Department of Pediatrics, Azienda Ospedaliero-Universitaria Ospedali Riuniti, Ancona, Italy.
Eur J Clin Pharmacol. 2011 May;67 Suppl 1:49-59. doi: 10.1007/s00228-010-0922-2. Epub 2010 Nov 23.
A wide range of outcome measures or endpoints has been used in clinical trials to assess the effects of treatments in paediatric respiratory diseases. This can make it difficult to compare treatment outcomes from different trials and also to understand whether new treatments offer a real clinical benefit for patients. Clinical trials in respiratory diseases evaluate three types of endpoints: subjective, objective and health-related outcomes. The ideal endpoint in a clinical trial needs to be accurate, precise and reliable. Ideally, the endpoint would also be measured with minimal risk and across all ages, easy to perform, and be inexpensive. As for any other disease, endpoints for respiratory diseases must be viewed in the context of the important distinction between clinical endpoints and surrogate endpoints. The association between surrogate endpoints and clinical endpoints must be clearly defined for any disease in order for them to be meaningful as outcome measures. The most common endpoints which are used in paediatric trials in respiratory diseases are discussed. For practical purposes, diseases have been separated into acute (bronchiolitis, acute viral-wheeze, acute asthma and croup) and chronic (asthma and cystic fibrosis). Further development of endpoints will enable clinical trials in children with respiratory diseases with the main objective of improving prognosis and safety.
在儿科呼吸疾病的临床试验中，广泛使用了多种结局指标或终点来评估治疗效果。这使得比较来自不同试验的治疗结果变得困难，也难以理解新的治疗方法是否为患者带来真正的临床获益。呼吸疾病的临床试验评估了三种类型的终点：主观、客观和与健康相关的结局。临床试验中的理想终点需要准确、精确和可靠。理想情况下，该终点还应具有最小风险，且适用于所有年龄段，易于实施，并且价格低廉。与其他任何疾病一样，呼吸疾病的终点必须在临床终点和替代终点之间的重要区别的背景下进行观察。为了使替代终点有意义，必须在任何疾病中明确定义替代终点与临床终点之间的关联。本文讨论了儿科呼吸疾病临床试验中最常用的终点。出于实际目的，疾病已分为急性（细支气管炎、急性病毒性喘息、急性哮喘和喉炎）和慢性（哮喘和囊性纤维化）。进一步开发终点将使儿童呼吸疾病的临床试验能够以改善预后和安全性为主要目标。
