Challenges and limitations of testing efficacy of aerosol device delivery in young children.
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.
1 Division of Pulmonary and Critical Care Medicine, The University of North Carolina at Chapel Hill , Chapel Hill, NC.
Jennifer L
JL
Goralski
Stephanie D
SD
Davis
D000280:Administration, Inhalation; D000336:Aerosols; D000367:Age Factors; D001944:Breath Tests; D001999:Bronchoscopy; D002648:Child; D002675:Child, Preschool; D003952:Diagnostic Imaging; D016503:Drug Delivery Systems / Q000295:instrumentation*; D023381:Endpoint Determination; D004867:Equipment Design; D006801:Humans; D007223:Infant; D008168:Lung / Q000473:pathology / Q000503:physiopathology; D008422:Materials Testing; D009079:Mucociliary Clearance; D009330:Nebulizers and Vaporizers; D009991:Oscillometry; D004364:Pharmaceutical Preparations / Q000008:administration & dosage*; D011237:Predictive Value of Tests; D012107:Research Design; D012129:Respiratory Function Tests
D000336:Aerosols; D004364:Pharmaceutical Preparations
cystic fibrosis; inhaled therapy; lung function; mucociliary clearance