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Comparative performance of common paediatric patient-reported outcome measures (P-PROMs) across health conditions
Renee Jones1, Nancy Devlin1, Kim Dalziel1
1Melbourne Health Economics, The University of Melbourne School of Population and Global Health, Melbourne, Victoria, Australia.
Insights
This study compared pediatric patient-reported outcome measures (P-PROMs) for common childhood conditions. The Paediatric Quality of Life Inventory (PedsQL) performed well across all assessed conditions, showing good reliability and validity.
Area of Science:
- Pediatric Health Outcomes Research
- Clinical Trial Methodology
- Psychometrics
Background:
- Patient-reported outcome measures (P-PROMs) are crucial for evaluating pediatric health conditions.
- Standardized comparison of P-PROMs across various pediatric conditions is needed to guide clinical practice and research.
- The Australian Paediatric Multi-Instrument Comparison study aimed to rigorously assess P-PROM measurement properties.
Purpose of the Study:
- To compare the measurement properties of various P-PROMs used in pediatric clinical trials and practice.
- To identify the most suitable P-PROMs for specific common childhood conditions.
- To provide evidence-based recommendations for P-PROM selection.
Main Methods:
- The study involved children aged 5-18 years across ten conditions and a healthy control group.
- Key measurement properties including acceptability, reliability, validity, and responsiveness were assessed for multiple P-PROMs.
- Instruments evaluated included PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, CHU9D, AQoL-6D, HI-3, and PROMIS-25.
Main Results:
- Several P-PROMs, including PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, CHU9D, and PROMIS-25, demonstrated good acceptability.
- All assessed measures showed known-groups validity, differentiating between healthy children and those with conditions.
- The PedsQL and EQ-5D-Y-5L exhibited the strongest test-retest reliability, with PedsQL performing favorably across all conditions.
Conclusions:
- The Paediatric Quality of Life Inventory (PedsQL) is a highly recommended P-PROM due to its favorable performance across all assessed pediatric conditions.
- Specific P-PROMs may be preferred for certain conditions, such as PedsQL and EQ-5D-Y-5L for Autism Spectrum Disorder (ASD).
- Further research is needed to evaluate the responsiveness of P-PROMs to changes in child health outcomes.
Objectives:
To compare the measurement properties of paediatric patient-reported outcome measures (P-PROMs) used in clinical trials and practice for common conditions.
Design:
Data from the Australian Paediatric Multi-Instrument Comparison study were used, including children aged 5-18 years with autism spectrum disorder (ASD) (n=510), asthma (n=487), eating disorder(s) (n=216), epilepsy (n=298), recurrent abdominal pain (n=392), sleep problems (n=346), dental problems (n=490), type 1 diabetes (T1D) (n=67), enuresis (n=182) and no health conditions (reference group) (n=1259). The acceptability, response distribution (floor/ceiling effects), known-groups validity, test-retest reliability, responsiveness and convergent validity of the Paediatric Quality of Life Inventory (PedsQL), EuroQol Group's Youth instruments with three and five response options (EQ-5D-Y-3L and EQ-5D-Y-5L), Child Health Utility 9D (CHU9D), Assessment of Quality of Life 6D (AQoL-6D), Health Utilities Index Mark 3 and Patient-Reported Outcomes Measurement Information System Paediatric Profile 25 (PROMIS-25) were assessed.
Results:
PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, CHU9D and PROMIS-25 were easy and quick to complete, indicating acceptability. Only the EQ-5D-Y-3L and EQ-5D-Y-5L demonstrated ceiling effect issues. All measures were able to differentiate between children with no health conditions and children with each condition (p<0.001, effect sizes between 0.47 and 3.5), indicating known-groups validity. Test-retest reliability varied by condition; however, the PedsQL and EQ-5D-Y-5L were the most reliable.
Conclusions:
Certain P-PROMs may be preferred in different conditions, that is, ASD (PedsQL and EQ-5D-Y-5L), asthma (PedsQL), eating disorder(s) (PedsQL and CHU9D), epilepsy (PedsQL and CHU9D), recurrent abdominal pain (PedsQL, EQ-5D-Y-5L and CHU9D), sleep problems (PedsQL), dental problems (PedsQL), T1D (PedsQL and CHU9D) and enuresis (PedsQL). Notably, the PedsQL performed favourably in all conditions assessed. More evidence is required on the responsiveness of P-PROMs to changes in child health.
Trial Registration Number:
ACTRN12621000657820.
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