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Effectiveness of pharmacotherapy in asthmatic preschool children
J C de Jongste1, H M Janssens, J Van der Wouden
1Department of Pediatrics, Division Pediatric Respiratory Medicine, Erasmus University and University Hospital/Sophia Children's Hospital, Rotterdam, The Netherlands.
Insights
Treatment effectiveness in young children with asthma differs from efficacy shown in trials. Re-evaluating inhaled asthma treatment guidelines for preschoolers is crucial, considering real-world factors and oral alternatives.
Area of Science:
- Pediatric Pulmonology
- Clinical Pharmacology
- Asthma Management
Background:
- Treatment efficacy is demonstrated in controlled trials, but real-world effectiveness can differ significantly.
- Asthma management in young children presents unique challenges impacting treatment outcomes.
- Factors influencing effectiveness include patient heterogeneity, treatment compliance, and device performance.
Purpose of the Study:
- To highlight the discrepancy between asthma treatment efficacy and effectiveness in preschool children.
- To identify key factors contributing to this difference in daily practice.
- To advocate for a re-evaluation of current asthma treatment guidelines for this age group.
Main Methods:
- Analysis of factors affecting asthma treatment effectiveness in young children.
- Comparison of controlled trial efficacy with real-world application.
- Review of current treatment guidelines for recurrent wheeze in preschoolers.
Main Results:
- Wheezing phenotype heterogeneity, compliance issues, and inhaler device limitations impact treatment effectiveness.
- Current guidelines may overestimate the effectiveness and feasibility of inhaled asthma treatments for all young children.
- A gap exists between optimal therapeutic action in trials and practical application in daily life.
Conclusions:
- Re-evaluation of asthma treatment recommendations for preschool children in real-world settings is necessary.
- Consideration of oral treatment options alongside inhaled therapies is warranted.
- Developing methods to differentiate pediatric asthma phenotypes is essential for targeted and optimized treatment.
Abstract:
The term "effectiveness" relates to the question of whether or not a certain treatment works in practice. Usually, such a treatment was first evaluated under tightly controlled conditions in selected patient populations, and the potential benefits were shown. There is, however, a great difference between the efficacy of a given treatment, indicating its optimal therapeutic action in controlled trials, and its effectiveness when applied to a less well-defined population of patients in daily practice. This is especially relevant for asthma in young children, where many factors are responsible for the difference. Among these are, first of all, the heterogeneity of the wheezing phenotype. Other factors include the compliance with prescribed treatments, as determined by the attitude of doctors and parents towards such treatment, the ease of administration and the perceived effects and side effects. Also, the performance of different inhaler devices may be insufficient for a good, reliable dose deposition in young children in daily life. As a result, the current treatment guidelines for preschool children with recurrent wheeze are probably too optimistic in assuming that inhaled treatment is most effective and feasible at all ages. We propose careful re-evaluation of such recommendations in a first-line setting resembling daily life as closely as possible, and consideration of oral treatments as well. Also, we need methods to separate the different phenotypes within the group of recurrently wheezing preschool children to optimize targeting of asthma treatment to those who have ongoing airway inflammation.