Slob E M A, Frederix G W J, Vijverberg S J H, Noij L C E, Ruffles T, Vasbinder E C, Mukhopadhyay S, van Schaik R H N, Kersten E T G, Pijnenburg M W, Koppelman G H, Maitland-van der Zee A H
Department of Pulmonary Medicine, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.
Department of Genetics, University Medical Center Groningen, Groningen, The Netherlands.
Pediatr Allergy Immunol. 2025 Jun;36(6):e70113. doi: 10.1111/pai.70113.
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.
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).
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).
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.
长效β2受体激动剂（LABA）常用于治疗哮喘。一些儿童对LABA反应不佳，这可能归因于编码β2受体的ADRB2基因中的+46G>A - /rs1042713（精氨酸16位氨基酸）。对于尽管使用吸入性糖皮质激素（ICS）但哮喘仍未得到控制的儿童，进行精氨酸16位甘氨酸ADRB2基因分型以指导治疗升级决策已被证明可减少哮喘发作。我们调查了ADRB2基因分型指导的治疗是否具有成本效益。
利用PUFFIN试验数据计算有和无哮喘发作儿童的半年总医疗保健和间接成本。102名荷兰和瑞士儿童被随机分为基因分型指导治疗组（加用LABA[甘氨酸16位甘氨酸]或双倍剂量ICS[精氨酸16位精氨酸/精氨酸16位甘氨酸]）或对照组，对照组儿童再随机分为LABA组或双倍剂量ICS组。我们使用PUFFIN试验和PACT试验的发作率来计算每个治疗组与哮喘相关的医疗保健成本，因为PACT试验设计与PUFFIN试验紧密匹配。PACT试验将91名来自英格兰和苏格兰且哮喘未得到控制的儿童随机分为基因分型指导治疗组（LABA[甘氨酸16位甘氨酸]或孟鲁司特[精氨酸16位精氨酸/精氨酸16位甘氨酸]）或对照组（按照英国胸科学会指南进行常规治疗）。
与对照组相比，基因分型指导治疗组每名儿童的半年平均成本总体低56.24欧元（分别为771.07欧元[范围616.86 - 925.28欧元，90名儿童中有23名经历发作]和827.31欧元[范围661.85 - 992.77欧元，103名儿童中有40名经历发作]）。
与常规治疗相比，包含ADRB2基因分型指导治疗的策略可能具有成本效益。与哮喘发作减少相关的医疗保健成本降低超过了基因分型产生的成本。
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