<p><strong>CONTEXT: </strong>Modifiable factors of health-related quality of life (HRQOL) are poorly described among children with advanced cancer. Symptom-distress may be an important factor for intervention.</p>

<p><strong>OBJECTIVES: </strong>We aimed to describe patient-reported HRQOL and its relationship to symptom distress.</p>

<p><strong>METHODS: </strong>Prospective, longitudinal data from the multicenter Pediatric Quality of Life and Symptoms Technology (PediQUEST) study included primarily patient-reported symptom-distress and HRQOL, measured at most weekly with the Memorial Symptoms Assessment Scale and Pediatric Quality of Life [PedsQL] inventory, respectively. Associations were evaluated using linear mixed-effects models adjusting for sex, age, cancer type, intervention arm, treatment intensity, and time since disease progression.</p>

<p><strong>RESULTS: </strong>Of 104 enrolled patients, 49% were female, 89% were white, and median age was 12.6 years. Nine hundred and twenty surveys were completed over nine months of follow-up (84% by patients). The median total PedsQL score was 74 (IQR 63-87) and was "poor/fair" (e.g., &lt;70) 38% of the time. "Poor/fair" categories were highest in physical (53%) and school (48%) compared to emotional (24%) and social (16%) subscores. Thirteen of 24 symptoms were independently associated with reductions in overall or domain-specific HRQOL. Patients commonly reported distress from two or more symptoms, corresponding to larger HRQOL score reductions. Neither cancer type, time since progression, treatment intensity, sex, nor age was associated with HRQOL scores in multivariable models. Among 25 children completing surveys during the last 12 weeks of life, 11 distressing symptoms were associated with reductions in HRQOL.</p>

<p><strong>CONCLUSION: </strong>Symptom-distress is strongly associated with HRQOL. Future research should determine whether alleviating distressing symptoms improves HRQOL in children with advanced cancer.</p>

<p><strong>PURPOSE: </strong>This study aimed to determine whether feeding back patient-reported outcomes (PROs) to providers and families of children with advanced cancer improves symptom distress and health-related quality of life (HRQoL).</p>

<p><strong>PATIENTS AND METHODS: </strong>This study was a parallel, multicentered pilot randomized controlled trial. At most once per week, children age ≥ 2 years old with advanced cancer or their parent completed the computer-based Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) survey consisting of age- and respondent-adapted versions of the Memorial Symptom Assessment Scale (MSAS), Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL4.0), and an overall Sickness question. In the intervention group (n = 51), oncologists and families received printed reports summarizing PROs; e-mails were sent to oncologists and subspecialists when predetermined scores were exceeded. No feedback was provided in the control group (n = 53). Primary outcomes included linear trends of MSAS, PedsQL4.0 total and subscale scores, and Sickness scores during 20 weeks of follow-up, along with child, parent, and provider satisfaction with PediQUEST feedback.</p>

<p><strong>RESULTS: </strong>Feedback did not significantly affect average MSAS, PedsQL4.0, or Sickness score trends. Post hoc subgroup analyses among children age ≥ 8 years who survived 20 weeks showed that feedback improved PedsQL4.0 emotional (+8.1; 95% CI, 1.8 to 14.4) and Sickness (-8.2; 95% CI, -14.2 to -2.2) scores. PediQUEST reports were valued by children, parents, and providers and contributed at least sometimes to physician initiation of a psychosocial consult (56%).</p>

<p><strong>CONCLUSION: </strong>Although routine feedback of PROs did not significantly affect the child's symptoms or HRQoL, changes were in expected directions and improvements observed in emotional HRQoL through exploratory analyses were encouraging. Importantly, children, parents, and providers value PRO feedback.</p>

<p><strong>PURPOSE: </strong>Thousands of children are living with advanced cancer; yet patient-reported outcomes (PROs) have rarely been used to describe their experiences. We aimed to describe symptom distress in 104 children age 2 years or older with advanced cancer enrolled onto the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) Study (multisite clinical trial evaluating an electronic PRO system).</p>

<p><strong>METHODS: </strong>Symptom data were collected using age- and respondent-adapted versions of the PediQUEST Memorial Symptom Assessment Scale (PQ-MSAS) at most once per week. Clinical and treatment data were obtained from medical records. Individual symptom scores were dichotomized into high/low distress. Determinants of PQ-MSAS scores were explored using linear mixed-effects models.</p>

<p><strong>RESULTS: </strong>During 9 months of follow-up, PQ-MSAS was administered 920 times: 459 times in teens (99% self-report), 249 times in children ages 7 to 12 years (96% child/parent report), and 212 times in those ages 2 to 6 years (parent reports). Common symptoms included pain (48%), fatigue (46%), drowsiness (39%), and irritability (37%); most scores indicated high distress. Among the 73 PQ-MSAS surveys administered in the last 12 weeks of life, pain was highly prevalent (62%; 58% with high distress). Being female, having a brain tumor, experiencing recent disease progression, and receiving moderate- or high-intensity cancer-directed therapy in the prior 10 days were associated with worse PQ-MSAS scores. In the final 12 weeks of life, receiving mild cancer-directed therapy was associated with improved psychological PQ-MSAS scores.</p>

<p><strong>CONCLUSION: </strong>Children with advanced cancer experience high symptom distress. Strategies to promote intensive symptom management are indicated, especially with disease progression or administration of intensive treatments.</p>

<p><strong>CONTEXT: </strong>Pediatric palliative care randomized controlled trials (PPC-RCTs) are uncommon.</p>

<p><strong>OBJECTIVES: </strong>To evaluate the feasibility of conducting a PPC-RCT in pediatric cancer patients.</p>

<p><strong>METHODS: </strong>This was a cohort study embedded in the Pediatric Quality of Life and Evaluation of Symptoms Technology Study (NCT01838564). This multicenter PPC-RCT evaluated an electronic patient-reported outcomes system. Children aged two years and older, with advanced cancer, and potentially eligible for the study were included. Outcomes included: pre-inclusion attrition (patients not approached, refusals); post-inclusion attrition (drop-out, elimination, death, and intermittent attrition (IA; missing surveys) over nine months of follow-up); child/teenager self-report rates; and, reasons to enroll/participate.</p>

<p><strong>RESULTS: </strong>Over five years, of the 339 identified patients, 231 were eligible (in 22, we could not verify eligibility); 84 eligible patients were not approached and 43 declined participation. Patients not approached were more likely to die or have brain tumors. We enrolled 104 patients. Average enrollment rate was one patient per site per month; shortening follow-up from nine to three months (with optional re-enrollment) increased recruitment by 20%. A total of 87 patients completed the study (24 died) and 17 dropped out. Median IA was 41% in the first 20 weeks of follow-up and more than 60% in the eight weeks preceding death. Child/teenager self-report was 94%. Helping others, low burden procedures, incentives, and staff attitude were frequent reasons to enroll/participate.</p>

<p><strong>CONCLUSION: </strong>A PPC-RCT in children with advanced cancer was feasible, post-inclusion retention adequate; many families participated for altruistic reasons. Strategies that may further PPC-RCT feasibility include: increasing target population through large multicenter studies, approaching sicker patients, preventing exclusion of certain patient groups, and improving data collection at end of life.</p>