prepared by: 2015 patient and caregiver study full report may 2015

Prepared by:

2015 Patient and Caregiver StudyFull Report

May 2015

2

Objectives and methodology 3 Summary of findings 5 Detailed findings

Information Sources 12Treatment Centers 22Adherence 25Quality of Life 36Clinical Trials 51About CAF 72Profile of Group Participants 106

Table of

Contents

3

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

Research Objectives

4

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

Methodology

68patients

37parents

1021

4participants

groups …most of these and others not in a group completed an online survey…

(67 patients, 35 parents)

105 respondentsin total in the online survey

5

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.

Summary of Findings

6

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.

Summary of Findings

7

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.

Summary of Findings

8

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.

Summary of Findings

9

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.

Summary of Findings

10

Patients Parents

Number of respondents 68 37

Percent reporting transfusions every 2 to 4 weeks

93% 92%

Adherence rating (5 = fully adherent) 4.4 4.8

Are or have participated in a clinical trial

63% 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 Summary

Web

Su

rvey

Detailed Findings

Focus Groups and Web Survey

Sources of Information

13

Preferred Sources of Information

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

Number of mentions cited above

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) 26Doctors/Hematologists/Clinics 24Online websites (including the drug

manufacturers website) 19Facebook groups 14

Other patients 6Conferences 6

Mobile apps (like ThalPal) 4(67 patients, 35 parents)

14

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

Sources of Information

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

peer health coach endocrinologisthematologist

15

What Patients and Parents are Looking For

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 effects, older patients, and standards of care.

Pare

nt

Gro

up

s

How often? # Mentions

As needed 11Daily 9

Monthly 7Quarterly 4

Yearly 4

Pati

en

t G

rou

ps

16

New drug developments Long-term effects of thalassemia and the treatments

Clinical trials Gene therapyHow other patients are handling treatments Bone marrow transplants

Chelation therapy Fertility and pregnancyWhat’s working for other thal patients About tests

About possible cures Drug interactionsRecommended regimen of tests and check-ups

The effects that Exjade can have on the liver

Stem cell research Cause of death of thalassemia patients

Symptoms Diet and supplements

The biology of thalassemia More information than what is currently available

Insurance information Anything CAF provides is goodMuscle deterioration AdoptionThe standard formula for transfusion amount based upon hemoglobin counts of children

Examples of Information

Needs Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

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

17

Preference in Accessing Information

Patients Parents How do you prefer to discover or receive

this information?

30 17 Online/ Website/ Social Media

19 9 Hematologist/ Doctor/ Nurse

18 10 Email

12 1 Conference

9 6 Regular Mail

5 2 Thal Centers

5 0 Other Patients

3 0 In Person0 3 Mobile app



Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

Like Facebook push notifications, it would be so nice to have information pushed out. --Parent

Most patients and parents prefer to access information about thalassemia online, via email or through their doctor.

18

What is Lacking in Information Available

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


A range of items surfaced when respondents were asked what was lacking in information available about thalassemia...

What is lacking in the information available about thalassemia? # Mentions

Clinical trial information/ updates 8Hard copies of information about Thalassemia (studies, patient care, newsletters, etc) 7Studies on long term effects/complications of the disease (older patients) 5Information for female patients on puberty, pregnancy/fertility 5Information on Insurance Issues for patients (causes them to have to choose between therapies) 5Info on chelation therapies 5Doctors familiar with the disease 5


19

Some patient and parent comments on what is lacking included...

“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

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


20

“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 also- someone said yesterday that patients with thal intermedia can't become iron overloaded. Completely and utterly false.” - Patient

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


21

“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

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


Treatment Centers

23

Awareness of Treatment Centers

Patients Parents

Which major treatment centers come to mind for persons with thalassemia? (unaided)

29 22 Oakland Childrens

26 14 Philadelphia Childrens/ CHOP

22 5 New York Presbyterian Childrens

16 17 Boston Childrens

16 4 Cornell

14 3 LA Childrens

7 6 Chicago Childrens

4 3 Texas Childrens

3 15 Atlanta Childrens (Children's Healthcare of Atlanta/ CHOA)(68 patients, 37 parents)


Web

Su

rvey

24

Usage of Treatment Centers

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 Oakland Childrens

6/7 5/5 Philadelphia Childrens/ CHOP

11/6 - New York Presbyterian Childrens

6/2 4/3 Boston Childrens

7/8 2/1 Cornell

6/2 - LA Childrens

2/1 - Chicago Childrens

1/0 - Texas Childrens

3/1 5/4 Atlanta Childrens (Children's Healthcare of Atlanta/ CHOA)(68 patients, 37 parents)


Web

Su

rvey

Adherence to Treatment Plan

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.

26

Average adherence rating now is

4.4

81%

14%

Average adherence rating now is

4.8

Level of Adherence Today

Patients (68) Parents (37)

59%

31%

7% "5" Fully adherent

"4"

"3"

"2"

"1" Not at all adherent

2%

Web

Su

rvey

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.

3%2%

3%

27

Average adherence rating then is

4.0

Level of Adherence Five Years Ago


48%

21%

16%

9%6%

"5" Fully adherent

"4"

"3"

"2"

"1" Not at all adherent

60%

19%

14%

Web

Su

rvey

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.

3%5%

Average adherence rating then is

3.7

28

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 medication as prescribed (18) “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

Where Not Fully Adherent

Web

Su

rvey

29

Appointments/ exams/ tests (6) “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

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

Where Not Fully Adherent

Web

Su

rvey

30

“I suspect he might have skipped medication when he studied late and fell asleep.’ - Parent

Why Not Fully Adherent

Web

Su

rvey

Some of the more frequently mentioned reasons for not being fully adherent included:

Logistics/ work/ not enough time (9) “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

31

Painful/ side effects (4) “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

Su

rvey

32

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 Cost/ lack of insurance (3)

“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

Su

rvey

33

Parents cited lack of proper treatment for those adopted from China. Some comments included:

Improper, little or no treatment in China (8) “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

Su

rvey

34

Patients

Parents

What has been the most valuable support mechanism in terms of enabling better adherence for your treatment for thalassemia?

47 9 Family/Peer/Patient support system15 7 Doctor/medical staff support system5 0 Desire to live/be healthy4 0 Positive attitude3 0 Deaths of other patients

3 7Understanding importance through information about disease/ Understanding the ‘why’/info about disease/treatments

3 0 ThalPal peers0 8 Advice from other patients’ parents0 2 Changing how meds are given0 2 CAF

(67 patients, 35 parents)Number of mentions cited above

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

Most Valuable Support for Adherence

35

Patients

Parents

Is there something that was beneficial in the past in terms of improving your adherence? Or something that would have been helpful?

12 0 Oral chelation10 2 Support (family/friends)6 5 Knowledge/better understanding3 0 Accessibility to treatment (hours, location)2 0 Less side effects1 0 One day off1 0 Better insurance

0 4 Getting to the United States (adopted from foreign countries)(68 patients, 37 parents)Number of mentions cited above

What Has or Would Help Adherence

Web

Su

rvey

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.

Quality of Life

To examine patient views of his or her quality of life, what impacts this, and conditions under

which this could be improved.

37

Overall Quality of Life

5Very good

4.1(patients –

focus groups)

4.5(parents -

focus groups)

1Not

good

Pati

en

t G

rou

ps

Pare

nt

Gro

up

sW

eb

Su

rvey

4.2(patients -

web survey)

4.5(parents -

web survey)

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

38

Much worse today

Somewhat worse today

No change Somewhat better today

Much better today

0%

10%

20%

30%

40%

50%

60%


Quality of Life Today vs. Five Years Ago

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.

Web

Su

rvey

39

Why Quality of Life is...


Better today: New/ better treatment

options (11) More adherent today (5) Better educated/

informed/ make better choices (3)

Worse today: Complications, pain

and/or fatigue increasing with age (8)

More pain today (4)

Better today: Improper, little or no

treatment in China (prior to adoption) (11)

New/ better treatment options (6)

Health has improved (3)

Worse today: Treatment centers less

flexible today (hours, etc.)/ logistics of treatment (3)

Web

Su

rvey

40

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.).

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

What Would Improve Quality of Life

Web

Su

rvey

41


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

“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

42

Better insurance/ financial support (12) “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

Su

rvey

43

Easier logistics for patients to get treatment (7) “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

Su

rvey

44

Greater knowledge/ expertise in thal treatments and best practices on the part of hematologist/ medical community (5) “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

Su

rvey

45

Greater knowledge/ expertise (continued...) “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

Su

rvey

46

Ways to Improve

Quality of Life

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

47

Level of Pain


22%

29%25%

19%

4%

No pain at all - "1" "2"

"3" "4"

Very painful - "5"

49%

Web

Su

rvey

5%

11%

35%

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.

48

0%

10%

20%

30%

40%

50%

60%


Who They Talk with Regarding Pain

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

Su

rvey

49

Reaction of Doctor/ Nurse/ Family Member

Patients Parents What kind of reaction did they have?

(when talking with them about pain)

20 10 Empathetic/ Supportive/ Do Things to Help

9 1 No Reaction/ Not Concerned4 0 “Get over it”/ Thought I was Complaining4 0 “Take Tylenol/ Aleve/ Ibuprofen”5 1 Don't understand but try to help2 0 Normal/ Appropriate (asked proper queues)3 1 Sent for testing0 3 Said it was normal/ expected


Web

Su

rvey

The most frequently mentioned reaction of doctors/ nurses/ family members when discussing pain with a thalassemia patient or parent is empathetic/ supportive.

50

How Patients and Parents Address Pain

Patients

Parents How do you manage pain?

26 7 Take Tylenol/ Aleve/ Ibuprofen10 1 Rest11 1 Deal with it/ Ignore It8 0 Yoga/ Exercise3 0 Ice/ heat3 0 Physical Therapy2 0 Deep Breathing2 0 Injections (cortizone)2 0 Chiropractor0 2 Antiseptic cream (before draws/transfusions)


Web

Su

rvey

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.

About Clinical TrialsTo identify awareness and interest in

clinical trials, reasons why patients have participated or would participate

or have not/ would not if given the opportunity.

52

Unaided Awareness of Clinical Trials

Patients

Parents Clinical Trial

33 16 Gene therapy15 0 Stem cell13 0 Chelation12 1 Exjade5 0 Ferriprox3 0 Jadenu3 0 Desferal3 0 Luspatercept0 6 Bluebird0 1 BMT



Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

Patients

Parents

Found out from…

28 5 Doctors/ hospitals12 9 CAF sites/ mailings10 0 CAF conferences3 0 Hematologists3 0 CHOP

0 3 Facebook parent groups(67 patients, 35 parents)

When asked what clinical trials related to thalassemia they are aware of, roughly half of patients and parents cited gene therapy.

Most discovered the trial(s)

via their doctor and/or CAF

53

"1" Definitely would not

"2" "3" "4" "5" Definitely would

0%

10%

20%

30%

40%

50%


Interest in Clinical Trials

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.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

54

Aided Awareness of Trials in Gene Therapy

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)

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


68%

32%

Yes No

66%

34%

Yes No

55



0%

10%

20%

30%

40%

50%

60%


Interest in Gene Therapy TrialUsing 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.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

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.

56

Why Participate in Gene Therapy

Patients

Parents

Reason to participate in gene therapy trial

23 7 Potential cure10 7 Reduce or eliminate transfusions6 5 Study currently looks promising3 0 Better than transplant options2 0 Help future generations1 2 Better health/ life expectancy

0 9 Less invasive than alternative treatments (like bone marrow transplants)

0 4 Improvement in quality of life(68 patients, 37 parents)


Web

Su

rvey

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.

57

Why Not Participate in Gene Therapy

Patients Parents Reason to not participate in gene

therapy trial

11 2 Risks7 0 Reduction in current health status5 1 Age (too young or old to participate)

4 3 Skepticism about trial/ long term effects unknown

3 2 Need more information/ insufficient data2 0 Invasiveness2 0 Fear of activating other disease genes2 0 Time2 0 Not in dire need (health is good now)1 1 Chemo


Web

Su

rvey

Those with reservations were concerned about the risk involved, impact on overall health, and lack of data regarding long term effects, etc.

58

Aided Awareness of Luspatercept Trials

Most patients and parents indicated that they were not aware of the ongoing clinical trials involving treatment of thalassemia with the protein Luspatercept.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


17%

83%

Yes No

9%

91%

Yes No

59



0%

10%

20%

30%

40%

50%


Interest in Luspatercept Trial

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.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

60

0%10%20%30%40%50%60%70%80%90%


Participation in Clinical Trials

Web

Su

rvey

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.

Number of clinical trials

% of patients

None 37%

One trial 25%

2 to 3 trials 15%

4+ trials 13%

DK/ “many” 10%

61

Participation in Clinical Trials

Patients

Parents Clinical Trial

13 4 Exjade7 2 SQUID6 1 MRI T26 0 Desferel



Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

Other trials and studies cited by one or two parents included ferriscan, MRI pituitary iron measurement, MRI T2* (EDICT), zinc, desferal with Exjade

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.

62

Considerations About Trials

Patients Parents Information Desired 37 22 Risk/ Safety vs Benefit19 8 Convenience - Impact on Work/ Family Time16 11 Side effects/ Impact on Life15 0 invasiveness/ Needles14 4 Time/ Length9 4 Importance in Advancement9 9 Benefit8 9 Location7 0 Complicated Study Process / Ease of Participation6 6 Trial History/ Phase (success rate)5 3 Trial Sponsor/ Reputation5 3 Long term effects5 0 Eligibility/ Opportunity4 0 Hospitalization4 0 Compensation


Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


63

Most and Least Attractive to Patients

Most Attractive Least Attractive

Patients Detractors

16 Risks

14 Difficult treatment plan (# of labs, etc)

12 Side effects9 Time consuming6 Criteria (not qualifying)5 Invasiveness5 Not well tested (too early in trial

phase)3 Chemo-like3 Lack of clear data patients can

understand

Patients Attractors

17 Potential for a cure15 Improved compliance (via better

ways/tasting medicine/treatments)11 Eliminating or reducing transfusions5 Better quality of life4 Better treatment options

Number of mentions cited below

Pati

en

t G

rou

ps

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.

Least appealing are the risks.

64

Most and Least Attractive to Parents

Most Attractive Least Attractive

Parents Detractors

3 Minimum age requirement too high to qualify

3 Chemo-like3 Travel3 Too early in trial phase3 Side effects2 Restrictions on qualifying for trial2 Invasive

1 Length of time from trial phase to market

Parents Attractors

12 Potential cure7 Reduces or eliminates need for

transfusions4 Long term benefit3 Current trials having good results2 Free medical care/treatment during

trial

Number of mentions cited below

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.

Pare

nt

Gro

up

s

Least appealing are the risks, chemo-like, travel, too early in trial phase and side effects.

65

Conditions Under Which Most Likely to Enter a Clinical Trial

Patients

Parents

Under what conditions would you be most likely to enter a clinical trial for thalassemia?

22 15 Little or no risk

16 12 Personal or overall benefit to patients/ known benefit to child

7 0 Time involved7 2 Convenience5 3 Full understanding of study details and it’s safety5 0 Non-invasive4 0 Compensation0 5 Child is not doing well on current medical protocol0 3 Improves child’s quality of life0 3 Potential cure0 2 Child has a life-threatening illness0 2 Limited side effects


Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

66

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.

To advance treatment options/ potential cure (18) “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

Reason(s) They Entered a Clinical Trial

Web

Su

rvey

67

To advance treatment options/ potential cure (continued...) “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

Su

rvey


68

Minimal risk/ negative impact (8) “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

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

Su

rvey


69

Conditions Under Which They Would Not Enter a Clinical Trial

Patients

Parents

Under what conditions would you definitely not enter a clinical trial for thalassemia?

31 28 Risk(s) too great/side effects13 0 Negative impact on health12 0 Hospital stay required10 4 Too much time involved6 4 If it’s invasive/ too invasive5 3 Too early in the trial5 4 Requires travel/ too much travel5 0 Being performed by a Pharmaceutical company4 0 Lack of information regarding study3 0 Experimental in nature



Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

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

70

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.

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

Reason(s) They Declined a Clinical Trial

Web

Su

rvey

71

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

Dislike one or more elements of treatment (5) “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

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

Web

Su

rvey

Reason(s) They Declined a Clinical Trial

Cooley’s Anemia Foundation - Awareness,

Perceptions & NeedsTo obtain perceptions of CAF, likes, dislikes, and

what is or would be most valuable from CAF.

73

"1" No awareness

"2" "3" "4" "5" Very familiar0%

10%

20%

30%

40%

50%

60%


Awareness/ Familiarity with CAF Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

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.

74

Patients Reason(s) for Contacting CAF

Web

Su

rvey

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..

75

Parents Reason(s) for Contacting CAF

Web

Su

rvey

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.

76

Involvement with CAF

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.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


41%

14%

44%

More Less Same

48%

24%

27%

More Less Same

77

Some respondents cited a chance in circumstance as the reason why they are more or less involved with CAF today.

“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

Comments About Involvement with CAF P

ati

en

t G

rou

ps

Pare

nt

Gro

up

s

78

Overall Impression of CAF


69%

21%"5" Excellent "4"

"3" "2"

"1" Poor 86%

11%

Web

Su

rvey

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%

Average rating = 4.6 Average rating = 4.8

79

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.

CAF resources/ knowledge/ information (22) “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

Reason(s) for Favorable Views of CAF

Web

Su

rvey

80

CAF resources/ knowledge (continued...) “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

Su

rvey

81

Dedication/ commitment/ personal interest (16) “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

Su

rvey

82

Exceptional staff (12) “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

Su

rvey

83

“...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.”

As one parent put it...

Web

Su

rvey

84

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

Development or enablement of support networks (8) “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) “They are an unbelievable support system to their patients.” -

Patient


Web

Su

rvey

85

Very responsive/ assessable/ easy to reach (6) “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

Su

rvey

86

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.

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

Value Most from CAF Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

87

Resources/ information/ being kept up to date (22) “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


en

t G

rou

ps

Pare

nt

Gro

up

s

88

Advocacy/ advancing the cause (12) “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

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


en

t G

rou

ps

Pare

nt

Gro

up

s

89

Ability to network with other patients/ parents (6) “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

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


en

t G

rou

ps

Pare

nt

Gro

up

s

90

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.

Integration of TAG members (2) “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

Reason(s) for Rating CAF a “3”

Web

Su

rvey

91

A handful of other isolated comments included...

“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

Reason(s) for Rating CAF a “3”

Web

Su

rvey

92

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.

CAF presence/ conference in NE/ not across U.S. (10) “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

Value Least from CAF Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

93

CAF presence/ conference in NE (continued...) “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

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

Value Least from CAF Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

94

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

What Would Be Even More Valuable P

ati

en

t G

rou

ps

Pare

nt

Gro

up

s

95

More networking opportunities/ a forum for patients/ parents (continued…) “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

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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

96

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

Training/ education/ network for hematologists/ medical community (7) “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

What Would Be Even More Valuable

97

Information/ support on clinical trials (5) “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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

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

“...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

98


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

Information on alternative treatments (continued...) “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

99

Expanded/ more patient resources (5) “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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

100

Suggestions for CAF

Sampling of suggestions to help even more…

Help organize more local chapters.

Ensure thal centers are in-sync.

Develop a thal patient registry.

Reach out to parents more.

Keep up the monthly email updatesConduct a study demonstrating the importance of new/fresh blood.

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.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

“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

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

A mobile

app

101

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.

CAF's Patient Outreach Director

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

36%

64%

Yes No

38%

62%

Patients (67)

Parents (35)

102

Awareness of CAF Social Worker

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.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


60%

40%

Yes No

45%55%

Yes No

103

Awareness of CAF Case Manager

Most patients and parents indicated that they were aware that CAF has an on staff Case Manager.

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s


38%

62%

Yes No

45%55%

Yes No

104

Additional comments or suggestions offered by patients and parents included...

“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

Other Comments/ Ideas from Respondents P

ati

en

t G

rou

ps

Pare

nt

Gro

up

s

105

“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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

106

“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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

107

“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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

108

“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


ati

en

t G

rou

ps

Pare

nt

Gro

up

s

109

Participant Information

Profile data and group composition

Type of Thalassemia


69%

22%

9% Beta thalassemia major

Beta thalassemia inter-media

Alpha thalassemia major

Hemoglobin H Disease

E beta thalassemia

87%

8%

Web

Su

rvey

3%3%

111

Gender of Patient


31%

69%

Male Female

51%49%

Web

Su

rvey

112

Caucasian Asian South Asian Other (Hispanic, African American,

etc.)-10%

0%10%20%30%40%50%60%

Patients (67) Parents - child is… (35)

Ethnicity

113

Pati

en

t G

rou

ps

Pare

nt

Gro

up

s

55%

6%

39%Italian

Greek

Non-Italian/ Non-Greek

1 to 3 4 to 6 7 to 13 14 to 20 21 to 29 30 to 39 40 to 49 50+0%

10%

20%

30%

40%


Age of Patient

Web

Su

rvey

114

Region


49%

12%

16%

24% Northeast

Midwest

South

West

Web

Su

rvey

11%

35%

115

46%

16%

22%

16%

Patient Group Participants (3/21)

Age EthnicityGend

erTransfusions

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 Every 2 or 3

weeks W Irvine, CA

22 Asian American M Every 3 weeks NE Cambridge, MA

30 Asian American 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

Pati

en

t G

rou

ps

116


Age EthnicityGend

erTransfusions

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

Pati

en

t G

rou

ps

117


Age EthnicityGend

erTransfusions

Region

City, State

28 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

Pati

en

t G

rou

ps

118


Age EthnicityGend

erTransfusions

Region

City, State

35 Asian American F Every 3 weeks W Pleasonton, CA


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

Pati

en

t G

rou

ps

119


Age EthnicityGend

erTransfusions

Region

City, State


61 Caucasian MNo (visits doctor 2 to 3 times per

year)W Austin, TX

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 Every 2 to 3 weeks NE Bronx, NY

26 Asian American F Every 3 weeks MW Minneapolis, MN

19 Caucasian F Every 3 weeks NE Buffalo, NY

Pati

en

t G

rou

ps

120


Age EthnicityGend

erTransfusions

Region

City, State


55 Caucasian F Every 3 weeks W Costa Mesa, CA

30 South Asian American M Used to - every 4

weeks S Houston, TX

42 Caucasian (Greek) F Every 4 weeks NE Mineola, NY

56 Caucasian F None, visit doctor once/6 months S Tampa, FL

35 Asian American 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

Pati

en

t G

rou

ps

121


Age EthnicityGend

erTransfusions

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

26 South Asian American F Three times per

year MW Wichita, KS

24 South Asian American F Every 3 weeks NE Newark, DE

Pati

en

t G

rou

ps

122


Age EthnicityGend

erTransfusions

Region

City, State

41 Caucasian F Every 2 weeks NE East Rockaway, NY

39 South Asian American F Every 2 weeks NE Long Island, NY

20 Asian American F Every 3 weeks W Redondo Beach,

CA

26 African american M Every 3 to 4

weeks MW Cassopolis, MI

28 Asian American M Every 3 weeks NE Hicksville, NY

27 Asian American M Every 3 weeks NE Hicksville, NY

57 Caucasian F Every 3 weeks NE Brooklyn, NY

Pati

en

t G

rou

ps

123


Age EthnicityGend

erTransfusions

Region

City, State

30 Caucasian F Every 2 weeks NE Homdel, NJ

45 Caucasian F Every 3 to 4 weeks MW Glenview, IL

36 Asian American F Every 3 weeks W Eagle River, AK

33 Asian American M Every 3 weeks W Danville, CA

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

Pati

en

t G

rou

ps

124

Parent Group Participants (3/28)

Age EthnicityGend

erTransfusions

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

Daughter is 14

South Asian American F Every 2 weeks MW Lexington, MO

Son is 14 Middle Eastern F Every 3 weeks S Mooresville, NC

Daughter is 5 Caucasian M Every 3 to 4

weeks MW Decatur, IL

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

Pare

nt

Gro

up

s

125


Age EthnicityGend

erTransfusions

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

Asian American F Every 4 weeks S Dacula, GA

Daughter is 15

Asian American M Every 4 weeks S Dacula, GA

Daughter is 5 Caucasian F Every 3 weeks NE Ambler, PA

Son is 3 Caucasian M Every 4 weeks NE Rochester, NY

Pare

nt

Gro

up

s

126


AgeEthnicit

yGend

erTransfusion

sRegio

nCity, State

Daughter is 4.5 Caucasian F Every 3 to 4

weeks NE Sayreville, NJ

Sons are 16 Asian American F Every 4 weeks NE Dover, NH

Daughters are 3 and 7

Asian American F Every 3 weeks NE Grove City, PA

Daughters are 3 and 7

Asian American M Every 3 weeks NE Grove City, PA

Daughter is 5 Asian American F Every 4 weeks W Farmington,

UT

Sons are 3 and 5

South Asian

AmericanF Every 4 weeks S Orlando, FL

Daughter is 4.5

Asian American F Every 3 to 4

weeks NE Richmond, VA

Pare

nt

Gro

up

s

127


AgeEthnicit

yGend

erTransfusions

Region

City, State

Daughter is 10

Asian American F Every 3 weeks MW Decatur, IL

Daughters are 7 and

11

Asian American F Every 3 weeks W Madera, CA

Daughter is 9

Asian American F Once/ year MW Salem, WI

Son is 8 Asian American F Every 3 weeks MW Sapulpa, OK

Daughters are 5.5 and 7

Asian American F Every 3 weeks NE Troy, PA

Daughter is 4 Caucasian M Every 3 weeks NE Sayerville, NJ

Son is 10 Asian American F Every 3 weeks NE Flushing, NY

Pare

nt

Gro

up

s

128


AgeEthnicit

yGend

erTransfusions

Region

City, State

Daughter is 22

monthsCaucasian F Every 4 weeks S Charlotte, TN

Sons are 18 and 28 Caucasian F Every 3 to 4

weeks S Suwanee, GA

Sons are 18 and 28 Caucasian M Every 3 to 4

weeks S Suwanee, GA

Son is 2 Caucasian M Every 4 weeks NE Lodi, NJ

Daughter is 2

Asian American F Every 3 to 4

weeks NE Floyd, VA

Daughter is 13

Asian American F Every 3 weeks W San Antonio, TX

Pare

nt

Gro

up

s

129

prepared by: 2015 patient and caregiver study full report may 2015

Documents