The PediQUEST Response Intervention Study

Background: Integration of palliative care (PC) has been associated with better health
related quality of life (HRQoL) and longer survival in adults and their caregivers. Yet, only
a few randomized controlled trials (RCTs) have evaluated whether PC integration improves
child and family outcomes. The investigators propose to evaluate the effects of an early PC
intervention (PediQUEST Response) for pediatric oncology patients (from here on called
patients) with advanced cancer on patient and parent outcomes.

Aims:

- Aim 1 To evaluate whether PediQUEST Response, compared to usual care, improves patient's
HRQoL (primary outcome) and symptom burden.

- Aim 2 To evaluate the impact of the intervention on parent psychological distress and
symptom-related stress outcomes.

- Aim 3 To compare family activation by measuring coping strategies between study entry
and 16 weeks, use of non-pharmacologic strategies for symptom treatment, and No. of
documented psychosocial clinician encounters between intervention and control arms.

Design: Multisite, randomized (1:1), controlled, un-blinded, effectiveness trial comparing
PediQUEST Response (intervention) vs usual cancer care (comparator).

Setting: Dana-Farber/Boston Children's Cancer and Blood Disorders Center (DFBCC), Seattle
Children's Hospital, Children's Hospital of Philadelphia Cancer Center, and St. Jude's
Research Hospital.

Participants: The target sample size (SS) is 136 patient-parent dyads (N=68/arm, 34/site) ≥2
years old receiving cancer care at the participating sites and with advanced cancer who have
not been referred to the palliative care team. To achieve this SS, 200 dyads will be
recruited.

Interventions:

- PediQUEST Response: consists of combining a patient-mediated activation intervention
(weekly feedback of patient-reported outcomes to families and providers using the online
PediQUEST web system or mobile App) with early integration of the PC team (consisting of
an initial meeting with providers and family with subsequent follow-up including a
monthly encounter or as needed number of encounters based on PediQUEST reports and other
clinical indications). PC teams will receive standardized training before starting the
intervention to learn how to interpret and react to PediQUEST reports and to encourage
them to build a close relationship with the patient's primary oncology team. Response
teams will also receive ongoing support throughout the intervention period through their
local PIs and an expert listserv.

- Usual Cancer Care (comparator): participants in this arm will receive usual cancer care
provided at the sites, which can include PC consultation as deemed necessary by
oncologist, and will use the PediQUEST web system to complete surveys but no reports
will be generated.

Methods: Following enrollment, patient (if older than 5 years) and parents of all enrolled
patients will receive weekly PediQUEST-Surveys including a patient HRQoL measure (PedsQL) and
a symptom burden scale (PediQUEST-MSAS). Parents will complete a Baseline Survey Packet (will
collect socio-demographic information, trait-anxiety, social support, stress, and coping
style). A two-week run-in period will identify and exclude non-responder dyads (i.e. <2
answered PediQUEST-Surveys out of 3 assigned). Responders will be randomized to the
intervention or control arms (concealed allocation) and followed up for 16-weeks (16-w).
Those assigned to the intervention will begin receiving PediQUEST Response (feedback reports
+ response team intervention). Parents in both arms will be assigned monthly questionnaires
(measuring parental distress, HRQoL, and use of complementary therapies; on weeks 8 and 16
symptom-related stress and coping will also be measured). All surveys will be administered
through PediQUEST web. Process measures will be collected throughout the study, and
semi-structured exit interviews conducted in participating patients, parents, and a
sub-sample of providers. Participants in the intervention arm will be offered continued care
by the PC team once the intervention ceases.

Outcomes: Average difference between the two treatment arms in: (i) Patient outcomes: primary
study outcome is the mean child's quality of life total score as reported by parent and
patient over 16 weeks; we will also look at quality of life subscale scores, mean patient
symptom burden scores, parent distress (anxiety, depression and symptom related stress), and
family activation (coping, and "symptom treatment activation"). See details below in outcomes
section.

Inclusion criteria:

Pediatric oncology patients (children, adolescents, or young adults), and one of their
parents, receiving routine cancer care at one of the participating centers -and not in
remission and off cancer-directed treatment—that have:

- (i) advanced cancer defined as: at least a 2-week history of progressive, recurrent,
or non-responsive cancer of any type, or any brainstem tumor, or a grade IV
Glioblastoma Multiforme, or decision not to pursue cancer-directed therapy in place,
and are

- (ii) palliative care naive.

Exclusion criteria:

The patient-parent dyad would be excluded if

- patients are older than 18 years of age and no parent is involved in his/her care, or

- if patient is under the care of foster parents who do not have legal guardianship, or

- if both parents do not speak English or Spanish, or are unable to understand and
complete surveys.