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.
Abstract

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