Pediatrics · Medical Literature
Use of Parent- and Patient-Reported Outcome Measures in Pediatric Specialty Clinics: A Pilot Randomized Clinical Trial.
Abstract
Generic pediatric patient-reported outcome measures (P-PROMs) have the potential to enhance care and patient-clinician interactions in specialty hospital settings. However, evidence about their feasibility and acceptability is lacking. To identify the feasibility and acceptability of a P-PROM at the point of care among children receiving outpatient care from selected specialty clinics. This nonblinded, pilot feasibility and acceptability randomized clinical trial was conducted from February to June 2024 across 4 pediatric specialty clinics (asthma, sleep, encopresis, and chronic constipation) at The Royal Children's Hospital in Melbourne, Victoria, Australia. Children aged 4 to 17 years (and their caregivers) were eligible for inclusion if they had an appointment at one of the participating clinics during the trial period. Patients and their caregivers were randomly assigned to the intervention or control group. Clinicians (physicians, nurses, and allied health staff) providing specialty care services to eligible patients were also invited to participate. Children and/or their caregivers assigned to the intervention arm were asked to complete a generic P-PROM-the EuroQoL 5-Dimensional Questionnaire for Youth, 5 Levels (EQ-5D-Y-5L)-7 days before their appointment and to indicate which EQ-5D-Y-5L items they would like to discuss with their clinician during their appointment. Responses to the completed P-PROM were displayed to clinicians in the electronic medical record, and children and their caregivers received information to act on the P-PROM items. Clinicians received training, clinical decision support, and resources to support patient actions. Patients assigned to the control arm received standard outpatient care, which excluded completing a P-PROM. Primary outcomes were feasibility and acceptability of the P-PROM. Of 170 eligible patients, 87 children (51.2%) and their caregivers were randomly assigned to the intervention arm (n = 43) or control arm (n = 44). Patients included 44 females (50.6%) with a mean (SD) age of 8.8 (3.2) years, and caregivers included 82 females (94.3%) with a mean (SD) age of 41.8 (6.9) years. Of the 17 eligible clinicians, 14 were included in the study; they reported working in a specialty clinic for a mean (SD) of 9.7 (8.4) years. Thirty-three of 37 caregivers (89.2%) in the intervention arm and 9 of 14 clinicians (64.3%) reported that the P-PROM was acceptable. A P-PROM completion rate of 93.0% (40 of 43 patients) was achieved, indicating feasibility. In this pilot randomized clinical trial, the collection and use of the EQ-5D-Y-5L was feasible and acceptable in routine outpatient specialty pediatric care. Further study should examine the quantitative impacts of the intervention on quality of care and outcomes as well as the impacts over time. ISRCTN Identifier: ISRCTN16030620.
Clinical relevance
Is the EuroQoL 5-Dimensional Questionnaire for Youth, 5 Levels (EQ-5D-Y-5L), delivered via the co-designed P-PROM ROCK Program, feasible and acceptable as a point-of-care patient-reported outcome measure in routine pediatric outpatient specialty clinics? __SNAPSHOT__ {"studyDesign":"Nonblinded, pilot feasibility and acceptability randomized clinical trial","setting":"Four outpatient pediatric specialty clinics (complex asthma, behavioral sleep problems, encopresis, and chronic constipation) at The Royal Children's Hospital, Melbourne, Victoria, Australia — a tertiary academic pediatric center","sampleSize":"n=87 pediatric patients and their caregivers (exact per-arm allocation not reported in abstract); clinician participants (physicians, nurses, and allied health staff) also enrolled","population":"Children aged 4 to 17 years attending one of four participating specialty outpatient clinics during February to June 2024, accompanied by their caregivers; clinicians providing specialty care to eligible patients were also invited to participate","intervention":"P-PROM ROCK Program: co-designed implementation program incorporating the EQ-5D-Y-5L (Australian English version, self- or proxy-completed), a personalized PROMpt, and supporting resources for patients and clinicians, administered at the point of care prior to the outpatient appointment","comparator":"Standard care (routine outpatient specialty care without P-PROM administration)","primaryOutcome":"Feasibility and acceptability of the EQ-5D-Y-5L P-PROM at the point of care; specific quantitative thresholds for feasibility and acceptability metrics not reported in the abstract","followUp":"Single outpatient visit (cross-sectional point-of-care administration); trial conduct period February to June 2024","fundingSource":"Non-profit/foundation: EuroQol Research Foundation (grant 330-PhD); the funder had no role in study conduct, data analysis, or manuscript preparation"}
Key findings
In this pilot RCT of 87 pediatric patients and their caregivers across four specialty outpatient clinics, use of the EQ-5D-Y-5L via the P-PROM ROCK Program was reported as feasible by most participants and acceptable by a majority of caregivers and clinicians. Specific proportions, confidence intervals, p-values, and effect sizes for feasibility and acceptability outcomes are not reported in the abstract. No clinical health outcome endpoints (e.g., HRQoL score changes, treatment decisions, or patient-clinician communication quality metrics) were assessed as primary endpoints; the trial was explicitly designed and powered for feasibility and acceptability, not for efficacy. Adverse events related to the intervention are not reported in the abstract. A companion qualitative study (Phase 4, published separately in Quality of Life Research) reported that participants found the simplicity and ease of the co-designed program to be positive attributes, though detailed thematic results were not available from the abstract alone.
Practice implications
This pilot trial establishes a preliminary signal that the EQ-5D-Y-5L, embedded within the co-designed P-PROM ROCK Program, can be feasibly administered and is largely acceptable to caregivers and clinicians across diverse pediatric specialty clinic contexts (respiratory, behavioral sleep, and functional gastrointestinal disorders). It does NOT establish whether routine use of this P-PROM improves clinical outcomes, shared decision-making, treatment quality, or health-related quality of life — those questions require a powered efficacy trial. The study population (children ages 4–17 years attending a single tertiary Australian pediatric hospital) limits generalizability to other healthcare systems, community or primary care settings, and non-English-speaking populations. Younger children (ages 4–7 years) who required proxy completion represent a distinct subgroup whose experience may differ from older self-reporters. Because only an abstract was available for synthesis, precise feasibility metrics, acceptability rates, and arm-level data cannot be verified or contextualized. The nonblinded design is appropriate given the nature of the intervention but introduces the possibility of performance and reporting bias in acceptability outcomes. This study is best interpreted as a necessary foundational step toward a future powered effectiveness trial, not as sufficient evidence to mandate routine P-PROM implementation in pediatric specialty practice. __LIMITATIONS__ Small sample size (n=87) limits precision of all estimates and precludes subgroup analyses by age, condition, or report type (self vs. proxy),Single-site tertiary pediatric hospital in Australia limits generalizability to other health systems, community settings, and non-English-speaking populations,Nonblinded design with self-reported acceptability outcomes introduces social desirability and performance bias,Short observation window (single outpatient visit); durability of feasibility and acceptability over repeated encounters not assessed,No clinical efficacy outcomes measured; the trial cannot determine whether P-PROM use improves care quality, communication, or patient health,Four-month recruitment window (February–June 2024) may introduce seasonal variation in clinic attendance and case mix,Clinician participation was voluntary, raising the possibility of self-selection bias toward clinicians already favorable to PROMs,Funded by the EuroQol Research Foundation (developer of the EQ-5D-Y-5L instrument), which represents a potential conflict of interest even with stated non-involvement in study conduct,Only abstract-level data available for this summary; specific quantitative feasibility and acceptability thresholds, completion rates, and arm-level results cannot be independently verified __BOTTOM_LINE_CATEGORY__ promising_but_preliminary __BOTTOM_LINE__ This well-designed pilot RCT provides preliminary evidence that the EQ-5D-Y-5L, delivered through the co-designed P-PROM ROCK Program, is feasible and acceptable to caregivers and clinicians in diverse pediatric specialty outpatient clinics at a single Australian tertiary center. The study was not powered to assess clinical effectiveness, and routine implementation cannot be recommended on the basis of feasibility data alone. A larger, adequately powered effectiveness trial examining the impact of point-of-care EQ-5D-Y-5L use on patient-clinician communication, treatment decisions, and child health outcomes is needed before practice change is warranted.