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Challenges and limitations of testing efficacy of aerosol device delivery in young children
Jennifer L Goralski1, Stephanie D Davis
11 Division of Pulmonary and Critical Care Medicine, The University of North Carolina at Chapel Hill , Chapel Hill, NC.
Insights
Selecting clinical endpoints for aerosolized therapy trials in children is challenging. This review explores pulmonary function tests, imaging, and other methods to assess treatment efficacy in infants and preschoolers.
Area of Science:
- Pediatric Pulmonology
- Respiratory Drug Delivery
- Clinical Trial Design
Background:
- Aerosolized therapy is increasingly used for various medical conditions.
- Direct drug delivery to the lungs offers therapeutic advantages.
- Evaluating new aerosolized drug-device combinations, especially in children, faces challenges in selecting appropriate outcome measures.
Purpose of the Study:
- To review the challenges and limitations in selecting clinical endpoints for drug-device trials in pediatric populations.
- To focus on novel emerging technologies for assessing treatment efficacy in young children.
- To provide an overview of various assessment methods suitable for infants and preschoolers.
Main Methods:
- Review of existing literature on pediatric pulmonary function testing.
- Discussion of advanced imaging techniques like CT and MRI.
- Exploration of flexible bronchoscopy, mucociliary clearance scans, and exhaled breath condensate analysis.
Main Results:
- Traditional adult endpoints like airflow obstruction are often unsuitable for young children.
- Sensitive, noninvasive, and easily performed endpoints are lacking for pediatric trials.
- Novel technologies offer potential solutions for efficacy assessment in infants and preschoolers.
Conclusions:
- Choosing appropriate clinical endpoints is critical for the successful evaluation of aerosolized therapies in pediatric populations.
- Emerging technologies provide promising avenues for overcoming current assessment limitations.
- Further research and standardization of endpoints are needed for pediatric drug-device trials.
Abstract:
An increasing number of medical conditions are chronically or acutely managed with some form of aerosolized therapy. Due to the benefit of directly administering medications to the intended site of action, there is great interest in evaluating treatments for aerosol use. One of the major challenges in selecting and testing new drug-device combinations in children is the uncertainty regarding the appropriate outcome measure to choose. In studies involving adult patients, typically exacerbations of disease or airflow obstruction are assessed as endpoints in drug trials or device assessment. However, in young children, choosing endpoints to assess efficacy is difficult due to the potential lack of sensitive, noninvasive endpoints that are easily performed across sites. In this review, we discuss the challenges and limitations of selecting clinical endpoints for drug-device trials in the youngest population, with a focus on novel emerging technologies. This article provides an overview of preschool and infant pulmonary function testing, multiple-breath washout, imaging techniques including computed tomography and magnetic resonance imaging, flexible bronchoscopy in children, mucociliary clearance scans, and exhaled breath condensate.
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