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Published on: June 29, 2021
Patient-reported outcome measures for paediatric acute lower respiratory infection studies
Daniel B Oakes1,2, Megan J Baker2, Charlie McLeod2,3
1School of Population and Global Health, The University of Western Australia, Crawley, Australia daniel.oakes@telethonkids.org.au.
Insights
Patient-reported outcome measures (PROMs) for pediatric acute lower respiratory infections (ALRIs) are under-validated. New PROMs are urgently needed, especially for Indigenous children, to accurately capture outcomes in clinical trials.
Area of Science:
- Pediatric Health Research
- Clinical Trial Methodology
- Health Outcomes Measurement
Background:
- Patient-reported outcome measures (PROMs) are crucial for clinical trials but underutilized in pediatric acute lower respiratory infections (ALRIs).
- Systematic reporting on PROMs for children with ALRIs is lacking.
- This study aimed to identify and characterize PROMs used in pediatric ALRI research and evaluate their measurement properties.
Purpose of the Study:
- To systematically identify and characterize patient-reported outcomes and PROMs used in pediatric ALRI studies.
- To summarize the measurement properties of identified PROMs.
- To highlight gaps in PROM validation for pediatric ALRI populations.
Main Methods:
- Comprehensive literature search of Medline, Embase, and Cochrane databases up to April 2022.
- Inclusion of studies reporting on PROM use or development in subjects under 18 years with ALRIs.
- Extraction of study, population, and PROM characteristics.
Main Results:
- 18 studies met inclusion criteria, utilizing 12 distinct PROMs.
- The Canadian Acute Respiratory Illness and Flu Scale and EuroQol-Five Dimensions-Youth system were the most frequent disease-specific and generic PROMs, respectively.
- Significant heterogeneity in validation methods was observed; identified PROMs lacked validation for young children and sufficient content validity for First Nations children.
Conclusions:
- There is a critical need for the development of novel PROMs for pediatric ALRIs.
- PROM development must consider populations disproportionately affected by ALRI burden.
- Enhanced validation is required for PROMs used in diverse pediatric populations, including Indigenous children.
Background:
Patient-reported outcome measures (PROMs) are recommended for capturing meaningful outcomes in clinical trials. The use of PROMs for children with acute lower respiratory infections (ALRIs) has not been systematically reported. We aimed to identify and characterise patient-reported outcomes and PROMs used in paediatric ALRI studies and summarise their measurement properties.
Methods:
Medline, Embase and Cochrane were searched (until April 2022). Studies that reported on patient-reported outcome (or measure) use or development and included subjects aged <18 years with ALRIs were included. Study, population and patient-reported outcome (or measure) characteristics were extracted.
Results:
Of 2793 articles identified, 18 met inclusion criteria, including 12 PROMs. Two disease-specific PROMs were used in settings in which they had been validated. The Canadian Acute Respiratory Illness and Flu Scale was the most frequently used disease-specific PROM (five studies). The EuroQol-Five Dimensions-Youth system was the most frequently used generic PROM (two studies). There was considerable heterogeneity in validation methods. The outcome measures identified in this review lack validation for young children and none involve sufficient content validity for use with First Nations children.
Conclusions:
There is an urgent need for PROM development that considers the populations in which the burden of ALRI predominates.
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