CommentBiologics for paediatric severe asthma: trick or TREAT?
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Future of biologics in pediatric asthma: Optimizing response, early introduction, and equitable access to treatment
2024, Annals of Allergy, Asthma and ImmunologyUnanswered questions on the use of biologics in pediatric asthma
2023, World Allergy Organization JournalDesign and analysis features used in small population and rare disease trials: A targeted review
2022, Journal of Clinical EpidemiologyCitation Excerpt :Rare disease trials face design challenges due to highly restricted recruitment. Participants to trials may also be limited if the focus is for a highly specialized sub-group; for example, a subset of asthmatic patients that do not respond to established therapeutic strategies [1] or trials where intervention was prohibitive, such as with expensive treatments or surgeries. With traditional parallel arm randomized controlled trials conducted in a frequentist framework, the required sample size is calculated through: desired power, significance level (type I error), and the minimal clinically important difference (MCID).
Difficult-to-Treat Asthma Management in School-Age Children
2022, Journal of Allergy and Clinical Immunology: In PracticeCitation Excerpt :In children, in whom there is little evidence of an association between blood and airway eosinophilic inflammation, however, the optimal cutoff may be different and may affect efficacy. This discrepancy and extrapolation from adult studies highlights the need for childhood studies that both assess the efficacy of anti-IL5 therapies and define pediatric-specific biomarkers of response.34 An important role of sputum eosinophils is to enable a distinction between the clinical phenotypes of difficult-to-treat asthma and true STRA.
Prevention and Outpatient Treatment of Asthma Exacerbations in Children
2021, Journal of Allergy and Clinical Immunology: In Practice