pills.” –
Patient
 “I either forget or am trying to stretch the supply as it is expensive and I cannot
afford it.” – Patient
 “Lack of insurance, loss of job.” – Patient
Web Survey
 Cost/ lack of insurance (3)
32
Parents cited lack of proper treatment for those adopted from China. Some
comments included:
 “My child was still living in China. She was receiving transfusions every 6
months and was not getting any chelation. She was fully compliant with the
treatment she was receiving, but it was in no way in line with the standards of
care.” – Parent
 “He was living in China at the time and they do not properly treat children with
thalassemia. I don't know exactly what their treatment standards were, but I do
know they weren't appropriate for his disorder.” – Parent
 “She was adopted so in her home country of China and in her orphanage, she
got transfusions but not regularly and no chelation treatment was given. I don't
even know what labs if any were done to monitor her.” – Parent
Web Survey
 Improper, little or no treatment in China (8)
33
Patient Groups
Patients
Parents
47
9
Family/Peer/Patient support system
5
4
3
0
0
0
Desire to live/be healthy
Positive attitude
Deaths of other patients
Understanding importance through information
about disease/ Understanding the ‘why’/info about
disease/treatments
ThalPal peers
Advice from other patients’ parents
Changing how meds are given
CAF
15
7
3
7
3
0
0
0
0
8
2
2
Doctor/medical staff support system
Parent Groups
What has been the most valuable support
mechanism in terms of enabling better adherence
for your treatment for thalassemia?
(67 patients, 35 parents)
Number of mentions cited above
34
In both the online survey and focus groups, patients and parents cite support
from family, friends, peers as beneficial in helping them follow treatment as
prescribed by their doctor.
Parents
12
10
0
2
0
4
6
3
2
1
1
5
0
0
0
0
Oral chelation
Support (family/friends)
Knowledge/better understanding
Accessibility to treatment (hours, location)
Less side effects
One day off
Better insurance
Getting to the United States (adopted from foreign
countries)
Web Survey
Patients
Is there something that was beneficial in the past
in terms of improving your adherence? Or
something that would have been helpful?
(68 patients, 37 parents)
Number of mentions cited above
35
To examine patient views of his or her quality of life, what
impacts this, and conditions under which this could be
improved.
Not
good
Very
good
4.5
4.1
(patients –
focus
groups)
4.2
Of male patients, the average quality of life rating
was 3.9. This compares to an average quality of
life rating of 4.3 given by female patients
(parents focus
groups)
4.5
Patient Groups
Parent Groups
5
Web Survey
1
(parents web survey)
(patients web survey)
37
Nearly half or over half of patients and parents indicate that their (or their
child’s) quality of life is better today than it was five years ago. Roughly one
third indicate that their has been no change and one quarter of patents noted
that it’s worse today.
60%
Web Survey
50%
40%
30%
20%
10%
0%
Much worse
today
Somewhat
worse today
No change
Patients (68)
Somewhat
better today
Much better
today
Parents (37)
38
Better today:

 New/ better treatment options
(11)
 More adherent today (5)
 Better educated/ informed/
make better choices (3)

 Improper, little or no treatment
in China (prior to adoption)
(11)
 New/ better treatment options
(6)
 Health has improved (3)
Worse today:
 Complications, pain and/or
fatigue increasing with age (8)
 More pain today (4)
Patients (68)
Better today:

Worse today:
 Treatment centers less flexible
today (hours, etc.)/ logistics of
treatment (3)
Web Survey

Parents (37)
39
 Support network (peers) (15)
 “Sometimes I feel kind of depressed or annoyed with the fact that I have
thalassemia. I wish there was some kind of support group or camp or something
of the sort for kids with thalassemia so we can discuss our feelings with people
who know what we are going through, and are going through similar treatment. I
often find thalassemia linked into different cancer groups and camps, I went to as
a kid. But it's not the same, because we don't have cancer, we have thalassemia.
And I wish there was an outlet for us, like if CAF put on a program for kids or
young adults with thalassemia, that would make a huge difference.” - Patient
 “A vehicle for them to talk to other patients who have walked in their shoes.” –
Parent
Web Survey
When asked what type(s) of support would improve their (or their child’s)
quality of life, respondents most frequently mentioned a support network
(peers), better insurance/ financial support, further advances in treatment of
thalassemia, and easier logistics for treatment (hours, appointments scheduled
together, etc.).
40
“Social support of family and friends always
helps. Also patients sharing experiences and
advice with each other can be helpful and
even lifesaving in some cases.” - Patient
“Our thal community is very small in Orlando. It
would be great to have a closer thal center but we
end up going to Atlanta for our yearly check-ups
and such. The short time there doesn't give us the
opportunity to meet others with Thal.” – Parent
I think a support group would be helpful.
Like an online forum--through CAF,
potentially--that encourages thalassemia
patients to talk, complain, vent, whatever.
Often, my major issue isn't the medical
stuff, it's the fact that I don't know anyone
else who's in the same situation as me: 21,
and spending a great deal of time in the
hospital. It can get relatively lonely, and I
think some sort of forum would be of
exceptional help to thalassemia patients'
psychological health. –Patient
41
 “Financial support for co payments etc. There is help for people who do not help
themselves and don't work. No help for people who work hard and use all of
their money for medical expenses. A personal assistant to help with tasks.” Patient
 “As a low income large family with a chronically ill child, our financial
resources are tapped. Any extra money we have is spent on copays and travel to
get back and forth to the hospital. We would love to take my daughter to do fun
things but financially cannot. Free movie passes or free passes to other evens
close to our home would be such a blessing.” - Parent
 Further advances in treatments (10)
 “Better treatment in pain management and general advancement of medicine
and knowledge related to thalassemia that can have a positive impact.” –
Patient
 “Pill form of chelation medicine that has to only be taken once/day and doesn't
require weekly testing to insure there are no negative side effects.” - Parent
Web Survey
 Better insurance/ financial support (12)
42
 “If the care centers could help schedule appointments that coincide with each
other, to make it helpful to visit the hospitals less. For example, I need my
Coumadin checked every month, sometimes more often. but why can't they just
check it when I go in for my routine blood work for my transfusion. No they
make me go to a separate clinic just for my INR. It’s more work for me to go back
and forth.” - Patient
 “...How can patients keep working if they have to go to an adult hematologist
who doesn't have office hours that can accommodate their schedule? ” – Patient
 “If we had an option for weekend or evening transfusions it would be a great
help.” - Parent
 “Flexible doctor visit and transfusion schedules.” - Parent
Web Survey
 Easier logistics for patients to get treatment (7)
43
 “Having adult hematologists know more about Thalassemia so they can be
strong alternatives for adult patients. Most Thal patients see pediatric
hematologists because they were the ones who treated them as children. But now,
many of us are way into our adult lives and sometimes the patient/doctor
relationship can be hindered when pediatric doctors are caring for middle aged
adults. Patients can feel misunderstood and maybe uncomfortable being cared
for in a pediatric center. However, the best doctors for Thalassemia that I have
had were all pediatric hematologists. It's difficult to make the transition to an
adult hematologist because most adult hematologists don't know how to care for
a patient with Thalassemia.” - Patient
Web Survey
 Greater knowledge/ expertise in thal treatments and best
practices on the part of hematologist/ medical community
(5)
44
 “It would be incredibly helpful to have her regular local hematologist
knowledgeable of thal and current best practices of care for her well being. We
experienced several situations that caused her to suffer more than was necessary
until our thal clinic helped us by communicating more directly with our local
hematologist. Then our local hematologist got on board with what she needs.” –
Parent
 “The local blood bank doesn't seem to understand the need for fresh blood. If
they could be better educated it would help.” – Parent
Web Survey
 Greater knowledge/ expertise (continued...)
45
I think it would be very impactful for
our kids over the long haul to get their
transfusions, MRIs, a lot of their
appointments and things in the
evenings and on weekends so they can
have more of a normal life and not
miss so many activities and things by
spending so much time in a hospital
setting. --Parent
46
Patients indicated higher levels of pain compared to parents rating the level
of pain experienced on a regular basis by their child(ren) with thalassemia.
5%
4%
19%
22%
"2"
11%
"3"
25%
29%
"4"
49%
35%
Web Survey
No pain at all - "1"
Very painful - "5"
Patients (68)
Parents (37)
47
The majority of patients and parents talk with someone about the pain they
experience, or pain experienced by their child. In most cases, this is a doctor,
nurse and/or family member.
Web Survey
60%
50%
40%
30%
20%
10%
0%
No One
Nurse
Patients (68)
Doctor
Family Member Other (spouse,
friends, patients/
parents)
Parents (37)
48
The most frequently mentioned reaction of doctors/ nurses/ family
members when discussing pain with a thalassemia patient or parent is
empathetic/ supportive.
Parents
20
10
9
4
4
5
2
3
0
1
0
0
1
0
1
3
What kind of reaction did they have? (when
talking with them about pain)
Empathetic/ Supportive/ Do Things to Help
No Reaction/ Not Concerned
“Get over it”/ Thought I was Complaining
“Take Tylenol/ Aleve/ Ibuprofen”
Don't understand but try to help
Normal/ Appropriate (asked proper queues)
Sent for testing
Said it was normal/ expected
Web Survey
Patients
(68 patients, 37 parents)
Number of mentions cited above
49
Those who experience pain on a regular basis were asked how they
manage it. The most popular responses were taking Tylenol/ Aleve/
Ibuprofen, getting rest, or just dealing with it/ ignoring it.
Parents
26
7
10
11
8
3
3
2
2
2
0
1
1
0
0
0
0
0
0
2
How do you manage pain?
Take Tylenol/ Aleve/ Ibuprofen
Rest
Deal with it/ Ignore It
Yoga/ Exercise
Ice/ heat
Physical Therapy
Deep Breathing
Injections (cortizone)
Chiropractor
Antiseptic cream (before draws/transfusions)
Web Survey
Patients
(68 patients, 37 parents)
Number of mentions cited above
50
To identify awareness and interest in clinical
trials, reasons why patients have participated or
would participate or have not/ would not if
given the opportunity.
Patient Groups
Patients
Parents
Clinical Trial
33
16
Gene therapy
15
13
12
5
3
3
3
0
0
0
0
1
0
0
0
0
6
1
Stem cell
Chelation
Exjade
Ferriprox
Jadenu
Desferal
Luspatercept
Bluebird
BMT
Most discovered the trial(s)
via their doctor and/or CAF
Parent Groups
When asked what clinical trials related to thalassemia they are aware of,
roughly half of patients and parents cited gene therapy.
Patients Parents Found out from…
28
12
5
9
10
3
3
0
0
0
0
3
(67 patients, 35
parents)
Doctors/ hospitals
CAF sites/ mailings
CAF conferences
Hematologists
CHOP
Facebook parent
groups
(67 patients, 35
parents)
Number of mentions cited above
52
Patient Groups
Parent Groups
Using a scale of 1 to 5, where 1 is definitely would not participate and 5 is
definitely would participate, respondents were asked to rate their intention/
desire to enter (or have their child enter) a clinical trial. Most were mixed to
positive.
50%
40%
30%
20%
10%
0%
"1" Definitely
would not
"2"
Patients (67)
"3"
"4"
Parents (35)
"5" Definitely
would
53
Patient Groups
Roughly two thirds of patients and parents indicated that they were
aware of the ongoing clinical trials in gene therapy for thalassemia.
(versus lower %s unaided)
32%
Parent Groups

34%
66%
68%
Yes
No
Patients (67)
Yes
No
Parents (35)
54
60%
Patient Groups
Of male patients, the
average interest rating was
4.7. This compares to an
average interest rating of
3.7 given by female
patients. Those under 40
gave an average rating of
4.3 while 40+ patients
average rating came to 3.7.
Parent Groups
Using a scale of 1 to 5, where 1 is definitely
would not participate and 5 is definitely
would participate, respondents were asked to
rate their intention/ desire to enter (or have
their child enter) a gene therapy trial. Interest
ratings were mixed.
50%
40%
30%
20%
10%
0%
"1" Definitely
would not
"2"
Patients (67)
"3"
"4"
Parents (35)
"5" Definitely
would
55
Parents and patients were asked why they expressed some level of
receptivity to a gene therapy clinical trial. Most popular answers were
that it is a potential cure, reduces or eliminates transfusions, and
promising results to date.
Parents
23
10
7
7
0
9
0
4
6
3
2
1
5
0
0
2
Reason to participate in gene therapy trial
Potential cure
Reduce or eliminate transfusions
Study currently looks promising
Better than transplant options
Help future generations
Better health/ life expectancy
Less invasive than alternative treatments (like
bone marrow transplants)
Improvement in quality of life
Web Survey
Patients
(68 patients, 37 parents)
Number of mentions cited above
56
Those with reservations were concerned about the risk involved, impact
on overall health, and lack of data regarding long term effects, etc.
Parents
11
2
4
3
3
2
2
2
2
1
2
0
0
0
0
1
7
5
0
1
Reason to not participate in gene therapy trial
Risks
Reduction in current health status
Age (too young or old to participate)
Skepticism about trial/ long term effects
unknown
Need more information/ insufficient data
Invasiveness
Fear of activating other disease genes
Time
Not in dire need (health is good now)
Chemo
Web Survey
Patients
(68 patients, 37 parents)
Number of mentions cited above
57
Patient Groups
Most patients and parents indicated that they were not aware of the
ongoing clinical trials involving treatment of thalassemia with the
protein Luspatercept.
9%
17%
83%
Yes
Parent Groups

91%
No
Patients (67)
Yes
No
Parents (35)
58
Patient Groups
Parent Groups
Using a scale of 1 to 5, where 1 is definitely would not participate and 5 is
definitely would participate, respondents were asked to rate their intention/
desire to enter (or have their child enter) a luspatercept trial. Interest ratings
were mixed.
50%
40%
30%
20%
10%
0%
"1" Definitely
would not
"2"
Patients (67)
"3"
"4"
Parents (35)
"5" Definitely
would
59
90%
80%
70%
60%
50%
40%
30%
20%
10%
0%
Have not
Have
participated in a participated in
clinical trial trial in the past
Patients (68)
Number of
clinical trials
% of
patients
None
37%
One trial
25%
2 to 3 trials
15%
4+ trials
13%
DK/ “many”
10%
Web Survey
The majority of patients indicate that they have participated or are participating
in one or more clinical trials. Many parents have children that are too young to
be eligible for most trials.
Currently in a
clinical trial
Parents (37)
60
Patient Groups
Parents
Clinical Trial
13
4
Exjade
7
2
SQUID
6
6
1
0
MRI T2
Desferel
(67 patients, 35 parents)
Other trials and studies cited
by two or three patients
included L1/ ferriprox,
fertility, vitamin D, zinc,
MRI to detect heart
iron/liver iron, quality of
life, stem cells gene therapy
research, pain, iron, Hep C.
Parent Groups
Patients
Number of mentions cited above
Other trials and studies cited by one or two parents included
ferriscan, MRI pituitary iron measurement, MRI T2* (EDICT), zinc,
desferal with Exjade
61
37
19
16
15
14
9
9
8
22
8
11
0
4
4
9
9
0
6
3
3
0
0
0
7
6
5
5
5
4
4
Information Desired
Risk/ Safety vs Benefit
Convenience - Impact on Work/ Family Time
Side effects/ Impact on Life
invasiveness/ Needles
Time/ Length
Importance in Advancement
Benefit
Location
Complicated Study Process / Ease of Participation
Trial History/ Phase (success rate)
Trial Sponsor/ Reputation
Long term effects
Eligibility/ Opportunity
Hospitalization
Compensation
(67 patients, 35 parents)
Patient Groups
Parents
Parent Groups
Patients
Number of mentions cited above
62
What patients find most attractive or
encouraging about the trials that they
are aware of are potential for a cure,
easier compliance, and reduction or
elimination of transfusions.
Patients Attractors
17
15
11
5
4
Potential for a cure
Improved compliance (via better
ways/tasting medicine/treatments)
Eliminating or reducing transfusions
Better quality of life
Better treatment options
Most Attractive
Patient Groups
Number of mentions cited below
Least appealing are the risks.
Patients Detractors
16
14
12
9
6
5
5
3
3
Risks
Difficult treatment plan (# of labs,
etc)
Side effects
Time consuming
Criteria (not qualifying)
Invasiveness
Not well tested (too early in trial
phase)
Chemo-like
Lack of clear data patients can
understand
Least Attractive
63
What parents find most attractive or
encouraging about the trials that they
are aware of are potential for a cure,
reduction or elimination of transfusions
and long term benefit.
Least appealing are the risks, chemolike, travel, too early in trial phase
and side effects.
Parents Detractors
3
Parents Attractors
12
7
4
3
2
Potential cure
Reduces or eliminates need for
transfusions
Long term benefit
Current trials having good results
Free medical care/treatment during
trial
Most Attractive
3
3
3
3
2
2
1
Parent Groups
Number of mentions cited below
Minimum age requirement too high
to qualify
Chemo-like
Travel
Too early in trial phase
Side effects
Restrictions on qualifying for trial
Invasive
Length of time from trial phase to
market
Least Attractive
64
Patient Groups
Parents
22
15
16
12
7
7
5
5
4
0
0
0
0
0
0
2
3
0
0
5
3
3
2
2
Under what conditions would you be most likely to
enter a clinical trial for thalassemia?
Little or no risk
Personal or overall benefit to patients/ known
benefit to child
Parent Groups
Patients
Time involved
Convenience
Full understanding of study details and it’s safety
Non-invasive
Compensation
Child is not doing well on current medical protocol
Improves child’s quality of life
Potential cure
Child has a life-threatening illness
Limited side effects
(67 patients, 35 parents)
Number of mentions cited above
65
When asked about the primary reasons they entered one or more clinical trials,
respondents indicated an interest in advancing treatment options for
themselves and others/ potential cure, a desire for less painful or better options
and/or minimal risk or negative impact in participating.
 “The trials I entered were non-invasive and were to improve cutting edge
research that could tell me more about how I was doing and improve the quality
of life for other patients. It was worth it for me.” - Patient
 “It was just something we did to improve our lives and the lives of all the other
patients.” - Patient
 “Patients before me did studies so I could have a healthier life and I want to give
back. I want future generations to have better treatments than I did.” - Patient
Web Survey
 To advance treatment options/ potential cure (18)
66
 “Potential benefit to their own health long term as well as to others with
thalassemia for the future.” – Parent
 “Importance of data collection for thalassemia research and the "Ex-jade Food
Study" came at a good time as he was struggling with drinking ex-jade on an
empty stomach, each morning. The study gave him options and put him more in
control of taking his medicine.” – Parent
 Less painful/ better alternative (10)
 “As a less painful alternative to intravenous chelation.” - Patient
 “I was on Desferal - painful, nightly, subcutaneous injections lasting 12 hours
for 5 nights each week - and wanted an oral chelator very badly for myself and
others like me.” - Patient
Web Survey
 To advance treatment options/ potential cure (continued...)
67
 Minimal risk/ negative impact (8)
 Doctor’s recommendation/ best option for me (5)
 “It was the doctor’s recommendation.” - Patient
 “At the time, he did not have medical coverage and we needed a way to supply
him with the medication and physician care. Additionally, because he had never
had chelation prior to arriving in the US at age 8, his feretin level was nearly
14,000, so we needed aggressive treatment.” - Parent
Web Survey
 “It was seemingly painless and also consequence-free. And, if it helped to
improve Thalassemia treatment, I thought it was worthwhile.” - Patient
 “It was for a good cause and non-interventional so no serious risks involved.” Patient
 “Risk was low, and results were necessary.” – Parent
68
Patient Groups
Patients
Parents
31
28
13
12
10
6
5
5
5
4
3
0
0
4
4
3
4
0
0
0
Under what conditions would you definitely not
enter a clinical trial for thalassemia?
Parent Groups
Roughly half of patients and 3/4 of parents indicated that they would not
enter (or have their child enter) a clinical trial if judged as risky and/or
including undesirable side effects
Risk(s) too great/side effects
Negative impact on health
Hospital stay required
Too much time involved
If it’s invasive/ too invasive
Too early in the trial
Requires travel/ too much travel
Being performed by a Pharmaceutical company
Lack of information regarding study
Experimental in nature
(67 patients, 35 parents)
Number of mentions cited above
69
 Risk/ benefit ratio not acceptable (8)
 “Too invasive. I'm a rather healthier thalassemia patient due to my adherence to
chelation and low transfusion rate. For me to risk an invasive trial, the reward
would have to astronomically outweigh the risks.” - Patient
 “I did not feel that the risks outweighed the odds for the outcome that would be
reached. The success rate compared to the process did not balance in my
opinion.” - Patient
 “Too high of risk at that time with my health status. I would be interested in it
now.” - Patient
Web Survey
When asked about the primary reasons they opted out of one or more clinical
trials, respondents indicated that the risk/ benefit ratio was not acceptable,
there was little or no track record for the treatment, they disliked one or more
elements of the trial, and/or the logistics of the trial (time, location, etc.) were
not desirable or workable.
70
 Minimal track record (5)
 “It was a Phase I(b) trial and enough safety data was not available to encourage
me to participate.” - Patient
 “I have concerns that a trial with unestablished results could endanger my
progress with my disease.” - Patient
 “Weekly visits were just too onerous and giving weekly samples would have
required more blood draws. Getting jabbed any more than absolutely necessary
was just not appealing.” – Parent
 “Because getting chemotherapy/ getting tested material put back into my body is
not something I'm willing to do.” – Patient
Web Survey
 Dislike one or more elements of treatment (5)
 Logistics (5)
 “I do not have time for trials that require repeat visits.” - Patient
 “New job, do not have the time for all the pre-enrollment tests and visits.” Patient
71
To obtain perceptions of CAF, likes, dislikes, and what is or
would be most valuable from CAF.
Patient Groups
Parent Groups
Using a scale of 1 to 5, where 1 is not at all aware and 5 is very familiar,
respondents were asked to rate their level of awareness or familiarity with
CAF. Most indicated that they had a significant level of familiarity with CAF.
60%
50%
40%
30%
20%
10%
0%
"1" No
awareness
"2"
Patients (67)
"3"
"4"
"5" Very familiar
Parents (35)
73
Web Survey
Some of the most frequently mentioned reasons that patients contact CAF
included insurance questions/ issues, questions about treatment/ options,
conferences/ events, Care Walk, and to make or help with donations/
fundraising..
74
Web Survey
Some of the most frequently mentioned reasons that parents contact CAF
included help with adoption/ expediting adoption, treatment questions/ options,
general advice, conferences/ events, and treatment centers/ doctors/
pharmacies.
75
41%
44%
Patient Groups
Group participants were asked to compare their level of involvement
with CAF five years ago with that of today. Only a minority of
patients and parents indicated a decrease. Over a third to nearly half
noted an increase.
Parent Groups

27%
48%
24%
14%
More
Less
Same
Patients (67)
More
Less
Same
Parents (35)
76
Patient Groups
 “When we first adopted our daughter and after I became award of Cooley's
we had a lot of engagement with Cooley's. They were super helpful in
understanding the standards of care and with connecting with other thals.
Twenty four years late our need is not as big so we don't have nearly the
engagement with them. However, if we needed it I know they are there and do
an excellent job.” – Parent
 “...It’s more than five years ago, previously my job was too busy and hectic to
allow me to really be involved with CAF; now that work is more reasonable,
I've gotten more involved.” - Patient
 “More because my daughter was younger then and as she has gotten older, I
have more time and also realize I need to be more involved to help myself and
others live well with this disease.” - Patient
Parent Groups
Some respondents cited a chance in circumstance as the reason why they are
more or less involved with CAF today.
77
Using a scale of 1 to 5, where 1 is poor and 5 is excellent, respondents were
asked to give their overall impression rating of CAF. Roughly 19 out of 20 rated
CAF a “4” or “5” with the majority of patients and parents rating CAF as
“Excellent”.
3%
"5" Excellent
11%
Web Survey
10%
"4"
21%
"3"
69%
"2"
86%
"1" Poor
Average rating =
4.6
Patients (68)
Average rating =
4.8
Parents (37)
78
Reasons for very favorable impressions of CAF included CAF resources/
knowledge/ information, CAF dedication/ commitment/ personal interest,
exceptional staff, advocacy/ research, development or enablement of support
networks, and serves as a valuable support system for patients and parents.
 “Cooley's Anemia, at the patient level, has brought the necessary Information
critical to my care that has enabled me to keep my physicians on track. They
have also provided the opportunity for me to gather with others with
Thalassemia which in turn has made me feel a part of a family and not as an
isolated case.” - Patient
 “CAF seems to be on top of every aspect of patient care - research, physicians,
standards of care, chelation etc. Love getting the newsletter. Though I haven't
contacted them recently, I know others who have. Eileen always seems to have
answers.” – Parent
Web Survey
 CAF resources/ knowledge/ information (22)
79
 “They are the best source for information for thal patients on research, new
medications and treatments, & raising awareness. They have also been there for
any kind of support I have needed. And I have also enjoyed working with them
for events.” - Patient
 “CAF has amazing easy to access information related to blood disorders.” –
Patient
 “CAF is a great organization in terms of providing resources and information to
patients and families, and in developing support networks.” – Patient
 “They are a reliable source of current information for thalassemia and provide
prompt information to us on new information regarding the safety of medications
or other vital information for our daughter's health. They have been incredible
supportive and easy to ask questions and give advice. We also found the Atlanta
thal clinic, which we love though CAF's website.” – Parent
Web Survey
 CAF resources/ knowledge (continued...)
80
 “Everyone at CAF really cares about patients. CAF employees are more like
family members than unattached workers who have no real connection to the
patients they serve.” - Patient
 “They have gone above and beyond to get me information, to help other patients
in need, and they have helped me when I was in need. They are an amazing
support to our cause. Without the Cooley's Anemia Foundation, this disease
would not be as close as it is to a cure. They have taken this cause seriously, and
have placed themselves within a structure that can compete with other highly
competitive and recognizable causes. I'm proud of this foundation.” - Patient
 “The impression I got is that not only does the Cooley's Anemia Foundation care
about children with thal, but they care about families.” – Parent
 CAF is on point about all things thalassemia-related. I really appreciate
everyone's hard work and dedication! – Parent
Web Survey
 Dedication/ commitment/ personal interest (16)
81
 “They work very hard to find a cure for us. they are always there for support.
Gina Cioffi is doing an excellent job. So is her staff. Great group of people.” –
Patient
 “The people there are dedicated to improving the lives of those with thalassemia
and take their work seriously.” – Patient
 “The staff are very helpful, kind and accommodating.” – Patient
 “I worked with Eileen Scott, who is amazing! She alleviated our fears. Helped to
recommend clinical trials, put us in touch with doctors, always available via
email and phone. Quick to respond and very real!” – Parent
 “Eileen was extremely helpful in getting us the letters. Then months later
actually followed up inquiring about how our daughter was doing.” – Parent
 “The staff is fantastic! The foundation works hard to help those with
Thalassemia.” – Patient
Web Survey
 Exceptional staff (12)
82
Web Survey
“...they are the only source for cutting edge
information when most of the information out there is
antiquated. They are staffed by the most thoughtful
and caring people in the non-profit industry. They
provided us with hope when we absolutely had none.
Our son would not be doing half as well as he is
today without the strength and the tools that CAF
gave us to be able to manage this disease. They
empowered us to be able to do what we are doing for
our son. We were absolutely two deer in the
headlights when we heard that our son had beta
thalassemia major. We were new parents, did not
know how to care for a child let alone a disease that
we were told was a death sentence. Once call to CAF
changed all of that and it has been one miracle after
another since then. We have a healthy, happy,
thriving little boy and we have CAF to thank for that.
CAF gave us the tools to manage this disease and
allowed us to care for our son when we couldn't do it
ourselves. We are eternally grateful.”
83
 Advocacy/ research/ advancing the cause (10)
 “It's the only resource at this point that provides me with any information, it has
really mobilized a small group of people around an issue, CAF does a lot of
advocacy which is why we have so many trials and treatment options.” - Patient
 “They are doing a great job of raising awareness for a rare disease.” - Patient
 “I love all the patients I have been able to meet through the organization. I also
love all the workers and doctors who have affiliations with CAF.” – Patient
 “Great communication, purpose, cause, great people, great at connecting others
to build our network.” - Parent
 Valuable support system for patients and parents (7)
Web Survey
 Development or enablement of support networks (8)
 “They are an unbelievable support system to their patients.” - Patient
84
 “They are open and available, have free services not otherwise allocated to
people like us. They help you any way possible, they never say no or leave you
hanging without answers or connections.” - Patient
 “Eileen contacted me right away. Got me in touch with nurses and doctors who
assist me in crafting a letter to the insurance company explaining why I needed
Exjade.” – Patient
 “Responsive staff, good communications, effective and educational events –
especially the conference.” – Patient
 “They have always been very helpful and responsive.” – Parent
Web Survey
 Very responsive/ assessable/ easy to reach (6)
85
Patient Groups
 Support/ encouragement/ sense of community (25)
 “I believe that they really care about all of us. I believe that that want us to
have a better quality of life, and to find a cure for thal. I feel like I am part of a
community.” - Patient
 “They are an amazing resource! They are the reason I feel such support from
the community and don't feel alone in this world.” - Patient
 “The warm friendly people that are always available to talk whenever you
need them, that's priceless.” - Patient
 “How open they are to questions and asking if you need any help with
anything from medical expenses or treatment, etc. They make you feel like
family…” - Parent
 “Most valuable is the relationship I have with them. They were there for us in
the beginning when we didn't know where to turn to. Plus the dedication they
have for their patients.” – Parent
Parent Groups
Focus group participants were asked what they vale most from CAF. Most
popular responses were support/ encouragement/ sense of community,
information/ expertise/ being kept up to date, and advocacy.
86
Patient Groups
 “The wealth of resources and information they gather and present to us information such as clinical trials info, new treatments. Information regarding
guidelines, things we need to do to better care for ourselves.” - Patient
 “I appreciate that they care enough to send me mail and keep me informed
even though I'm not involved.” - Patient
 “The educational information about thal, thal treatments, and new best
practices of care that are available and updated on their website, the
immediate mailings to address new concerns such as the need to stop
chelation if the child has a fever, as well as the support from Eileen anytime
we have contacted her.” - Parent
 “They are a readily accessible source of information of information, especially
about clinical trials and new developments in thalassemia treatment.” –
Parent
 “CAF is the most comprehensive resource for anyone with Thalassemia” Patient
Parent Groups
 Resources/ information/ being kept up to date (22)
87
Patient Groups
 “I love that CAF has been and continues to advocate for thal patients whether in
in social issues, political issues, continuing research and networking.” – Parent
 “CAF is by far the strongest patient advocate organization.” – Parent
 “I love their dedication to their cause. They have really improved their services
to patients in the last 10-20 years and I feel that Thalassemia is a better known
organization and disease due to the work they do to get their name out there,
and raise public awareness.” – Patient
 “They are dedicated to thalassemia. They work also to change law and policy as
it relates to thalassemia. They do great work on Capitol Hill. They also fund a
lot of research.” – Patient
Parent Groups
 Advocacy/ advancing the cause (12)
 Exceptional staff (10)
 “That the people there care about what they are doing and truly want to support
and help.” – Parent
 “I love their social worker, Kathleen Durst that is available to patients. I value
that they go to DC to fight for funding and all their fund-raising events.” –
Patient
88
Patient Groups
 “I value the ability to meet others with my diagnosis and learn from them, as
well as the information they have and their commitment to educating others
and improving our lives.” – Patient
 “Their patient/ parent community building…” – Parent
 “I value the community of people. I value knowing that there is a group of
people I can express my issues to and that they understand and work with
Thal. I also value the people (doctors and patients) that I meet.” – Patient
Parent Groups
 Ability to network with other patients/ parents (6)
 CAF conferences (6)
 “The conferences are a great place to ask questions.” – Patient
 “…the opportunity to attend conferences.” – Parent
 Research/ studies/ trial information (5)
 “I like that they keep us posted with new thal related research and news.” Patient
 “Information and newsletters about the study trials for Thal.” – Patient
89
The small minority of respondents who rated CAF a “3” in terms of overall
impression indicated that they have not contacted CAF/ had no interactions
(and thus could not rate CAF). Two patients brought up changes since TAG.
 “The individual outreach has decreased significantly since TAG (Thalassemia
Action Group) was dissolved. When TAG was active it made us a community that
could reach out and touch one another across the whole US and gave people
hope and connection. Now if you want connection to Thals you have to go to a
center of excellence or get on the Internet to talk to someone from CAF but it will
not necessarily allow you to connection to another Thal.” – Patient
 “They do not treat all patients equally and those who were on TAG got shut out. I
have reached out to them with no response.” – Patient
Web Survey
 Integration of TAG members (2)
90
A handful of other isolated comments included...
Web Survey
 “I am sure they are helpful for patients with thalassemia major but nothing they
could add for intermedia. Not sure they are that familiar with it. Its the rare of
the rare.” – Patient
 “I feel like when I ask questions I get transferred to the next person.” – Parent
91
Patient Groups
 CAF presence/ conference in NE/ not across U.S. (10)
Parent Groups
Most patients and parents could not think of anything that they valued least.
The only dislikes or things they valued least included CAF presence and
conference focus in the northeast/ not across U.S. and that TAG was
disbanded.
 “I haven't come across anything as of yet. But I wish CAF was more
accessible around the country physically whether by having more than one
office or multiple regional conferences to keep the Thal community and the
Thal medical researchers close together and consistently.” – Patient
 “I do feel as if the focus is on certain regions of the country ... and its a bit
isolating for those of us who live in other parts of the country. I would love to
go to more conferences or to participate in things like the care walk, but I just
don't know if there is any sort of population in Atlanta, I know in Detroit it was
super small and disconnected.” – Patient
 “There should be branches across the US.” – Patient
 “Events are often on the east coast.” – Patient
92
Patient Groups
 “I wish we were closer to attend some of their events.” – Parent
 “That they are in New York and don't have smaller chapters spread out.” –
Parent
 “I wish they could help organize local chapters more, more local support in
different regions of the country.” – Parent
Parent Groups
 CAF presence/ conference in NE (continued...)
 TAG disbanded (3)
 “I dislike the fact that TAG was disbanded. TAG was a great resource and
provided an invaluable outreach to other patients. This is not only a physical
disease- the many psyco-social issues that surround the patients could only be
addressed by patients. TAG provided an emotional shelter.” – Patient
 “Losing the TAG group.” – Patient
93
Patient Groups
Parent Groups
Respondents were asked how CAF could become even more valuable. The
most popular responses included more networking opportunities/ a forum
for patients/ parents (online, in-person), CAF presence/ conferences in other
parts of U.S., training/ education/ network for hematologists/ medical
community, and (more) information/ support on clinical trials.
 More networking opportunities/ a forum for patients/
parents (online, in-person) (9)
 “CAF could be more valuable to me if they provided more of a place where
patients could really communicate with each other. So, we can discuss what we
are going through.” – Patient
 “I would love for a way to better stay connected to others like me of my age in the past there were forums and I would find peace in reading those but I
haven't seen those recently.” – Patient
 “For me, CAF could be more valuable if there were opportunities to see
patients from around the country more often than once a year .” – Patient
 “Maybe have a website forum for thal patients to chat and ask questions. –
Patient
94
Patient Groups
 “Find a way for more social interactions between patients across the
country.” – Patient
 “More interaction with other patients. I know that's impossible because it
takes money and time, but it would be great if we had more gatherings like the
annual conference more than annually.” – Patient
 “Additional networks built through CAF - parent networks, etc.” – Parent
 “More frequent sessions like these where parents can provide input that could
help CAF and other parents.” – Parent
Parent Groups
 More networking opportunities/ a forum for patients/
parents (continued…)
 CAF presence/ conference in regions outside NE U.S. (8)
 “Be more accessible for people on the west coast.” – Patient
 “I wish they could help organize local chapters more, more local support in
different regions of the country,” – Parent
 “Setting up regional conferences for patients and their families so that we
wouldn't have to travel so far.” – Parent
95
Patient Groups
 “Providing training and education to hematologists and
training sites of hematologists.” – Parent
 “It would be helpful if there was a way for them to provide
information to those in the medical field, so they can
become more knowledgeable on thalassemia. Many
doctors who do not deal directly with Thalassemia patients
do not have in-depth knowledge of the disease.” – Parent
 “Create a network for Hematologist that treat Thal
patients. I believe this would be especially helpful to
patients who are not near a Treatment center. This way all
Thal physicians have the same information.” – Patient
 “Get more information out about thalassemia to areas that
are not familiar with it. Help to link all the doctors
together - the patients who aren't getting transfused at
clinical centers, and have hematologists outside of a
network that may only have one or two patients.” – Patient
Parent Groups
 Training/ education/ network for
hematologists/ medical community (7)
96
 “I think they can be more valuable to me assisting with the clinical trials
process. It would be great if they offer in depth help with apply and getting
qualified for trials.” – Patient
 “Send more information on clinical trials, send email blasts monthly with
medical information or future events.” – Patient
“...it would be so great to get notifications that say, ‘Hey, the Bluebird trial is now
being offered at these additional centers.’ It would be so nice to have that kind of
information pushed out.” - Parent
Patient Groups
Parent Groups
 Information/ support on clinical trials (5)
 Information on alternative treatments (3)
 “Have a greater outreach to more holistic care. Also diet and
more natural treatment options. Also more tips on how
patients can best juggle the communication between the
multiple doctors and specialists we see.” – Patient
 “I would like to have a forum of holistic health methods that
patients can share with one another.” – Patient
97
Patient Groups
 “Practically all of the information that we get in America is just that any of the
research that’s going on here, what really lacks is what’s going on in other
countries and nutrition was mentioned, there’s a whole school just like so much
other information, alternative information that other research gets going on in
other countries, I’ll take Israel for instance, about how to chelate naturally
using food and other things.” – Patient
Parent Groups
 Information on alternative treatments (continued...)
98
Patient Groups
 “Creating better patient resources - doctor locator guides, plans of care,
guidelines for travel, pregnancy research, etc.” – Patient
 “Make sure every patient has a packet of information for their parents,
doctors, schools. Maybe assign each patient to a buddy, to talk to or penpal
with.” – Parent
 “Online resources or forms to submit, forums for discussions, with surveys,
reaching out to patients addressing specific challenges. in other words
surveys would help define what specific challenges a patient needs to address,
needs assistance with. Then appropriate outreach can be set up.” – Patient
 “I was thinking about videos for patients who are teenagers, who are getting
to know what to do or how to deal with thalassemia. ...help with other things
more in terms of how to take care of thalassemia or what to do with insurance
or what appointments I need to go to and stuff like that. An educational,
animated video kind of thing might be useful.” – Patient
Parent Groups
 Expanded/ more patient resources (5)
99
Ensure thal centers are in-sync.
Develop a thal patient registry.
Reach out to parents more.
Keep up the monthly email updates
Conduct a study demonstrating the importance of new/fresh
blood.
Patient Groups
Help organize more local chapters.
Parent Groups
Sampling of suggestions to help even more…
Provide information to those in the medical field to address
their lack of thalessemia knowledge.
Offer even more information.
Continue to address the importance issues (e.g. blood safety,
insurance coverage, better medicine, the cure).
Send out individualized updates.
More parent network building.
Set up regional conferences for patients and their families to
reduce travel.
Develop more programs for new thal patient.
Create an online directory of doctors along with patient
reviews.
Make themselves available to consult with physicians.
10
0
Patient Groups
Parent Groups

“Have an app with publications, clinical trials,
charting of hgb, ferritins, other labs, chelation,
weight, height, medication history, other resources.
testing that needs to be done monthly, annually,
appointments, etc.” - Parent
101
38%
62%
Yes
Parents (35)
Patient Groups
Parent Groups
Patients (67)
Focus group patients were asked “Are you
aware that CAF's Patient Outreach Director
is available to help provide information on
the clinical trial process?” Just over 1/3 of
patients and parents indicated that they were
aware of this.
No
36%
64%
102
Patient Groups
Most patients indicated that they were aware that CAF has an on staff
Social Worker. The slight majority of parents noted that they were not
aware of this.
40%
60%
Yes
No
Patients (67)
Parent Groups

45%
55%
Yes
No
Parents (35)
103
Patient Groups
Most patients and parents indicated that they were aware that CAF
has an on staff Case Manager.
38%
45%
55%
62%
Yes
No
Patients (67)
Parent Groups

Yes
No
Parents (35)
104
Patient Groups
 “If CAF could have more doctors involved on their site that would be nice. if
doctors could write some articles or share data that would be great.” Patient
 “People are living longer with Thalassemia and as I said before that working
adult professionals have needs so that they can continue to work and not have
to sit home and collect disability due to their appointments.” - Patient
 “I would like to discuss options for adult patients to see adult hematologists
that have late night clinic hours for working patients.” - Patient
 “There are many pediatric hematologist operating with outdated information
with regard to quality of life and even life expectancy of thal kids. They are
very detrimental to the attitudes of parents and potential parents. I'm not sure
how to get better information out to doctors.” – Parent
Parent Groups
Additional comments or suggestions offered by patients and parents
included...
105
Patient Groups
Parent Groups
 “On the topic of giving info on support perhaps CAF could have a list of
families who are willing to accept phone calls or contact from new families.” –
Parent
 “…don't grow stagnant and keep doing the same things over and over, look to
reinvent yourself and try new things and raise the bar. CAF has to grow.” –
Parent
 “We need more adult hematologists that care properly for thalassemia
major/intermedia patients.” - Patient
 “Up and coming treatments, updates on the gene therapy trials, more
information about stem cell transplants.” – Patient
 “You could ask what is the rate of care that you receive and why?” – Patient
 “Perhaps studies on psychology / patient perspectives. oh, better ways to
connect doctors to the latest information about Thalassemia - That would be
very important. Yes, better ways for doctors to connect with CAF and the latest
Thalassemia treatment options. Ways for doctors to collaborate with other
specialists. Doctor education and outreach programs.” - Patient
106
Patient Groups
Parent Groups
 “One of the things I've found handy is my daughter actually has a medical ID
bracelet and we found it out by another patient that has Beta Thalassemia. I don't
really see a lot of people promoting that. Whether it's the doctors or I haven't seen
anything on CAF about it, but I think it's something really good. Especially for
little kids where if, god forbid, you're in a car accident with them and you can't
talk and your child is too young to explain anything, having that medical ID
bracelet or knowing you have this option of getting this medical ID bracelet, that
way if something were to happen they could go to this website and get all the
information regarding their blood type, the hospital that they go, when was the
last transfusion, any immunization records. Something like that.” – Parent
 “We hear about clinical trials, but actually getting on the trials isn't so straight
forward. I hear about this one is under clinical investigation, but if I think I'm a
right candidate, how would I get it? It forces us to rely heavily on our doctors,
who are, obviously, very busy. I'm not saying they don't try to help, but it would be
nice if we could be more of our own advocates and make our own calls and say
‘Hey, I read about this trial. I heard about this trial. This is my situation. I'd like
to be involved." - Patient
107
Patient Groups
Parent Groups
 “I don't know if this is something that CAF can do, but I think it would be a
really good resource for thalassemia patients to contact CAF for the ability to
speak to maybe a nutritionist or a dietitians or fitness trainers, things like that.
Our bodies have a very different tendency to react to blood transfusions and
chelators. Sometimes I hear people say that thalassemia patients have a
specific dietary regimen, but I've never ever been on a dietary regimen myself.
I think to maintain a very healthy lifestyle is really important. If we have
things like that at our disposal, it would make us well informed patients. We'd
be more in charge of our health in that way instead of dealing with blood
transfusions and everything and not living a healthy lifestyle. ...That and
clinics, the psychologists or whatever, just having those specialists or
therapists available to us. I think that would be beneficial.” - Patient
108
Patient Groups
Parent Groups
 “I think if CAF made a booklet just specifically for the doctors so that
whenever we get home, and the doctors are saying, ‘Oh, I'm an expert, I know
what I'm doing,’ but then they really don't. CAF could maybe have something
to send to them because they don't want to work with us parents because they
think we don't know what we're talking about. It doesn't matter if you said
that you read this on the internet. They don't believe that either.” - Parent
 “I’d really like some sort of very specific comparison charts or something
along those lines to compare different chelation options because we’re getting
different recommendations. The specialty clinic versus the local hematologist
and it’s really stressful to have get online and just base my final decision on
which one we’re going to do based on what I can find on the Internet. ... I’d
like something that compares the positives to negatives and risks of each
chelation then.” - Parent
109
Profile data and group
composition
3%
3%
Beta thalassemia
intermedia
Alpha thalassemia major
9%
22%
69%
8%
Hemoglobin H Disease
87%
E beta thalassemia
Patients (68)
Web Survey
Beta thalassemia major
Parents (37)
111
Male
Female
49%
69%
Patients (68)
51%
Web Survey
31%
Parents (37)
112
Patient Groups
Italian
39%
Greek
Non-Italian/
Non-Greek
Parent Groups
55%
6%
60%
50%
40%
30%
20%
10%
0%
Caucasian
Patients (67)
Asian
South Asian
Parents - child is… (35)
Other (Hispanic,
African American,
etc.)
113
40%
30%
10%
0%
1 to 3
4 to 6
7 to 13
14 to 20
Patients (68)
21 to 29
30 to 39
40 to 49
50+
Web Survey
20%
Parents (37)
114
24%
Northeast
49%
Midwest
South
16%
12%
Patients (68)
West
46%
22%
35%
Web Survey
11%
16%
16%
Parents (37)
115
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
33
Caucasian
M
Every 2 weeks
MW
Chicago, IL
46
Caucasian
F
Every 2 weeks
NE
New York, NY
21
Caucasian
F
Every 3 weeks
S
Baton Rouge, LA
32
Middle Eastern
F
W
Irvine, CA
22
Asian
American
Asian
American
Every 2 or 3
weeks
M
Every 3 weeks
NE
Cambridge, MA
M
Every 3 weeks
W
San Francisco, CA
23
Caucasian
M
Every 3 weeks
S
Atlanta, GA
54
Caucasian
F
Every 2 weeks
NE
Homdel, NJ
30
116
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
22
Asian
American
F
Every 3 weeks
W
Salt Lake City, UT
39
Caucasian
F
Every 3 weeks
NE
Madison, NJ
42
Caucasian
F
Every 2 weeks
W
Kingston, WA
42
Caucasian
M
Every 3 weeks
S
Winter Park, FL
52
Caucasian
M
Every 2 to 3
weeks
NE
Newark, DE
41
Caucasian
F
Every 4 weeks
MW
Arlington
Heights, IL
117
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
28
South Asian
American
South Asian
American
South Asian
American
South Asian
American
South Asian
American
South Asian
American
South Asian
American
South Asian
American
F
Every 3 to 4
weeks
S
Atlanta, GA
M
Every 3 weeks
NE
Bayonne, NJ
F
Every 4 to 5
weeks
NE
Rockville, MD
M
Every 2 weeks
S
Raliegh, NC
F
Every 3 weeks
NE
Bensalem, PA
M
Every 4 weeks
NE
Baltimore, MD
M
Every 4 weeks
NE
Washington, DC
F
Every 4 weeks
NE
New York, NY
26
38
38
27
34
38
38
118
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
35
Asian
American
F
Every 3 weeks
W
Pleasonton, CA
38
Caucasian
F
Every 3 weeks
NE
New York, NY
53
Caucasian
F
Every 4 weeks
MW
Chicago, IL
25
Asian
American
F
Every 3 to 4
weeks
S
Memphis, TN
58
Caucasian
F
Every 2 weeks
NE
Interlaken, NJ
41
Hispanic
F
Every 3 weeks
W
Gilbert, AZ
35
Caucasian
F
Every 3 weeks
W
Anchorage, AK
49
Italian
American
F
Every 2 weeks
NE
Melville, NY
119
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
26
Caucasian
F
Every 2 weeks
NE
New York, NY
W
Austin, TX
61
Caucasian
M
No (visits doctor 2
to 3 times per
year)
44
Caucasian
F
Every 3 weeks
NE
Throop, PA
30
Caucasian
F
Every 3 weeks
NE
Port Chester, NY
45
Caucasian
F
Every 2 weeks
NE
Hackettstown, NJ
49
Caucasian
F
NE
Bronx, NY
26
Asian
American
Every 2 to 3
weeks
F
Every 3 weeks
MW
Minneapolis, MN
19
Caucasian
F
Every 3 weeks
NE
Buffalo, NY
120
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
50
Caucasian
F
Every 3 weeks
NE
New York, NY
55
Caucasian
F
Every 3 weeks
W
Costa Mesa, CA
M
Used to - every 4
weeks
S
Houston, TX
F
Every 4 weeks
NE
Mineola, NY
30
42
South Asian
American
Caucasian
(Greek)
56
Caucasian
F
S
Tampa, FL
35
Asian
American
None, visit doctor
once/6 months
M
Every 3 weeks
NE
New York, NY
47
Caucasian
F
Every 3 weeks
NE
Philadelphia, PA
32
South Asian
American
M
Every 2 weeks
S
Atlanta, GA
121
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
29
South Asian
American
M
Every 2 weeks
W
Glendora, CA
45
Caucasian
F
Every 2 weeks
MW
Omaha, NE
41
Caucasian
F
Every 4 weeks
NE
Arlington, MA
38
Caucasian
F
Every 4 weeks
NE
Ridgewood, NJ
F
Three times per
year
MW
Wichita, KS
F
Every 3 weeks
NE
Newark, DE
26
24
South Asian
American
South Asian
American
122
Patient Groups
Age
Ethnicity
Gender
41
Caucasian
39
20
26
28
27
57
Transfusions
Region
City, State
F
Every 2 weeks
NE
East Rockaway,
NY
F
Every 2 weeks
NE
Long Island, NY
South Asian
American
Asian
American
African
american
Asian
American
Asian
American
F
Every 3 weeks
W
Redondo Beach,
CA
M
Every 3 to 4
weeks
MW
Cassopolis, MI
M
Every 3 weeks
NE
Hicksville, NY
M
Every 3 weeks
NE
Hicksville, NY
Caucasian
F
Every 3 weeks
NE
Brooklyn, NY
123
Patient Groups
Age
Ethnicity
Gender
Transfusions
Region
City, State
30
Caucasian
F
Every 2 weeks
NE
Homdel, NJ
45
Caucasian
F
Every 3 to 4
weeks
MW
Glenview, IL
F
Every 3 weeks
W
Eagle River, AK
M
Every 3 weeks
W
Danville, CA
36
33
Asian
American
Asian
American
29
Other
M
Every 3 weeks
W
Richmond, CA
21
Asian
American
M
Every 3 weeks
W
Redondo Beach,
CA
18
Other
F
Every 2 weeks
W
Freemont, CA
30
Asian
American
M
Every 5 weeks
MW
Minneapolis, MN
124
Ethnicity
Gender
Transfusions
Region
City, State
Son is 6
Asian
American
F
Every 3 weeks
S
Cumming, GA
Son is 7
Caucasian
F
Every 3 weeks
NE
West Babylon, NY
Son is 7
Caucasian
M
Every 3 weeks
NE
West Babylon, NY
F
Every 2 weeks
MW
Lexington, MO
F
Every 3 weeks
S
Mooresville, NC
M
Every 3 to 4
weeks
MW
Decatur, IL
Daughter South Asian
is 14
American
Middle
Son is 14
Eastern
Daughter
Caucasian
is 5
Son is 22
Asian
American
M
Every 3 weeks
W
Torrence, CA
Son is 7
Caucasian
M
Every 3 weeks
NE
Southington, CT
Son is 17
Caucasian
F
Every 2 to 3
weeks
NE
Nutley, NJ
Parent Groups
Age
125
Ethnicity
Gender
Transfusions
Region
City, State
Son is 6
Caucasian
F
Every 2 weeks
W
Rio Rancho, NM
Son is 6
Caucasian
M
Every 2 weeks
W
Rio Rancho, NM
Daughter
is 15
Daughter
is 15
Daughter
is 5
Asian
American
Asian
American
F
Every 4 weeks
S
Dacula, GA
M
Every 4 weeks
S
Dacula, GA
Caucasian
F
Every 3 weeks
NE
Ambler, PA
Son is 3
Caucasian
M
Every 4 weeks
NE
Rochester, NY
Parent Groups
Age
126
Daughter is
4.5
Ethnicity Gender
Caucasian
Asian
American
Daughters are
Asian
3 and 7
American
Daughters are
Asian
3 and 7
American
Asian
Daughter is 5
American
Sons are 3
South Asian
and 5
American
Daughter is
Asian
4.5
American
Sons are 16
Transfusions
Region
City, State
F
Every 3 to 4
weeks
NE
Sayreville, NJ
F
Every 4 weeks
NE
Dover, NH
F
Every 3 weeks
NE
Grove City, PA
M
Every 3 weeks
NE
Grove City, PA
F
Every 4 weeks
W
Farmington,
UT
F
Every 4 weeks
S
Orlando, FL
F
Every 3 to 4
weeks
NE
Richmond, VA
Parent Groups
Age
127
Ethnicity
Gender
Transfusions
Region
City, State
Daughter
is 10
Daughters
are 7 and
11
Daughter
is 9
Asian
American
F
Every 3 weeks
MW
Decatur, IL
Asian
American
F
Every 3 weeks
W
Madera, CA
F
Once/ year
MW
Salem, WI
F
Every 3 weeks
MW
Sapulpa, OK
Asian
American
F
Every 3 weeks
NE
Troy, PA
Caucasian
M
Every 3 weeks
NE
Sayerville, NJ
Asian
American
F
Every 3 weeks
NE
Flushing, NY
Son is 8
Daughters
are 5.5
and 7
Daughter
is 4
Son is 10
Asian
American
Asian
American
Parent Groups
Age
128
Ethnicity
Daughter is
Caucasian
22 months
Sons are 18
Caucasian
and 28
Sons are 18
Caucasian
and 28
Son is 2
Caucasian
Daughter is
Asian
2
American
Daughter is
Asian
13
American
Gender
Transfusions
Region
City, State
F
Every 4 weeks
S
Charlotte, TN
S
Suwanee, GA
S
Suwanee, GA
F
M
Every 3 to 4
weeks
Every 3 to 4
weeks
M
Every 4 weeks
NE
Lodi, NJ
F
Every 3 to 4
weeks
NE
Floyd, VA
F
Every 3 weeks
W
San Antonio, TX
Parent Groups
Age
129