NCT Number: NCT03408314
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.
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Notify Me2 year and older
All sexes
Interventional
Not applicable
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
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:
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:
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.
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
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:
Exclusion criteria
The patient-parent dyad would be excluded if
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.
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).
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.
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).
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.
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).
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.
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).
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.
Time frame: 16 weeks (measured weekly from point of randomization)
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.
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).
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.
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
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.
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
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.
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was 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.
Time frame: Baseline and 16 weeks (measured at weeks 8 and 16 from point of randomization, the 16-week average was used).
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.
Time frame: Baseline and 16 weeks (measured at weeks 8 and 16 from point of randomization, the 16-week average was used).
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.
Time frame: Baseline and 16 weeks (measured monthly from point of randomization, the 16-week average was used).
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.
Time frame: Data were collected from randomization point to 16 weeks point.
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.
Joanne Wolfe, MD, MPH
Other
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
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