背景:支气管扩张症是一种全球性的慢性肺部疾病,其加重很常见。它影响所有年龄段的人,尤其是高收入国家的土著居民。尽管是慢性肺病的主要原因,目前尚无获得许可的支气管扩张治疗方法,在儿童和成人中进行的随机对照试验(RCT)相对较少.我们的RCT将通过评估新型粘液活性剂是否满足这些未满足的需求,erdosteine,对支气管扩张的儿童和成人有治疗作用。我们的主要目的是确定2-49岁支气管扩张的儿童和成人与安慰剂相比,在12个月内定期使用埃多司坦是否可以减少急性呼吸道恶化。我们的主要假设是,经常使用erdosteine的支气管扩张患者的恶化程度比接受安慰剂的患者少。我们的次要目标是确定试验药物对生活质量(QoL)和其他临床结果的影响(恶化持续时间,下一次恶化的时间,住院治疗,肺功能,不良事件)。我们还将评估干预措施的成本效益。
方法:我们正在进行国际多中心,双盲,安慰剂-RCT评估12个月的erdosteine是否对支气管扩张的儿童和成人有益。我们将招募194名患有支气管扩张症的儿童和成人,澳大利亚八个地点的优势RCT,马来西亚和菲律宾。我们的主要终点是超过12个月的恶化率。我们的主要次要结果是QoL,恶化持续时间,下一次恶化的时间,住院和肺功能。
背景:昆士兰州儿童健康人类研究伦理委员会(HREC)(适用于所有澳大利亚网站),马来亚大学医学中心(马来西亚)和圣卢克医学中心(菲律宾)批准了这项研究。我们将公布结果,并与学术界和医学界分享结果,资金和相关患者组织。
背景:ACTRN12621000315819。
BACKGROUND: Bronchiectasis is a worldwide chronic lung disorder where exacerbations are common. It affects people of all ages, but especially Indigenous populations in high-income nations. Despite being a major contributor to chronic lung disease, there are no licensed therapies for bronchiectasis and there remain relatively few randomised controlled trials (RCTs) conducted in children and adults. Our RCT will address some of these unmet needs by evaluating whether the novel mucoactive agent, erdosteine, has a therapeutic role in children and adults with bronchiectasis.Our primary aim is to determine in children and adults aged 2-49 years with bronchiectasis whether regular erdosteine over a 12-month period reduces acute respiratory exacerbations compared with placebo. Our primary hypothesis is that people with bronchiectasis who regularly use erdosteine will have fewer exacerbations than those receiving placebo.Our secondary aims are to determine the effect of the trial medications on quality of life (QoL) and other clinical outcomes (exacerbation duration, time-to-next exacerbation, hospitalisations, lung function, adverse events). We will also assess the cost-effectiveness of the intervention.
METHODS: We are undertaking an international multicentre, double-blind, placebo-RCT to evaluate whether 12 months of erdosteine is beneficial for children and adults with bronchiectasis. We will recruit 194 children and adults with bronchiectasis to a parallel, superiority RCT at eight sites across Australia, Malaysia and Philippines. Our primary endpoint is the rate of exacerbations over 12 months. Our main secondary outcomes are QoL, exacerbation duration, time-to-next exacerbation, hospitalisations and lung function.
BACKGROUND: The Human Research Ethics Committees (HREC) of Children\'s Health Queensland (for all Australian sites), University of Malaya Medical Centre (Malaysia) and St. Luke\'s Medical Centre (Philippines) approved the study. We will publish the results and share the outcomes with the academic and medical community, funding and relevant patient organisations.
BACKGROUND: ACTRN12621000315819.