Clinical trial NCT03408314: The PediQUEST Response Intervention Study
Official title: A Multisite, Parallel, RCT Comparing the Effectiveness of an Early Palliative Care Intervention, the PediQUEST Response to Pediatric Oncology Symptom Experience , Versus Usual Cancer Care in Children and Adolescents With Advanced Cancer
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Advanced Cancer
Keywords: Advanced Cancer
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Children's Hospital of Philadelphia
name: Seattle Children's Hospital
name: St. Jude Children's Research Hospital
name: Children's Hospitals and Clinics of Minnesota
name: Nationwide Children's Hospital
name: University of Alabama at Birmingham
name: Deakin University
name: Boston Children's Hospital
name: Ann & Robert H Lurie Children's Hospital of Chicago
name: Baylor College of Medicine
name: Dana-Farber Cancer Institute
name: National Institute of Nursing Research (NINR)
First posted: 2018-01-24
Enrollment: 196
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: PediQUEST Response proposes a new system of care that expects to improve quality of life in children, adolescents, and young adults with advanced cancer and their parents. The investigators want to learn whether patients that are cared for using PediQUEST Response do in fact feel better than those receiving usual care.

National recommendations call for early palliative care (PC) integration for seriously ill children to ease suffering, however, very few randomized controlled trials (RCTs) have evaluated whether PC improves child and family outcomes. In prior work, the investigators developed the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST/PQ), a software that collects electronic Patient-Reported Outcomes (e-PROMS) and generates feedback reports.

Now, the PI and research team developed PediQUEST Response (Response to Pediatric Oncology Symptom Experience). PediQUEST Response includes an enhanced PediQUEST system (web-based and with an App that allows to answer surveys and see reports), that is coupled with early integration of a palliative care consulting team (Response team). This dual strategy will help to standardize the family report of distress, which will be done through the PediQUEST system. It will also help standardize the providers' response to such distress, as providers will be specifically trained. Pilot work for PediQUEST Response found it feasible, well received by families and oncologists, and potentially effective.

Thus, the overall goal of this study is to conduct a RCT of PQ Response versus usual care at four large pediatric oncology centers among 136 children ≥2 years old with advanced cancer. Hypotheses include a) children receiving the intervention will have better (higher) quality of life scores b) parents of children in the intervention group will report better state-anxiety, depression and symptom-related stress scores, and c) intervention group families will demonstrate higher levels of activation.

Detailed description: 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.

Eligibility criteria: 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, or or any other progressive/recurrent solid or brain tumor, and are
* (ii) palliative care naive, defined as the palliative care team not currently integrated into their regular cancer care.

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, or
* if the patient has a non-brainstem low-grade glioma with localized progression/relapse only, or
* is expected to receive a stem cell transplant within the next 18 weeks, or
* life expectancy is less than two months.

Primary outcomes:
measure: Child Quality of Life
timeFrame: 16 weeks (measured weekly from point of randomization)
Secondary outcomes:
measure: Child Quality of Life - subscales
timeFrame: 16 weeks (measured weekly from point of randomization)
measure: Child Symptom Burden
timeFrame: 16 weeks (measured weekly from point of randomization)
measure: Child Symptom Burden- subscales
timeFrame: 16 weeks (measured weekly from point of randomization)
measure: Parental distress (anxiety)
timeFrame: 16 weeks (measured every four weeks from point of randomization)
measure: Parental distress (depression)
timeFrame: 16 weeks (measured every four weeks from point of randomization)
measure: Parental distress (stress)
timeFrame: 16 weeks (measured at study entry and 16 weeks)
measure: Family Activation (coping)
timeFrame: 16 weeks (measured at study entry and 16 weeks)
measure: Family Activation (symptom treatment activation - use of complementary therapies (UCT) - number)
timeFrame: 16 weeks (measured every four weeks from point of randomization)
measure: Family Activation (symptom treatment activation - UCT - types)
timeFrame: 16 weeks (measured every four weeks from point of randomization)
measure: Family Activation (symptom treatment activation - use of psychosocial services)
timeFrame: 16 weeks (measured every four weeks from point of randomization)
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Research Institute
state: Washington
Study officials:
affiliation: Dana-Farber Cancer Institute
name: Joanne Wolfe, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Dussel V, Orellana L, Holder R, Porth R, Avery M, Wolfe J. A multisite randomized controlled trial of an early palliative care intervention in children with advanced cancer: The PediQUEST Response Study Protocol. PLoS One. 2022 Nov 8;17(11):e0277212. doi: 10.1371/journal.pone.0277212. eCollection 2022.
citation: Feifer D, Merz AF, Avery M, Tsuchiyose E, Eche-Ugwu IJ, Awofeso O, Wolfe J, Dussel V, Requena ML. Parent Views on Parent and Child-reported Outcomes in Pediatric Advanced Cancer: A Qualitative Study. J Pain Symptom Manage. 2025 Feb;69(2):e131-e138. doi: 10.1016/j.jpainsymman.2024.10.001. Epub 2024 Oct 15.
citation: Eche-Ugwu IJ, Orellana L, Becker D, Bona K, Avery M, Feudtner C, Freedman JL, Kang TI, Rosenberg AR, Waldman ED, Ullrich CK, Dussel V, Wolfe J. Household material hardship and distress among parents of children with advanced cancer: A report from the PediQUEST Response trial. Cancer. 2024 Oct 15;130(20):3540-3548. doi: 10.1002/cncr.35432. Epub 2024 Jun 12.
citation: Merz A, Feifer D, Avery M, Tsuchiyose E, Eche I, Awofeso O, Wolfe J, Dussel V, Requena ML. Patient-Reported Outcome Benefits for Children with Advanced Cancer and Parents: A Qualitative Study. J Pain Symptom Manage. 2023 Sep;66(3):e327-e334. doi: 10.1016/j.jpainsymman.2023.05.016. Epub 2023 Jun 6.