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.