Abstract / Description of output
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 (MCID) 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 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 to placebo, clinicians considered median MCID change in WSS at 4 and 12 hrs as 41% (IQR 30-51) and 50% (37-63), and 5.5 hrs (4-6) for LOS, and 2 days (2-3) for time back to normal. Parents identified MCID 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.
Data availability statement
All data relevant to the study are included in the article or uploaded as supplementary information.
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 (MCID) 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 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 to placebo, clinicians considered median MCID change in WSS at 4 and 12 hrs as 41% (IQR 30-51) and 50% (37-63), and 5.5 hrs (4-6) for LOS, and 2 days (2-3) for time back to normal. Parents identified MCID 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.
Data availability statement
All data relevant to the study are included in the article or uploaded as supplementary information.
Original language | English |
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Journal | Archives of Disease in Childhood |
DOIs | |
Publication status | Published - 15 Jan 2025 |