ADRB2 genotype-guided treatment for childhood asthma: Cost analysis of the PUFFIN and PACT trials

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Abstract

Background: Long-acting β2-agonists (LABA) are commonly used to treat asthma. Some children do not respond well to LABA, which may be due to +46G>A−/rs1042713 (Arg16 amino acid) in the ADRB2 gene encoding the β2 receptor. Arg16Gly ADRB2 genotyping to guide treatment step-up decisions in children with uncontrolled asthma despite inhaled corticosteroids (ICS) has been shown to reduce asthma exacerbations. We investigated whether ADRB2 genotype-guided treatment is cost-saving. Methods: Total semi-annual healthcare and indirect costs for children with and without exacerbations were calculated using PUFFIN trial data. One hundred and two Dutch and Swiss children were randomised to a genotype-guided treatment arm (adding LABA [Gly16Gly] or double dose ICS [Arg16Arg/Arg16Gly]) or a control arm, where children were again randomised to LABA or double dose ICS. We used exacerbation rates of the PUFFIN and the PACT trials to calculate asthma-related healthcare costs per treatment arm, as PACT closely matches the PUFFIN design. The PACT trial randomised 91 children from England and Scotland with uncontrolled asthma to the genotype-guided treatment arm (LABA [Gly16Gly] or montelukast [Arg16Arg/Arg16Gly]) or the control arm (routine care as per British Thoracic Society guidelines). Results: Overall mean semi-annual costs per child were €56.24 lower in the genotype-guided treatment arm compared to the control arm (€771.07 [range €616.86–€925.28, 23 of 90 children experienced exacerbations] and €827.31 [range €661.85–€992.77, 40 of 103 experienced exacerbations], respectively). Conclusion: A treatment strategy that includes ADRB2 genotype-guided treatment is potentially cost-saving compared to usual care. The decreased healthcare costs associated with a reduction in asthma exacerbations more than offset the incurred genotyping costs.

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Slob, E. M. A., Frederix, G. W. J., Vijverberg, S. J. H., Noij, L. C. E., Ruffles, T., Vasbinder, E. C., … Maitland-van der Zee, A. H. (2025). ADRB2 genotype-guided treatment for childhood asthma: Cost analysis of the PUFFIN and PACT trials. Pediatric Allergy and Immunology, 36(6). https://doi.org/10.1111/pai.70113

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