Defining Treatment Response for Clinical Trials of Pediatric Acute Asthma

Nidhya Navanandan*, Talia Thompson, Laura Pyle, Todd A. Florin

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


Background: An agreed-upon definition of treatment response for clinical trials of pediatric acute asthma does not exist, limiting meaningful comparisons among therapeutic interventions and advances in asthma management. Objective: To develop a consensus definition of treatment response for clinical trials of pediatric acute asthma. Methods: A multidisciplinary panel of 22 experts participated in a Web-based modified Delphi process to achieve consensus on a definition of treatment response. Round 1 consisted of closed- and open-ended questions in which panelists ranked measures of treatment response developed by literature review, suggested additional measures, and explained their responses. In rounds 2 and 3, panelists reviewed summary statistics of the panel's rating from prior rounds and reconsidered their rankings. In round 3, pairwise ranking was performed to determine the ranked importance of components. Consensus was defined as 70% or greater agreement among panelists choosing Likert-scale values of 1 to 6 (extremely unimportant to extremely important) and an interquartile range less than 2. Results: Drawing on results from the expert panel, we developed a definition of treatment response that includes Clinical Severity Score, need for additional therapies, and hospitalization. Clinical Severity Score encompassed most ranked criteria (eg, respiratory distress, wheeze) for a treatment response definition. Panelists recommended that a valid and pragmatic severity score be used consistently across institutions. Panelists also achieved consensus on the top 10 criteria that appropriately classify need for hospitalization. Conclusions: This consensus definition of treatment response can be used in clinical trials of children with acute asthma to standardize outcome measurement and report meaningful outcomes.

Original languageEnglish (US)
Pages (from-to)1450-1458.e1
JournalJournal of Allergy and Clinical Immunology: In Practice
Issue number5
StatePublished - May 2023


  • Asthma
  • Asthma exacerbation
  • Children
  • Consensus
  • Delphi
  • Pediatric
  • Response
  • Treatment

ASJC Scopus subject areas

  • Immunology and Allergy


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