POPULATION RESEARCH SEMINAR SERIES
Sponsored by the Statistics and Survey Methods Core of the U54
Partnership
Child-Reported Outcomes in
Pediatric Advanced Cancer: Are They Helpful?
Joanne Wolfe, MD, MPH
Division Chief, Pediatric Palliative Care
Department of Psychosocial Oncology and Palliative Care
Dana-Farber Cancer Institute
Director, Pediatric Palliative Care
Boston Children’s Hospital
POPULATION RESEARCH SEMINAR SERIES
Sponsored by the Statistics and Survey Methods Core of the U54
Partnership
Questions? Comments?
Send us an email!
u54.ssmc@gmail.com
This study is dedicated to Dr. Jane C. Weeks who
served as my primary research mentor. Her
wisdom and insightfulness will have a lasting
impact on numerous investigators, patients and
families for many generations to come.
Questions
1. Are children capable of reporting on their own
symptoms and quality of life?
2. How do children’s reports vary with developmental
stage?
3. What are the effects of feeding back children’s
reports to clinicians and parents in children with
advanced cancer? Results of the PediQUEST
Randomized Controlled Trial
Background
• Thousands of children are living with advanced cancer,
1400 die annually. (ACS 2012)
• Several studies among adults with advanced cancer
suggest that presenting providers and patients/families with
PROs may improve patient’s HRQoL (Velikova et al JCO 2004, Berry et al JCO 2011)
• One study evaluating a web-based PRO feedback
intervention (KLIK PROfile) in children with rheumatological
illness using a sequential cohort design. (Engelen V et al, Patient Educ Couns 2010)
Pediatric Palliative Care
Current Research Focus
Early Research Focus
Hope for cure, life extension, a miracle…
Individualized blending of care directed
at underlying illness
and
physical, emotional, social, and spiritual
End-oflife care
needs of child and family
with continuous reevaluation and adjustment
Hope for comfort, meaning…
Bereavement
care
Examples of Earlier Studies
Percent of Children
100
Symptom Prevalence
Suffering
Timing of Understanding That Child Had
No Realistic Chance for Cure
80
60
40
Duration of disease - 32 months
Physician - 7months
Parent - 3.5 months
20
0
Wolfe et al. NEJM 2000.
Wolfe et al JCO 2008
Wolfe et al. JAMA 2000
PediQUEST Study
Pediatric Quality of Life and Evaluation of Symptoms Technology:
Computer-based data collection system that collects child (or
parent) reported symptoms and QoL and has the ability of
generating printed feedback reports and email alerts.
The PediQUEST Study
Hypothesis
Increasing awareness of providers and parents about
child’s symptoms and quality of life will decrease the
child’s experience of suffering
The PediQUEST Study
Study Goals
1.
To assess the feasibility of performing a randomized
controlled supportive care study in a population of
children with advanced cancer using child-reported
outcomes (Feasibility Study)
2.
To describe child-reported symptom distress and quality
of life in pediatric advanced cancer (Descriptive Study)
3.
Preliminarily assess if routine feedback of symptom and
QoL data to providers and families has any effect on
child’s symptoms and QOL (Evaluation Study)
Enrollment
Baseline measurement
Individual Randomization
(block randomization by site)
Control
Intervention
= control +
PedsQL, MSAS and FPS-R
At least once a month
at most once a week
X 3 Months (w/re-enrollment)
Descriptive Study
+ @-mails
Satisfaction assessed
at 4th and 8th PQ survey
The PediQUEST Study
Study Population
• Inclusion criteria
– Children  2 years old
–  2 weeks of progressive, recurrent, or non-responsive cancer or
decision to not pursue cancer-directed therapy
– 1 parent with command of English and able to complete paper and
pencil and/or computerized questionnaires
• Overall Follow-up
– Individual patient follow-up: every 3 month reenrollment, or until death
• Target Sample Size
– 120 consecutive patients (between the 3 study sites BCH, CHOP,
Seattle Children’s)
The PediQUEST Study
Methods
Study Instruments
•
PediQUEST
(children or parents)
• At most once a week
• Clinic, inpatient ward, home (by phone)
•
Survey about Caring for Children with Cancer-SCCC
(Parents and physicians)
• 0-3-6-9 months
• Post-death Survey
•
Satisfaction Surveys
(children, parents and physicians)
PediQUEST Instruments
• MSAS
– Adult oncology symptom assessment tool adapted for use in children
(Collins et al, JPSM 2000, 2002)
– MSAS 7-12, 10-18 and nurse proxy version adapted to PQ-MSAS Proxy
full, PQ-MSAS 7-12) and PQ-MSAS proxy-supplemental, PQ-MSAS 13-18
• PedsQL 4.0 TM
– Standardized patient self-report and parent-report instrument designed to
systematically assess pediatric patient’s HRQoL outcomes.
(Varni et al Med Care, 2001)
– Consists of a 22-item core measure of HRQoL
– Four proxy and three self-report age-adapted versions are available (age
groups are 2-4, proxy only; 5-7, 8-12 and 13 and over, each with a selfreport and proxy version available)
• Sickness Question
– Overall, how have you/your child been feeling during the past week?
– Two response scales: 3 point faces scale (5-6 yo), VAS (7 and above), “not
sick at all”, “very sick”
Instruments, response options, age group
and respondent
Baseline PQ survey
Control arm
completed PediQUEST surveys at
clinic, hospital or home
Feedback intervention
same as control +
printed reports to providers and
families + emails when thresholds
reached
Feedback report
Feedback report
Feedback report
Results
1. Are children capable of reporting
on their own symptoms and quality of
life?
Willingness to complete
PediQUEST
• Total PediQUEST surveys
administered: 1126
• Surveys answered by children: 96%
(of 965 potential child administrations)
Proportion of Child Response
by Age
Child Response by age group
100%
100
29
14
4
0.5
80%
60%
Parent
Child/Teen
40%
20%
0%
2-4yo
5-6yo
7 yo
8-12 yo 13 yo and
above
Self-reported symptoms 20 weeks of follow up
Prevalence and proportion with moderate to severe distress
(n=704 surveys)
0.0%
10.0%
20.0%
30.0%
Pain
Fatigue
Drowsy
Irritability
Nausea
Anorexia
Sleep
Diarrhea
Vomiting
Nervousness
Sadness
Cough
Worrying
Concentration
Itching
Skin issues
Constipation
Dyspnea
Image
Dry mouth
Numbness
Sweating
Dysphagia
Dysuria
Mod-Sev distress
40.0%
50.0%
60.0%
PediQUEST Symptom Burden
during the first twenty weeks
of follow-up
High symptom burden
• 88% of children experienced at least one episode of
moderate to high distress in 20 weeks of follow-up
(regardless of study arm).
• On average, patients reported moderate to high distress
from approximately 4.2 symptoms
2. How do children’s reports vary with
developmental stage?
Consistency increases with age
• MSAS and QoL scores correlation increases with age
Child 7-12 yo
MSAS, mean score (SD)
PedsQL, mean score(SD)
Spearman correlation
13.1 (13.5)
75.2 (15)
-0.35
Teens >=13 yo
10.7 (9.02)
71.9 (15.1)
-0.59
Consistency increases with age
• Comparison of similar MSAS and QoL items further
supports hypothesis
Correlation between MSAS and PedsQL comparable items
Child 7-12 yo
Teens >=13 yo
Spearman Correlation
Spearman Correlation
Symptom
Pain
Fatigue
Sadness
Worry
Sleep Problems
-0.51
-0.20
-0.36
-0.37
-0.57
-0.71
-0.71
-0.57
-0.45
-0.76
Differences in child report by age
• Teens report higher prevalence on all (comparable)
symptoms assessed
Symptom Prevalence (%)
60.00
50.00
40.00
30.00
7-12 yo
20.00
13 yo and up
10.00
0.00
8 MSAS Symptoms
Differences in child report by age
• Teens report higher scores (>distress) on all (comparable)
symptoms assessed*
30
MSAS Symptom Score
25
20
15
7-12 yo
10
13 yo and up
5
0
*scales would favor higher scores among
younger children
8 MSAS Symptoms
3. Effects of feeding back children’s
reports to clinicians and parents in
children with advanced cancer:
Results of the PediQUEST
Randomized Controlled Trial
Satisfaction with Feedback
Satisfaction with Feedback
Intervention Effect
• Main Outcomes:
– MSAS total scores and subscores
– PedsQL total scores and subscores
• Covariates:
– Time in study
• Analysis strategy:
– Multivariate linear mixed effects models (to account for having repeated
measures on the same subjects, i.e. observations that are correlated).
Strengths and Limitations
Strengths
• Prospective – longitudinal PRO data
Limitations
• Validity of tool scores/measurement tools
• Selection bias (relatively healthy cohort)
PediQUEST Conclusions
• Child-reported outcomes are feasible
• RCT supportive care trial is feasible in
children with advanced cancer
• Children with advanced cancer
experience many distressing
symptoms
• Feedback alone has some effect on
child suffering but can be strengthened
Question
Why was feedback alone insufficient to
significantly improve symptom distress
and quality of life outcomes?
Possible Explanations
• PediQUEST feedback had a limited effect in
activating physicians to attend to symptom
distress, as suggested by satisfaction
results
• Physician attitudes/beliefs may play a strong
role in symptom assessment and
management
Next Steps
Next Steps – PediQUEST
Champion Intervention
• Hypothesis: compared to an enhanced feedback system
alone, combining feedback with intensive follow-up by an
pediatric nurse practitioner champion will more effectively
reduce child symptom distress.
• Rationale: embedding symptom management support
within oncology will –
– provide more targeted and timely telephone and/or face-to-face
response to a child’s distress experience.
Web platform
PediQUEST: A Peppercorn Collaboration
DFCI/CHB Team
Veronica Dussel, MD,MPH, Christina Ullrich, MD, MPH, Bridget
Neville, MPH, Kun Chen PhD, Liliana Orellana PhD
CHOP Team
Karen Carroll, Tammy Kang MD, Chris Feudtner MD PhD MPH
Seattle Team
Abby Rosenberg, MD, Karina Schmidt, Russ Geyer MD MPH,
DFHCC Collaborators
Fran Cook ScD, Jane Weeks MD MSc
Collaborating Services
DFCI Research Information Services
Communication Core DFHCC
Thank you!