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Defining Treatment Response for Clinical Trials of Pediatric Acute Asthma
Nidhya Navanandan1, Talia Thompson2, Laura Pyle3
1Section of Emergency Medicine, Children's Hospital Colorado, Department of Pediatrics, University of Colorado School of Medicine, Aurora, Colo.
Insights
A consensus definition for pediatric acute asthma treatment response was developed. This standardized approach will improve clinical trial comparisons and advance asthma management for children.
Area of Science:
- Pediatric Pulmonology
- Clinical Trial Methodology
- Asthma Research
Background:
- Lack of a standardized definition for treatment response in pediatric acute asthma clinical trials hinders meaningful comparisons.
- This deficiency limits the evaluation of therapeutic interventions and progress in asthma management.
Purpose of the Study:
- To establish a consensus definition for treatment response in clinical trials involving pediatric acute asthma.
Main Methods:
- A modified Delphi process involving 22 multidisciplinary experts was employed.
- Consensus was achieved through iterative rounds of ranking and reconsidering measures of treatment response.
- Consensus was defined by 70% agreement and an interquartile range less than 2.
Main Results:
- A definition of treatment response was developed, incorporating Clinical Severity Score, need for additional therapies, and hospitalization criteria.
- Clinical Severity Score, encompassing respiratory distress and wheeze, was identified as a key component.
- Consensus was reached on the top 10 criteria for classifying hospitalization needs.
Conclusions:
- The developed consensus definition provides a standardized method for outcome measurement in pediatric acute asthma clinical trials.
- This standardization will enable more meaningful reporting of outcomes and facilitate advancements in asthma care for children.
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.
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