E M A Slob, G W J Frederix, S J H Vijverberg, L C E Noij, T Ruffles, E C Vasbinder, S Mukhopadhyay, R H N van Schaik, E T G Kersten, M W Pijnenburg, G H Koppelman, A H Maitland-van der Zee
{"title":"ADRB2基因型引导治疗儿童哮喘：PUFFIN和PACT试验的成本分析","authors":"E M A Slob, G W J Frederix, S J H Vijverberg, L C E Noij, T Ruffles, E C Vasbinder, S Mukhopadhyay, R H N van Schaik, E T G Kersten, M W Pijnenburg, G H Koppelman, A H Maitland-van der Zee","doi":"10.1111/pai.70113","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>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).</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":520742,"journal":{"name":"Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology","volume":"36 6","pages":"e70113"},"PeriodicalIF":4.5000,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12134971/pdf/","citationCount":"0","resultStr":"{\"title\":\"ADRB2 genotype-guided treatment for childhood asthma: Cost analysis of the PUFFIN and PACT trials.\",\"authors\":\"E M A Slob, G W J Frederix, S J H Vijverberg, L C E Noij, T Ruffles, E C Vasbinder, S Mukhopadhyay, R H N van Schaik, E T G Kersten, M W Pijnenburg, G H Koppelman, A H Maitland-van der Zee\",\"doi\":\"10.1111/pai.70113\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>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).</p><p><strong>Conclusion: </strong>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.</p>\",\"PeriodicalId\":520742,\"journal\":{\"name\":\"Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology\",\"volume\":\"36 6\",\"pages\":\"e70113\"},\"PeriodicalIF\":4.5000,\"publicationDate\":\"2025-06-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12134971/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1111/pai.70113\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1111/pai.70113","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
ADRB2 genotype-guided treatment for childhood asthma: Cost analysis of the PUFFIN and PACT trials.
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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