Craig Butler, National Communications Director CAF

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Craig Butler,

National Communications Director

CAF

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 examine patient views of his or her quality of life, what impacts this, and conditions under which this could be improved

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 obtain perceptions of CAF; likes, dislikes, and what is or would be most valuable from CAF

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N=102 (67 patients, 32 parents) in focus groups

(An additional 1 patient and 2 parents who could not participate in the groups filled out an online survey)

Most participants were diagnosed as beta thalassemia major.

Of patients who participated, 69% were female, 31% were male.

Current level of adherence reported on scale of 1 to 5 was 4.4 among patients, 4.8 among parents of patients (compared with 4.0 and 3.7, respectively, 5 years ago).

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Areas in Which Not Fully Adherent

Chelation

Appointments/Exams/Test

Diet/supplements

Reasons Not Adherent

Logistics/work/not enough time

Treatment is painful/has side effects

Taste of treatment

Forgot (especially when not at home)

Cost/lack of insurance

Lack of treatment in China (adopted children)

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Patients ParentsWhat 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 healthy

4 0 Positive attitude

3 0 Deaths of other patients

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

3 0 ThalPal peers

0 8 Advice from other patients’ parents

0 2 Changing how meds are given

0 2 CAF

(67 patients, 35 parents)

Number of mentions cited above

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

12 0 Oral chelation

10 2 Support (family/friends)6 5 Knowledge/better understanding

3 0 Accessibility to treatment (hours, location)

2 0 Less side effects

1 0 One day off

1 0 Better insurance

0 4Getting to the United States (adopted from foreign countries)



Web

Surv

ey

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.

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Patients – 4.1

Parents – 4.5

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.

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 there has been no change and one quarter of patents noted that it’s worse today.

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Today

(Scale 1 to 5)

5 Years

Ago

Patients (68) Parents (37)

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

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Support network (peers)

Better insurance/financial support

Advances in treatment

Easier logistics (e.g., clinic hours, etc.)

Greater expertise among medical providers

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22%

29%25%

19%

4%

No pain at all - "1"

"2"

"3"

"4"

Very painful - "5"

49%

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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.

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0%

10%

20%

30%

40%

50%

60%

No One Nurse Doctor Family Member Other (spouse,friends, patients/

parents)


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.

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Patients Parents How do you manage pain?

26 7 Take Tylenol/ Aleve/ Ibuprofen10 1 Rest

11 1 Deal with it/ Ignore It

8 0 Yoga/ Exercise

3 0 Ice/ heat

3 0 Physical Therapy

2 0 Deep Breathing

2 0 Injections (cortizone)

2 0 Chiropractor

0 2 Antiseptic cream (before draws/transfusions)



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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.

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Intention/desire to enter trial – mixed to positive responses

Interest in participating in gene therapy trial – responses were mixed. 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 4.3while 40+ patients average rating came to 3.7.

Most common reasons for interest in GT trial: potential for a cure or a reduction/elimination in transfusions.

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Patients Parents Information Desired

37 22 Risk/ Safety vs Benefit19 8 Convenience - Impact on Work/ Family Time16 11 Side effects/ Impact on Life

15 0 Invasiveness/ Needles

14 4 Time/ Length

9 4 Importance in Advancement

9 9 Benefit

8 9 Location

7 0 Complicated Study Process / Ease of Participation

6 6 Trial History/ Phase (success rate)

5 3 Trial Sponsor/ Reputation

5 3 Long term effects5 0 Eligibility/ Opportunity4 0 Hospitalization4 0 Compensation


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

22 15 Little or no risk

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

7 0 Time involved

7 2 Convenience

5 3 Full understanding of study details and it’s safety

5 0 Non-invasive

4 0 Compensation

0 5 Child is not doing well on current medical protocol

0 3 Improves child’s quality of life

0 3 Potential cure

0 2 Child has a life-threatening illness

0 2 Limited side effects



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

31 28 Risk(s) too great/side effects13 0 Negative impact on health

12 0 Hospital stay required

10 4 Too much time involved

6 4 If it’s invasive/ too invasive

5 3 Too early in the trial

5 4 Requires travel/ too much travel

5 0 Being performed by a Pharmaceutical company

4 0 Lack of information regarding study

3 0 Experimental in nature



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upsRoughly 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

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69%

21%

10%"5" Excellent

"4"

"3"

"2"

"1" Poor

86%

11%

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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

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Support/encouragement/sense of community

Resources/information/being kept up to date

Advocacy

Exceptional staff

Networking with other patients/parents

Annual conference

Research/studies/trial information

CAF presence/Conference not equally available across U.S.

Lack of patient group

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Things

Value

Least

from CAF

More networking opportunities

CAF presence/Conference in more areas of country

Training/education for hematologists

Clinical trials information

Information on alternative treatments

Even more patient resources

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Thank you to all who

participated.

This information will provide an

important guide to CAF as we

plan our future activities,

publications and projects.

Craig Butler, National Communications Director CAF

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