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The PediQUEST Study: Evaluation of Pediatric Quality of Life and Evaluation of Symptoms Technology

2 Years
25 Years
Not Enrolling
Pediatric Neoplasm, Quality of Life, Outcome Assessment, Palliative Care

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Trial Information

The PediQUEST Study: Evaluation of Pediatric Quality of Life and Evaluation of Symptoms Technology

Introduction: Cancer is the leading cause of nonaccidental death in childhood.(Arias et al.
2003) In spite of significant scientific progress, one quarter of children with cancer still
do not survive(Greenlee et al. 2000), and over 50% experience substantial suffering that
could be alleviated.(Wolfe et al. 2000a) Pediatric palliative care is now an expected
standard of care and research in this field has been established as a national priority by
the Institute of Medicine (IOM).(Field et al. 2003) Earlier work by our group suggested that
when parents and physicians recognized at an early stage that a child had no realistic
chance of cure, they were more likely to integrate elements of effective palliation into the
child's care. However, this early stage concordance about prognosis was rare.(Wolfe et al.
2000b) Communication and delivery of optimal care in the advanced stages of illness is
especially challenging in children.(Shelton 1999) The anticipation of losing a child and
physicians' own difficulties(Saunders 1979-80; Hilden et al. 2001) may hinder discussions
about poor prognosis and impact on decision-making and integration of palliative care. Thus
attention to symptom management and quality of life (QoL) in children with advanced cancer
may be limited.

The collection of standardized symptom and QoL data has shown to enhance physician's
recognition, knowledge and accuracy in the assessment and palliation of symptoms, and to
increase patient-provider discussions about QoL issues and directly improve patient's
QoL.(Detmar et al. 2002; Taenzer et al. 2000; Velikova et al. 2004) Whether these findings
are applicable to children remains to be established.

One of the obstacles to routine symptom and QoL assessment in children is the need of
developmentally adapted(Eiser et al. 2001b; Eiser et al. 2001c; Hain 1997) and parent-proxy
tools(Eiser et al. 2001a), making the paper and pencil format extremely impractical.
Computerized surveys offered a potential solution. Patient acceptance of such devices has
been found to be high.(Buxton et al. 1998; Velikova et al. 2002; Velikova et al. 1999;
Velikova et al. 2001) Our current project -the PediQUEST Study- consists of a pilot for a
randomized controlled trial (RCT) aimed at assessing the effect of routinely reporting
symptom and QoL data. We will use an innovative data collection strategy: a hand-held
computer devise, the Pediatric Quality of Life and Evaluation of Symptoms Technology
(PediQUEST) that readily administers a user-appropriate survey instrument and generates
reports summarizing how the patient feels.

As far as we are aware of, this will be the first RCT conducted in children with advanced
cancer to evaluate a palliative care intervention. Given the challenges with doing research
in this population, we propose to simultaneously focus on several goals.

Aims: Aim 1: (Feasibility study) Assess the feasibility of conducting a randomized
controlled supportive care trial (RCT) in children with advanced cancer. Aim 2: (PediQUEST
Evaluation) Preliminarily assess the effect of routinely feeding back QoL and symptom data
to families and primary oncologists, and alerting supportive care teams about highly
distressing symptoms. Aim 3: (Descriptive study) Prospectively explore determinants of
child's suffering and parental-physician discordance regarding prognosis and treatment goals
in children with advanced cancer.

Design: Aim 1: Cohort survey study; Aim 2: Pilot prospective RCT; Aim 3: Cohort study
embedded in an RCT.

Setting: Three large US pediatric cancer centers. Subjects: 104 children 2-years old and
above with advanced cancer and their parents were enrolled between December 2004 and June

Follow-up: Patients were initially followed up for 9 months or until death whatever occurred
first. However, an interim analysis of the first 29 patients showed that 75% were alive at
nine months, suggesting the enrollment of a healthier cohort. We therefore changed the
follow-up period to 3 months with the option of multiple re-enrollments until the end of
data collection (December 2009) or death.


Feasibility study: All subjects approached for enrollment were invited to complete a
one-time paper and pencil "Consent survey" regardless of their decision about whether to
participate. All enrolled subjects were asked to complete a paper and pencil "Participation
survey" after completing the study or dropping-out in order to explore determinants of

PediQUEST Evaluation and Descriptive studies: Symptom and QOL scores were tracked up to once
a week. Surveys were embedded in the PediQUEST system and administered through tablet

Participants' recruitment: Eligible children were identified through daily review of clinic
roster with clinic staff including nurses, oncologists and/or nurse practitioners and
verified with the patient's primary oncologist using a checklist sent over email. If after
being introduced to the study and having had the opportunity to ask questions, child and
parent(s) were willing to participate, parent(s) were asked to review and sign the informed
permission/assent document covering the parent(s) and child's participation. A provider's
completion of the eligibility checklist will indicate informed consent of providers.

Study Instruments:

1. PediQUEST survey (PQ-Survey): assessed symptoms, quality of life, and overall sickness.
Nine survey versions were developed to allow for consistent measurement across the age
ranges and respondents. Self-report started at 5 years of age; children aged 5 to 12
years answered shorter, age-adapted questionnaires which were complemented with
additional parental questionnaires; teens answered a full self-report questionnaire.
Parents of children between 2 and 4 years old answered a full survey on their child's
behalf. If a child did not feel well, parents could complete the full questionnaire on
their behalf. PQ-Surveys were pilot tested in 15 patients showing good acceptability
and comprehensibility. PQ-surveys used four tools: 1) PQ-Memorial Symptom Assessment
Scale (MSAS), adapted from previously validated child(Collins et al. 2000; Collins et
al. 2002) and proxy(Drake et al. 2003) MSAS, measures frequency, severity and distress
for 24 symptoms; 2) Pediatric Quality of Life Inventory (PedsQL), a 22-item health
related quality of life instrument;(Seid et al. 1999; Varni et al. 2002; Varni et al.
2003) 3) Faces Pain Scale Revised (a faces pain scale, used to measure current pain
intensity);(Hicks et al. 2001) 4) Sickness VAS scale (overall sickness measure created
ad hoc).

2. Survey about Caring for Children with Cancer (SCCC): In addition, parent and physician
perspectives regarding prognosis and treatment goals were collected at baseline and
then every 3 months using a paper-and-pencil survey SCCC, adapted from our survey for
bereaved parents.(Wolfe et al. 2000b)

3. Post death survey: If patients died within the study period parents were invited to
complete a post-death survey at least four months following the death to assess
distress during the last month of life and retrospective perspectives regarding the
overall quality of care.

4. Satisfaction Surveys: Child and parent surveys were adapted from existing
questionnaires.(Grogan et al. 1995; McCusker 1984; Weaver et al. 1993) Satisfaction
with PQ technology was assessed using seven items for children eight years old and
above and 11 items for parents. Surveys were embedded in the PediQUEST system and
administered at fourth and eighth PediQUEST administration. A survey evaluating
participant's satisfaction with the PediQUEST intervention (see below) was administered
concurrently (eight items (parents), five items (children eight years old and above))
to all intervention participants. Provider satisfaction with the PQ intervention, and
perceived usefulness of the PQ-system was assessed through an online survey
administered at the end of data collection.

Intervention: Patients were randomly allocated to receive routine feedback about symptoms
and QOL. The feedback intervention had two components: (i) PQ-reports: printed reports were
made available to providers (inserted in charts before the visit) and families (handed
immediately after survey completion at clinic or ward). PQ-reports consisted of bar plots of
PedsQL and MSAS symptom scores from current and four prior administrations, a summary
highlighting changes since last report, and a list of available resources for symptom
control (for families), or generic pain management recommendations (for providers). Training
on how to interpret PQ-reports was offered to families at enrollment and on an annual basis
to providers; (ii) PQ emails: were automatically generated if PQ-MSAS individual symptom
scores were ≥70, or if on two consecutive administrations the patient or parent reported a
score of ≥70 for any frequency, severity or distress item, or a PedsQL total score ≤ 40.
E-mails were sent to primary providers (oncologist, nurse, and psychosocial clinician), the
local palliative care service, and, when pain was reported, the pain service (or
equivalent). No instructions were imparted on how to respond to emails. Families and
providers of patients in the control arm of the study did not receive feedback reports. The
allocation process was embedded in PediQUEST and blinded to investigators until the patient
completed the first survey. Because of the nature of the intervention this was an open label
study. As a token of our appreciation, small non-monetary incentives were provided to
children, parents, and providers.

Outcomes: This study explores the feasibility of conducting an RCT assessing a supportive
care intervention in children with advanced cancer (aim 1). It also provides estimates of
consent and attrition rates, its determinants, and the burden associated with repeated
measurements. In addition, this pilot study will preliminarily assess the impact of feedback
on child's distress and quality of life. Main outcomes of this efficacy study (aim 2) are
presented below. This pilot study will render sufficient data for accurate sample size
calculation for future studies and serve to explore determinants of suffering in children
with advanced cancer (aim 3).

Statistical Analysis:

PediQUEST Evaluation (Aim 2): To assess the effect of PediQUEST, the proportion of patients
with unrelieved symptoms in each arm will be compared using odds ratios and associated
chi-square tests. To assess the progression of reported levels of child's distress and QOL
trends, we will run a mixed effects model that assumes a linear trend in the outcome over 20
weeks of follow up for each subject. The model will examine the difference in the average
trend over time (slope) between the intervention and control group by including a fixed
effect term that represents the interaction of treatment status and time since study entry.
Lack of difference between slope coefficients will support the null hypothesis of no effect
by the intervention. Single point secondary outcomes will be analyzed by comparing both arms
with a t test or Wilcoxon rank-sum test for continuous outcomes and odds ratios and
associated chi-square tests for proportions.

Sample Size and Power Calculations A formal sample size estimation was not possible given
the lack of data on distribution of scores in an advanced cancer population or their
variation over time. For this reason we originally proposed to recruit 120 patients and
their parents, a sample size driven by practical rather than statistical considerations that
would serve as a basis for future sample size calculations. We estimated that 60 subjects
per arm would allow for an adequate balance between arms and likely provide sufficient power
to detect large clinical effects for single point measurements.

Inclusion Criteria:

- Children >=2-years old with at least a 2-week history of progressive, recurrent, or
non-responsive cancer of any type or decision not to pursue cancer directed therapy.

- Child's parent must have spoken and written command of English or Spanish.

- Child's parent must have the ability to understand and complete self-administered

Exclusion Criteria:

- Patients who have an isolated relapsed solid tumor treated with surgery or radiation

- Patients with hematological malignancies who have achieved remission after the first
induction attempt, whose treatment plan includes their first Stem Cell Transplant
(SCT), and have an identified donor (must meet all 3 criteria to be excluded).

- Patients who are not regular patients at one of the two participating institutions.

- Foster parents who do not have legal guardianship.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

Child Distress - unrelieved symptoms

Outcome Description:

Child distress-unrelieved symptoms will be assessed through the proportion of patients with unrelieved symptoms (any symptom/s reported as causing moderate to high distress or FPS-R score >=8 in 2 consecutive PediQUEST evaluations)

Outcome Time Frame:

20 weeks

Safety Issue:


Principal Investigator

Joanne Wolfe, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Institutional Review Board

Study ID:




Start Date:

November 2004

Completion Date:

June 2010

Related Keywords:

  • Pediatric Neoplasm
  • Quality of Life
  • Outcome Assessment
  • Palliative Care
  • Pediatric Advanced Cancer
  • Children
  • Quality of Life
  • Health Information technology
  • Patient reported outcomes
  • Neoplasms



Dana Farber Cancer Institute Boston, Massachusetts  02115
Seattle Children's Hospital Seattle, Washington  98105
Boston Children's Hospital Boston, Massachusetts  02115
Children's Hospital of Philapdelphia Philadelphia, Pennsylvania  19104