Division of Infection, Immunity & Respiratory Medicine, Manchester University.
Manchester Adult Cystic Fibrosis Centre, Manchester University Hospitals NHS Foundation Trust, Manchester.
Curr Opin Pulm Med. 2020 Nov;26(6):671-678. doi: 10.1097/MCP.0000000000000732.
Early stage lung disease has long been synonymous with infancy and childhood. As diagnosis happens earlier and conventional management improves, we are seeing larger proportions of people with cystic fibrosis (CF) in adolescence and even adulthood with well preserved lung health. The availability of highly effective cystic fibrosis transmembrane conductance regulator modulator drugs for a large proportion of the CF population will impact even further. Transitioning into adult care with 'normal' lung function will become more common. However, it is crucial that we are not blasé about this phase, which sets the scene for future lung health. It is well recognized that lung function assessed by spirometry is insensitive to 'early' changes occurring in the distal, small airways. Much of our learning has come from studies in infants and young children, which have allowed assessment and optimization of alternative forms of monitoring.
Here, as a group of paediatric and adult CF specialists, we review the evidence base for sensitive physiological testing based on multibreath washout, lung imaging, exercise and activity monitoring, assessment of infection and quality of life measures.
We seek to emphasise the importance of further work in these areas, as outcome measures become widely applicable to a growing CF population.
早期肺部疾病长期以来一直是婴儿和儿童的代名词。随着诊断的提前和常规治疗的改善，我们看到越来越多的囊性纤维化（CF）患者处于青少年甚至成年期，且肺部健康状况良好。对于大部分 CF 患者而言，高有效性的囊性纤维化跨膜电导调节体调节剂药物的出现将会产生更大的影响。随着“正常”肺功能向成人护理的过渡将会更加常见。然而，我们对于这一阶段必须保持重视，因为它为未来的肺部健康奠定了基础。人们已经充分认识到，通过肺活量测定法评估的肺功能对远端小气道中发生的“早期”变化不敏感。我们的大部分知识来自于对婴儿和幼儿的研究，这些研究允许评估和优化替代形式的监测。
在这里，作为一组儿科和成人 CF 专家，我们回顾了基于多呼吸冲洗、肺部成像、运动和活动监测、感染评估以及生活质量措施的敏感生理测试的证据基础。
我们试图强调在这些领域进一步开展工作的重要性，因为随着 CF 患者群体的不断增长，这些结果衡量标准将会得到更广泛的应用。
