Post on 04-May-2022
1
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
2
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)
4
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
Pati
ent
Gro
ups
Pare
nt
Gro
ups
5
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)
(68 patients, 37 parents)
Number of mentions cited above
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
Surv
ey
8
Support network (peers)
Better insurance/financial support
Advances in treatment
Easier logistics (e.g., clinic hours, etc.)
Greater expertise among medical providers
9
Patients (68) Parents (37)
22%
29%25%
19%
4%
No pain at all - "1"
"2"
"3"
"4"
Very painful - "5"
49%
Web
Surv
ey
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.
10
0%
10%
20%
30%
40%
50%
60%
No One Nurse Doctor Family Member Other (spouse,friends, patients/
parents)
Patients (68) Parents (37)
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
Surv
ey
11
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)
(68 patients, 37 parents)
Number of mentions cited above
Web
Surv
ey
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
(67 patients, 35 parents)
Pati
ent
Gro
ups
Pare
nt
Gro
ups
Number of mentions cited above
14
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
(67 patients, 35 parents)
Number of mentions cited above
Pati
ent
Gro
ups
Pare
nt
Gro
ups
15
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
(67 patients, 35 parents)
Number of mentions cited above
Pati
ent
Gro
ups
Pare
nt
Gro
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
16
Patients (68) Parents (37)
69%
21%
10%"5" Excellent
"4"
"3"
"2"
"1" Poor
86%
11%
Web
Surv
ey
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
18
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
19
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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.