Hatter Lee, Bruce Pepa, Holliday Mark, Anderson Augustus J, Braithwaite Irene, Corin Andrew, Eathorne Allie, Grimes Arthur, Harwood Matire, Hills Thomas, Kearns Ciléin, Kerse Kyley, Martindale John, Montgomery Barney, Riggs Lynn, Sheahan Davitt, Shortt Nick, Zazulia Katja, Weatherall Mark, McNamara David, Byrnes Catherine A, Bush Andrew, Dalziel Stuart R, Beasley Richard
Medical Research Institute of New Zealand, Wellington, New Zealand.
National Heart and Lung Institute, Imperial College London, London, UK.
ERJ Open Res. 2021 Oct 11;7(4). doi: 10.1183/23120541.00271-2021. eCollection 2021 Oct.
Asthma is the most common chronic disease in children, many of whom are managed solely with a short-acting β-agonist (SABA). In adults, the evidence that budesonide-formoterol as sole reliever therapy markedly reduces the risk of severe exacerbations compared with SABA alone has contributed to the Global Initiative for Asthma recommending against SABA monotherapy in this population. The current lack of evidence in children means it is unknown whether these findings are also relevant to this demographic. High-quality randomised controlled trials (RCTs) are needed.
The aim of this study is to determine the efficacy and safety of as-needed budesonide-formoterol therapy compared with as-needed salbutamol in children aged 5 to 15 years with mild asthma, who only use a SABA.
A 52-week, open-label, parallel group, phase III RCT will recruit 380 children aged 5 to 15 years with mild asthma. Participants will be randomised 1:1 to either budesonide-formoterol (Symbicort Rapihaler) 50/3 µg, two actuations as needed, or salbutamol (Ventolin) 100 µg, two actuations as needed. The primary outcome is asthma attacks as rate per participant per year. Secondary outcomes assess asthma control, lung function, exhaled nitric oxide and treatment step change. A cost-effectiveness analysis is also planned.
This is the first RCT to assess the safety and efficacy of as-needed budesonide-formoterol in children with mild asthma. The results will provide a much-needed evidence base for the treatment of mild asthma in children.
哮喘是儿童中最常见的慢性疾病，其中许多儿童仅使用短效β受体激动剂（SABA）进行治疗。在成人中，与单独使用SABA相比，布地奈德-福莫特罗作为单一缓解药物疗法能显著降低严重加重风险的证据，促使全球哮喘防治创议建议在该人群中不采用SABA单药治疗。目前儿童中缺乏相关证据，意味着尚不清楚这些研究结果是否也适用于这一人群。因此需要高质量的随机对照试验（RCT）。
本研究旨在确定按需使用布地奈德-福莫特罗疗法与按需使用沙丁胺醇相比，对仅使用SABA的5至15岁轻度哮喘儿童的疗效和安全性。
一项为期52周、开放标签、平行组、III期RCT将招募380名5至15岁的轻度哮喘儿童。参与者将按1:1随机分组，分别按需使用50/3μg布地奈德-福莫特罗（信必可都保）两次喷压，或按需使用100μg沙丁胺醇（万托林）两次喷压。主要结局是每位参与者每年的哮喘发作率。次要结局评估哮喘控制情况、肺功能、呼出一氧化氮和治疗步骤变化。还计划进行成本效益分析。
这是第一项评估按需使用布地奈德-福莫特罗对轻度哮喘儿童安全性和疗效的RCT。研究结果将为儿童轻度哮喘的治疗提供急需的证据基础。
