Dobra Rebecca, Bentley Siân, Edmondson Claire, Ovens Maxine, Saunders Clare, Short Christopher, Wilson Gemma, Davies Jane C, Bush Andrew
National Heart and Lung Institute, Imperial College, London SW3 6LY, UK.
Paediatric Respiratory Medicine Department, Royal Brompton Hospital, London SW3 6NP, UK.
Children (Basel). 2022 Jul 20;9(7):1080. doi: 10.3390/children9071080.
This is an exciting time for research and novel drug development in cystic fibrosis. However, rarely has the adage, "Children are not just little adults" been more relevant. This article is divided into two main sections. In the first, we explore why it is important to involve children in research. We discuss the potential benefits of understanding a disease and its treatment in children, and we highlight that children have the same legal and ethical right to evidence-based therapy as adults. Additionally, we discuss why extrapolation from adults may be inappropriate, for example, medication pharmacokinetics may be different in children, and there may be unpredictable adverse effects. In the second part, we discuss how to involve children and their families in research. We outline the importance and the complexities of selecting appropriate outcome measures, and we discuss the role co-design may have in improving the involvement of children. We highlight the importance of appropriate staffing and resourcing, and we outline some of the common challenges and possible solutions, including practical tips on obtaining consent/assent in children and adolescents. We conclude that it is unethical to simply rely on extrapolation from adult studies because research in young children is challenging and that research should be seen as a normal part of the paediatric therapeutic journey.
对于囊性纤维化的研究和新型药物开发而言，这是一个激动人心的时代。然而，“儿童并非小大人”这句格言从未像现在这样贴切。本文分为两个主要部分。第一部分，我们探讨让儿童参与研究为何重要。我们讨论了解儿童疾病及其治疗方法的潜在益处，并强调儿童与成人一样拥有接受循证治疗的同等法律和道德权利。此外，我们还讨论了为何从成人研究推断可能并不合适，例如儿童的药物药代动力学可能不同，而且可能会出现不可预测的不良反应。在第二部分，我们讨论如何让儿童及其家庭参与研究。我们概述了选择合适结局指标的重要性和复杂性，并讨论了共同设计在提高儿童参与度方面可能发挥的作用。我们强调了配备合适人员和资源的重要性，并概述了一些常见挑战及可能的解决方案，包括获取儿童和青少年同意/赞成的实用技巧。我们得出结论，仅仅依靠成人研究推断是不道德的，因为幼儿研究具有挑战性，而且研究应被视为儿科治疗过程的正常组成部分。
