Patient-reported outcome measures for paediatric acute lower respiratory infection studies

Daniel B Oakes; Megan J Baker; Charlie McLeod; Barbara Nattabi; Christopher C Blyth

doi:10.1183/16000617.0229-2022

Patient-reported outcome measures for paediatric acute lower respiratory infection studies

Eur Respir Rev. 2023 Mar 8;32(167):220229. doi: 10.1183/16000617.0229-2022. Print 2023 Mar 31.

Authors

Daniel B Oakes^{1

2}, Megan J Baker², Charlie McLeod^{2

3}, Barbara Nattabi⁴, Christopher C Blyth^{2

3

5

6}

Affiliations

¹ School of Population and Global Health, The University of Western Australia, Crawley, Australia daniel.oakes@telethonkids.org.au.
² Wesfarmers Centre of Vaccines and Infectious Diseases, Telethon Kids Institute, University of Western Australia, Nedlands, Australia.
³ Department of Infectious Diseases, Perth Children's Hospital, Nedlands, Australia.
⁴ School of Population and Global Health, The University of Western Australia, Crawley, Australia.
⁵ School of Medicine, University of Western Australia, Perth, Australia.
⁶ Department of Microbiology, PathWest Laboratory Medicine, Perth, Australia.

Abstract

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.

Publication types

Review

MeSH terms

Adolescent
Canada
Child
Child, Preschool
Humans
Patient Reported Outcome Measures*
Quality of Life
Respiratory Tract Infections* / diagnosis
Respiratory Tract Infections* / therapy