Department of Paediatric Pulmonology, Maastricht University Medical Centre, Maastricht, The Netherlands.
Department of Paediatric Pulmonology and Pediatric Allergology, Beatrix Children's Hospital, and GRIAC Research Institute, University Medical Centre Groningen, University of Groningen, Groningen, The Netherlands.
BMC Public Health. 2023 Apr 3;23(1):629. doi: 10.1186/s12889-023-15465-6.
The prevalence of asthma-like symptoms in preschool children is high. Despite numerous efforts, there still is no clinically available diagnostic tool to discriminate asthmatic children from children with transient wheeze at preschool age. This leads to potential overtreatment of children outgrowing their symptoms, and to potential undertreatment of children who turn out to have asthma. Our research group developed a breath test (using GC-tof-MS for VOC-analysis in exhaled breath) that is able to predict a diagnosis of asthma at preschool age. The ADEM2 study assesses the improvement in health gain and costs of care with the application of this breath test in wheezing preschool children.
This study is a combination of a multi-centre, parallel group, two arm, randomised controlled trial and a multi-centre longitudinal observational cohort study. The preschool children randomised into the treatment arm of the RCT receive a probability diagnosis (and corresponding treatment recommendations) of either asthma or transient wheeze based on the exhaled breath test. Children in the usual care arm do not receive a probability diagnosis. Participants are longitudinally followed up until the age of 6 years. The primary outcome is disease control after 1 and 2 years of follow-up. Participants of the RCT, together with a group of healthy preschool children, also contribute to the parallel observational cohort study developed to assess the validity of alternative VOC-sensing techniques and to explore numerous other potential discriminating biological parameters (such as allergic sensitisation, immunological markers, epigenetics, transcriptomics, microbiomics) and the subsequent identification of underlying disease pathways and relation to the discriminative VOCs in exhaled breath.
The potential societal and clinical impact of the diagnostic tool for wheezing preschool children is substantial. By means of the breath test, it will become possible to deliver customized and high qualitative care to the large group of vulnerable preschool children with asthma-like symptoms. By applying a multi-omics approach to an extensive set of biological parameters we aim to explore (new) pathogenic mechanisms in the early development of asthma, creating potentially interesting targets for the development of new therapies.
Netherlands Trial Register, NL7336, Date registered 11-10-2018.
学龄前儿童哮喘样症状的患病率很高。尽管已经做出了许多努力，但目前仍然没有临床可用的诊断工具来区分学龄前哮喘儿童和一过性喘息儿童。这导致了对症状消退的儿童的过度治疗，以及对可能患有哮喘的儿童的潜在治疗不足。我们的研究小组开发了一种呼吸测试（使用 GC-tof-MS 进行呼气中挥发性有机化合物分析），能够预测学龄前儿童的哮喘诊断。ADEM2 研究评估了在喘息学龄前儿童中应用这种呼吸测试在改善健康获益和降低医疗成本方面的效果。
这是一项多中心、平行分组、两臂、随机对照试验和多中心纵向观察队列研究的组合。随机分配到 RCT 治疗组的学龄前儿童根据呼气测试接受哮喘或一过性喘息的概率诊断（和相应的治疗建议）。常规护理组的儿童不接受概率诊断。参与者进行纵向随访，直到 6 岁。主要结局是随访 1 年和 2 年后的疾病控制情况。RCT 参与者与一组健康的学龄前儿童一起参与了平行观察队列研究，该研究旨在评估替代 VOC 感应技术的有效性，并探索其他许多潜在的有区别的生物参数（如过敏致敏、免疫标志物、表观遗传学、转录组学、微生物组学），以及随后确定潜在的疾病途径与呼气中区别性 VOC 的关系。
这种用于学龄前喘息儿童的诊断工具具有重要的社会和临床影响。通过呼吸测试，将有可能为大量有哮喘样症状的脆弱学龄前儿童提供个性化和高质量的护理。通过对广泛的生物参数进行多组学分析，我们旨在探索哮喘早期发展中的（新）发病机制，为新疗法的开发创造潜在的有趣靶点。
荷兰试验注册处，NL7336，注册日期 2018 年 10 月 11 日。
