Advanced CancerNCT03408314
The PediQUEST Response Intervention Study
A study testing PediQUEST Response and Usual Cancer Care in people with Advanced Cancer, run by Joanne Wolfe, MD, MPH at 6 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Advanced Cancer
- What is being tested?
- PediQUEST Response and Usual Cancer Care
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 194 participants
- Where
- 6 sitesUnited States
- Study dates
- 1 Mar 2018to 14 Sep 2022
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Boston Children's Hospital
Boston, Massachusetts, 02215
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Texas Children's Hospital
Houston, Texas, 77030
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318
Seattle Children's Research Institute
Seattle, Washington, 98145-5005
About this 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
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.
Read more
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.
Read the 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.
- Conditions
- Advanced Cancer
- Being tested
- PediQUEST Response, Usual Cancer Care
- Keywords
- Advanced Cancer
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PediQUEST ResponseOther
PediQUEST Response consists of regular feedback of electronic patient reported outcomes (child symptoms and quality of life) to providers and families coupled with involvement of the palliative care team, who will meet with families and providers within three weeks of randomization and follow-up at least monthly as deemed necessary based on PediQUEST feedback reports and other clinical indications.
Usual Cancer CareOther
Patients assigned to the usual cancer care arm, will receive the standard care provided by participating sites + be required to complete PediQUEST surveys (but no reports will be generated).
| Study group | Role | What they receive |
|---|---|---|
| PediQUEST Response | Experimental | * Weekly PediQUEST surveys are automatically assigned to parents and children (if 5 years old or older) and sent 48 hours prior to participant's usual clinic day
* Once a PediQUEST survey is assigned, automated email reminders/app notifications are sent daily for two days
* After 48 hours, unanswered or incomplete surveys are auto-submitted
* PQ-feedback report generated automatically after a PQ Survey is answered
* A pdf of the report is automatically emailed/available on mobile App to designated recipients
* Will also receive oncology-PC integrated care through the Response team
* Duration of follow-up: 18 weeks (2-week run-in period, followed by a 16-week post-randomization follow-up) Receives: Other: PediQUEST Response |
| Usual Cancer Care | Other | * Will receive the usual cancer care provided at the participating sites
* Will complete weekly PQ-Surveys (no feedback reports will be generated)
* Can receive regular palliative care consultations following the site's usual referral procedures
* Same follow-up (18 weeks) Receives: Other: Usual Cancer Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Child Quality of Life
The primary outcome was child Pediatric Quality of Life Inventory 4.0 (PedsQL) total score, analyzed by respondent: (a) parent-reported for all enrolled children (n=154), and (b) child-reported (if 5 years of age or older) (n=103). PedsQL total scores are calculated as the average of the 23 items that integrate the tool. Scores range from 0-100 (100 is excellent quality of life). A score of <70 is considered fair/poor quality of life.
Intervention effect was measured as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Secondary outcomes (12)
Change in Child Quality of Life - Physical Subscale
Secondary child PedsQL subscale outcomes included: (a) PedsQL physical (8 items) and (b) psychosocial (15 items) subscale scores (range 0-100, higher is better).
Intervention effect was measured as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Child Quality of Life - Psychosocial Subscale
Secondary child PedsQL subscale outcomes included: (a) PedsQL physical (8 items) and (b) psychosocial (15 items) subscale scores (range 0-100, higher is better).
Intervention effect was measured as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Child Symptom Burden-PQMSAS Total
Secondary child outcome PediQUEST-Memorial Symptom Assessment Scale (PQ-MSAS) total scores calculated as the average of individual symptom scores (26 for teens and caregivers and 8 for children aged 7-12). Individual symptom scores are calculated as the average of the three symptom subquestions. All symptom scores and total scores range from 0-100, 100 indicates higher symptom burden.
Intervention effect calculated as: mean difference between arms of the difference between the 16-week average and baseline scores as reported by (a) the parent for all enrolled children and (b) the patient, if 7 years of age or older. Effect estimated using mixed linear models with child as a random effect. Unadjusted models included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Child Symptom Burden- PQMSAS-physical Subscale
Secondary child outcome PediQUEST-Memorial Symptom Assessment Scale (PQ-MSAS) physical subscale scores calculated as the average of eight physical symptom scores (only for teens and caregivers). Scores range from 0-100, 100 indicates higher symptom burden.
Time Frame: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Intervention effect was measured as the mean difference between arms of the difference between the 16-week average and baseline scores as reported by (a) the parent for all enrolled children and (b) the patient, if ≥13 years of age. Effect estimated using mixed linear models with child as a random effect. Unadjusted models included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: 16 weeks (measured weekly from point of randomization)
Change in Child Symptom Burden- PQMSAS-psychosocial Subscale
Secondary child outcome PediQUEST-Memorial Symptom Assessment Scale (PQ-MSAS) psychosocial subscale scores calculated as the average of 6 psychological symptom scores (only for teens and caregivers). Scores range from 0-100, 100 indicates higher symptom burden.
Intervention effect was measured as the mean difference between arms of the difference between the 16-week average and baseline scores as reported by (a) the parent for all enrolled children and (b) the patient, if ≥13 years of age. Effect estimated using mixed linear models with child as a random effect. Unadjusted models included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Parental Distress (Anxiety)
Parent anxiety measured using Spielberger's-State Anxiety Inventory-State tool (STAI-S). Scores range from 20 to 80, with higher scores correlating with greater anxiety.
Intervention effect estimated as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Parental Distress (Depression)
Parent depression measured using the Center for Epidemiologic Studies Short Depression Scale (CES-D-10) tool. Scores range from 0 to 30 (Score of 10 or higher indicates the presence of significant depressive symptoms).
Intervention effect estimated as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Family Activation (Symptom Treatment Activation - Number of Complementary Therapies (UCT) Used)
Number of complementary therapies used over the past month were reported by parents using an ad hoc checklist of 33 therapies based on an NIH Center for Complementary and Integrative Health list. Parents reported both use (yes/no) of each therapy and reasons for use.
Intervention effect estimated as the mean difference between arms of the difference between the 16-week average and baseline number of complementary therapies used estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Parental Distress (Stress)
Parent stress was measured using an adapted version of the Response to Stress Questionnaire-Pain portion. The tool assesses 12 potential stress sources. Scores range from 1-4 (higher means more stress). Symptom-related stress score calculated as the sum of the 12 item scores.
Intervention effect estimated as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline and 16 weeks (measured at weeks 8 and 16 from point of randomization, the 16-week average was used).
Change in Family Activation (Coping-instrumental Support)
Coping was measured using selected items from the short version of the Coping Orientation to Problems Experienced Inventory (BRIEF COPE) tool including the instrumental support scale score. Scale score is calculated as the average of the items that measure the strategy. Items scored 1-4 (higher means using that strategy a lot).
Intervention effect estimated as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects. We report below on instrumental support scores.
Measured: Baseline and 16 weeks (measured at weeks 8 and 16 from point of randomization, the 16-week average was used).
Change in Family Activation (Symptom Treatment Activation - UCT - Types)
Types of complementary therapies used were measured using an ad hoc checklist of complementary therapies as explained for outcome 9. Complementary measures were grouped as physical, psychological, nutritional, combined, and whole-system approaches based on NCCIH framework.
Intervention effect estimated as the mean difference between arms of the difference between the 16-week average and baseline types of therapies used estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Family Activation (Symptom Treatment Activation - Use of Psychosocial Services)
Information on use of psychosocial services was gathered from the medical record and operationalized as "number of encounters documented with psychosocial clinicians." Intervention effect estimated as the mean difference between arms of the 16-week average of encounters with psychosocial clinicians estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
Measured: Data were collected from randomization point to 16 weeks point.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2018
- Study start1 Mar 2018
- Primary completion14 Sep 2022
- Study completion14 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Advanced CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 194 participants (actual)
Sponsor and investigators
- Lead sponsor
- Joanne Wolfe, MD, MPH
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital of Philadelphia, Children's Hospitals and Clinics of Minnesota, University of Alabama at Birmingham, Deakin University, Boston Children's Hospital, Ann & Robert H Lurie Children's Hospital of Chicago, Baylor College of Medicine, Dana-Farber Cancer Institute, National Institute of Nursing Research (NINR), St. Jude Children's Research Hospital, Nationwide Children's Hospital, Seattle Children's Hospital
- Responsible party
- Sponsor-investigator, Joanne Wolfe, MD, MPH, Massachusetts General Hospital
- Investigators
- Joanne Wolfe, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03408314
- Sponsor's protocol ID
- 17-102
- Other IDs
- R01NR016720-03
- Status last verified
- Aug 2026
- First submitted
- 17 Jan 2018
- Last updated
- 7 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Study Data Center will oversee the data sharing process, with input from the Data and Safety Monitoring Board. Once the database is consolidated, all study investigators that are interested in leading a particular analysis, will be given access to the final password-protected de-identified data sets. Out of study investigators may be allowed access to the datasets after a formal analysis proposal is approved by the steering committee.
Publications
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.
PubMed 36346783Feifer 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.
PubMed 39414121Eche-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.
PubMed 38865435Merz 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.
PubMed 37290730Dussel V, Orellana L, Requena ML, Avery M, Becker D, Ullrich CK, Feudtner C, Freedman JL, Kang TI, Waldman ED, Gerhardt CA, Bakitas MA, Baker JN, Friedrichsdorf SJ, Rosenberg AR, Wolfe J. Integrating Electronic Patient-Reported Outcomes and Palliative Care in Pediatric Advanced Cancer: The PediQUEST Response Multisite Randomized Controlled Trial. J Clin Oncol. 2026 Mar 10;44(8):685-697. doi: 10.1200/JCO-25-01036. Epub 2026 Feb 4.
PubMed 41637683Tsuchiyose E, Feifer D, Merz AF, Avery M, Eche-Ugwu IJ, Awofeso O, Orellana L, Becker D, Feudtner C, Freedman JL, Kang TI, Rosenberg AR, Waldman ED, Ullrich CK, Wolfe J, Requena ML, Dussel V. Research Burden in Pediatric Cancer: A Mixed Methods Study From the PediQUEST Response Trial. J Pain Symptom Manage. 2025 Dec;70(6):649-663. doi: 10.1016/j.jpainsymman.2025.08.032. Epub 2025 Sep 1.
PubMed 40902740
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03408314
- Acronym / sponsor ID
- 17-102
- Conditions
- Advanced Cancer
- Keywords
- Advanced Cancer
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 194 participants (actual)
- Lead sponsor
- Joanne Wolfe, MD, MPHUniversity, hospital or other
- Collaborators
- Children's Hospital of Philadelphia, Children's Hospitals and Clinics of Minnesota, University of Alabama at Birmingham, Deakin University, Boston Children's Hospital, Ann & Robert H Lurie Children's Hospital of Chicago, Baylor College of Medicine, Dana-Farber Cancer Institute, National Institute of Nursing Research (NINR), St. Jude Children's Research Hospital, Nationwide Children's Hospital, Seattle Children's Hospital
- Study start
- 1 Mar 2018 (actual)
- Primary completion
- 14 Sep 2022 (actual)
- Study completion
- 14 Sep 2022 (actual)
- First posted
- 24 Jan 2018 (actual)
- Results
- Posted 7 Aug 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PediQUEST Response | Experimental | Other: PediQUEST Response* Weekly PediQUEST surveys are automatically assigned to parents and children (if 5 years old or older) and sent 48 hours prior to participant's usual clinic day * Once a PediQUEST survey is assigned, automated email reminders/app notifications are sent daily for two days * After 48 hours, unanswered or incomplete surveys are auto-submitted * PQ-feedback report generated automatically after a PQ Survey is answered * A pdf of the report is automatically emailed/available on mobile App to designated recipients * Will also receive oncology-PC integrated care through the Response team * Duration of follow-up: 18 weeks (2-week run-in period, followed by a 16-week post-randomization follow-up) |
| Usual Cancer Care | Other | Other: Usual Cancer Care* Will receive the usual cancer care provided at the participating sites * Will complete weekly PQ-Surveys (no feedback reports will be generated) * Can receive regular palliative care consultations following the site's usual referral procedures * Same follow-up (18 weeks) |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PediQUEST ResponsePediQUEST Response consists of regular feedback of electronic patient reported outcomes (child symptoms and quality of life) to providers and families coupled with involvement of the palliative care team, who will meet with families and providers within three weeks of randomization and follow-up at least monthly as deemed necessary based on PediQUEST feedback reports and other clinical indications. | Other | PediQUEST Response |
| Usual Cancer CarePatients assigned to the usual cancer care arm, will receive the standard care provided by participating sites + be required to complete PediQUEST surveys (but no reports will be generated). | Other | Usual Cancer Care |
Primary outcome
Change in Child Quality of Life
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
The primary outcome was child Pediatric Quality of Life Inventory 4.0 (PedsQL) total score, analyzed by respondent: (a) parent-reported for all enrolled children (n=154), and (b) child-reported (if 5 years of age or older) (n=103). PedsQL total scores are calculated as the average of the 23 items that integrate the tool. Scores range from 0-100 (100 is excellent quality of life). A score of <70 is considered fair/poor quality of life. Intervention effect was measured as the mean difference between arms of the difference between the 16-week average and baseline scores estimated using mixed linear models with child as a random effect. For unadjusted models we included study arm, time (categorical, 0 to 16 weeks) and arm-by-time interaction as fixed effects.
12 secondary outcomes
Change in Child Quality of Life - Physical Subscale
Time frame: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Child Quality of Life - Psychosocial Subscale
Time frame: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Child Symptom Burden-PQMSAS Total
Time frame: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Child Symptom Burden- PQMSAS-physical Subscale
Time frame: 16 weeks (measured weekly from point of randomization)
Change in Child Symptom Burden- PQMSAS-psychosocial Subscale
Time frame: Baseline (average of run-in scores, i.e. 2-3 scores) and 16 weeks (measured weekly from point of randomization, the 16-week average was used).
Change in Parental Distress (Anxiety)
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Parental Distress (Depression)
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Family Activation (Symptom Treatment Activation - Number of Complementary Therapies (UCT) Used)
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Parental Distress (Stress)
Time frame: Baseline and 16 weeks (measured at weeks 8 and 16 from point of randomization, the 16-week average was used).
Change in Family Activation (Coping-instrumental Support)
Time frame: Baseline and 16 weeks (measured at weeks 8 and 16 from point of randomization, the 16-week average was used).
Change in Family Activation (Symptom Treatment Activation - UCT - Types)
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
Change in Family Activation (Symptom Treatment Activation - Use of Psychosocial Services)
Time frame: Data were collected from randomization point to 16 weeks point.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 36346783Dussel 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.
- Results publication: PubMed 39414121Feifer 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.
- Results publication: PubMed 38865435Eche-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.
- Results publication: PubMed 37290730Merz 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.
- Derived from this study: PubMed 41637683Dussel V, Orellana L, Requena ML, Avery M, Becker D, Ullrich CK, Feudtner C, Freedman JL, Kang TI, Waldman ED, Gerhardt CA, Bakitas MA, Baker JN, Friedrichsdorf SJ, Rosenberg AR, Wolfe J. Integrating Electronic Patient-Reported Outcomes and Palliative Care in Pediatric Advanced Cancer: The PediQUEST Response Multisite Randomized Controlled Trial. J Clin Oncol. 2026 Mar 10;44(8):685-697. doi: 10.1200/JCO-25-01036. Epub 2026 Feb 4.
- Derived from this study: PubMed 40902740Tsuchiyose E, Feifer D, Merz AF, Avery M, Eche-Ugwu IJ, Awofeso O, Orellana L, Becker D, Feudtner C, Freedman JL, Kang TI, Rosenberg AR, Waldman ED, Ullrich CK, Wolfe J, Requena ML, Dussel V. Research Burden in Pediatric Cancer: A Mixed Methods Study From the PediQUEST Response Trial. J Pain Symptom Manage. 2025 Dec;70(6):649-663. doi: 10.1016/j.jpainsymman.2025.08.032. Epub 2025 Sep 1.
- Individual participant dataSharing planned: Yes. The Study Data Center will oversee the data sharing process, with input from the Data and Safety Monitoring Board. Once the database is consolidated, all study investigators that are interested in leading a particular analysis, will be given access to the final password-protected de-identified data sets. Out of study investigators may be allowed access to the datasets after a formal analysis proposal is approved by the steering committee.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joanne Wolfe, MD principal investigator
Dana-Farber Cancer Institute
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PediQUEST Response and Usual Cancer Care in people with Advanced Cancer, run by Joanne Wolfe, MD, MPH at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Aug 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Child Quality of Life (time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03408314, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.