Consensus outcomes between health professionals and parents for oral corticosteroids in treating preschool wheeze: a multi-national survey and nominal group technique study

Bohee Lee; Stephen W Turner; Jasmine Hine; Ann McMurray; Damian Roland; Meredith Louise Borland; Peter Csonka; Jonathan Grigg; Theresa W Guilbert; Tuomas Jartti; Abraham Oommen; Steff Lewis; Steve Cunningham

doi:10.1136/archdischild-2024-327696

Consensus outcomes between health professionals and parents for oral corticosteroids in treating preschool wheeze: a multi-national survey and nominal group technique study

Arch Dis Child. 2025 Jan 15:archdischild-2024-327696. doi: 10.1136/archdischild-2024-327696. Online ahead of print.

Authors

Bohee Lee^{1

2

3}, Stephen W Turner⁴, Jasmine Hine^{5

3}, Ann McMurray^{5

6}, Damian Roland^{7

8}, Meredith Louise Borland^{9

10}, Peter Csonka^{11

12}, Jonathan Grigg¹³, Theresa W Guilbert^{14

15}, Tuomas Jartti^{16

17}, Abraham Oommen¹⁸, Steff Lewis^{5

19}, Steve Cunningham²⁰

Affiliations

¹ Asthma UK Centre for Applied Research, Edinburgh, UK [email protected].
² Centre for Population Health Sciences, University of Edinburgh, Edinburgh, UK.
³ National Heart and Lung Institute, Imperial College London, London, UK.
⁴ Royal Aberdeen Children's Hospital, Aberdeen, UK.
⁵ Asthma UK Centre for Applied Research, Edinburgh, UK.
⁶ Royal Hospital for Sick Children, Edinburgh, UK.
⁷ SAPPHIRE Group, Health Sciences, University of Leicester, Leicester, UK.
⁸ Paediatric Emergency Medicine Leicester Academic (PEMLA) Group, University Hospitals of Leicester NHS Trust, Leicester, UK.
⁹ Emergency Medicine, Perth Children's Hospital, Nedlands, Western Australia, Australia.
¹⁰ Divisions of Paediatrics and Emergency Medicine, The University of Western Australia - Perth Campus, Perth, Western Australia, Australia.
¹¹ Center for Child, Adolescent, and Maternal Health Research, Tampere University, Tampere, Finland.
¹² Paediatrics, Terveystalo Healthcare Services, Tampere, Finland.
¹³ Centre for Child Health, Blizard Institute, Queen Mary University of London, London, UK.
¹⁴ Division of Pulmonology Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
¹⁵ Department of Pediatrics, University of Cincinnati, Cincinnati, Ohio, USA.
¹⁶ Department of Pediatrics, TYKS Turku University Hospital, Turku, Finland.
¹⁷ Department of Pediatrics, University of Turku, Turku, Finland.
¹⁸ Department of Paediatrics, Milton Keynes University Hospital NHS Foundation Trust, Milton Keynes, UK.
¹⁹ Edinburgh Clinical Unit, University of Edinburgh, Edinburgh, UK.
²⁰ Department of Child Life and Health, University of Edinburgh Institute for Regeneration and Repair, Edinburgh, UK.

PMID: 39814533
DOI: 10.1136/archdischild-2024-327696

Abstract

Objective: To obtain priority consensus for outcome measures of oral corticosteroid treatment of preschool wheeze that represent stakeholder groups.

Design: (1) A systematic review to identify a set of outcome measures; (2) an international survey for healthcare professionals (HCPs) and a nominal group meeting with parents; (3) a final consensus nominal group meeting with key HCPs (trial investigators and paediatric emergency medicine clinicians) and the same parent group.

Main outcome measures: Consensus priority of treatment outcome measures, outcome minimal clinically important differences (MCIDs) and level of concerns about adverse effects.

Results: Through an iterative process engaging HCPs and parents, the final consensus on a primary outcome was Wheezing Severity Score (WSS). Secondary outcomes prioritised as a revisit to general practice/emergency department, rehospitalisation, length of hospital stay (LOS), time back to normal, doses of short-acting beta-agonists and additional steroid course. Compared with placebo, clinicians considered the median MCID change in WSS at 4 and 12 hours as 40% (IQR 29-51%) and 50% (37-63%) and 5 hours (4-6 hours) for LOS, and 2 days (2-3 days) for a time back to normal. Parents identified MCIDs which were frequently longer than physiologically observed impacts in trials. Concerns about multiple steroid doses were most prevalent.

Conclusions: Stakeholders prioritised change in WSS as the most favourable outcome measure. Our study demonstrated the potential of parent/patient engagement in co-creating patient research outcomes. Incorporating this result in the design of future clinical research will provide a more holistic assessment of the impact of treatment while ensuring relevant primary research outcomes.

Keywords: Paediatric Emergency Medicine; Paediatrics; Respiratory Medicine.