PULSE-CF: Precision in Understanding and Preventing Exacerbations in Cystic Fibrosis - Trial Platform

People with CF frequently experience ‘exacerbations’, where they become more unwell from underlying lung infections. They may need to go to hospital for antibiotic treatment directly into a vein. Exacerbations disrupt normal life and damage the lungs, the infections can be life-threatening, and treatment may also have side effects. Preventing exacerbations and understanding what triggers them is therefore a priority of CF care. However, little is known about what actually causes exacerbations nor therefore to best prevent them. For example, we think that viruses like common colds frequently contribute, but these interact with other factors that are less well understood. To address this, we will monitor a group of people with CF when they are well and as they start to become unwell to get more evidence. We will ask 200 adults to monitor their lung function, symptoms and viruses they catch by posting samples back to us. They will do this every 2 weeks when well, and to make this easy to manage they will do this from home. We will also ask 100 additional CF adults at three centres for some extra blood, sputum and nasal samples, so we can monitor changes in these in response to infections. Once we know more about the causes of exacerbations, we will use this information to work with industry partners to design and run a clinical trial to prevent exacerbations, helping people with CF keep well. This addresses JLA CF research priorities (https://www.jla.nihr.ac.uk/priority-setting-partnerships/cystic-fibrosis-refresh/top-10-priorities.htm) two (diagnosing lung infections) and five (reducing antibiotics).