Effect of Grass Sublingual Tablet Immunotherapy is Similar in Children and Adults: A Bayesian Approach To Design Pediatric Sublingual Immunotherapy Trials

Publication date: Available online 5 December 2017
Source:Journal of Allergy and Clinical Immunology
Author(s): Amarjot Kaur, David Skoner, Joseph Ibrahim, Qing Li, Richard F. Lockey, Michael Blaiss, Albrecht Bufe, Jens Strodl Andersen, Giorgio Walter Canonica, Hendrik Nolte
BackgroundLarge sample sizes are needed for sublingual immunotherapy (SLIT) trials due to inherent data variability secondary to inconsistent allergen exposure. Obtaining large sample sizes for pediatric SLIT trials is challenging, but a Bayesian approach using prior adult data can reduce the necessary sample size.ObjectiveTo describe how a Bayesian framework utilizing prior information from adult trials can be used to improve pediatric SLIT clinical development.MethodsData were compiled using a frequentist approach (conventional clinical trial approach independent of prior data) from trials conducted during the clinical development of timothy grass SLIT-tablet.ResultsThe treatment effect of timothy grass SLIT-tablet was considered similar between pediatric (N=795) and adult (N=2299) data pools, with relative total combined symptom plus medication score improvement (95% CI) vs placebo of 21% (11.0, 30.4) and 20% (14.6, 24.4), respectively. Phleum pratense-specific IgG4 and IgE blocking factor increased from baseline in both children and adults treated with timothy grass SLIT-tablet. Given the reasonable assumption in similarity of treatment response between adults and children, a Bayesian approach is described to demonstrate rigorous efficacy criterion for pediatric trials incorporating information from prior adult trials, and thereby reduce the sample size.ConclusionsData support the similarity of efficacy and immunologic changes between children and adults treated with SLIT for allergic rhinoconjunctivitis. Therefore, it is appropriate to utilize data from adult trials to design feasible trials in children, which may reduce unsafe off-label use by promoting more quickly proper labeling of approved products.

Teaser

Capsule Summary SLIT effects are similar between children and adults. Analytical approaches that allow borrowing information from adult data are appropriate and will make future pediatric SLIT trials more feasible and therefore, quicker to complete, possibly reducing unsafe off-label use of non-approved SLIT formulations.


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