BEAT CF platform: A digital infrastructure and data collection tool to evaluate treatments for pulmonary exacerbations in children and adults with Cystic Fibrosis.

The BEAT CF platform is a data collection instrument that will collect health and medical data from consented participants’ medical records. There are no specified interventions or exposures for participants who enrol in the BEAT CF platform. All information that is entered into the BEAT CF platform database - both treatment and diagnostic - is gathered as a part of standard clinical care for these patients. Participants will be requested to partake in completion of quality of life questionnaires in accordance with their regular clinic visit schedule, which is expected to be approximately every 3 months. The quality of life data is in addition to data gathered from routine standard clinical care. Patients with Cystic fibrosis commonly experience Pulmonary exacerbations, or flare-ups of the lung disease. These exacerbations or flare ups often require hospitalisation for intensive treatment including with intravenous antibiotics. Pulmonary exacerbations remain an important driver of progressive loss of lung function and premature death. Up to 25% of CF patients do not recover their baseline lung function, typically measured as the forced expiratory volume in one second (FEV1), after an exacerbation. Preventing loss of lung function with each exacerbation may be key to improving survival. Antibiotics are a cornerstone of treatment for pulmonary exacerbations, but most antibiotic regimens are only informed by old, underpowered, or poor-quality trials. There is no consensus on the treatment of pulmonary exacerbations of CF. Across Australia, CF centres use a range of approaches and antibiotic regimens. Because preservation of lung function is important for extending life and quality of life, there is a need to determine the most effective empirical treatments of exacerbations. As part of the BEAT CF platform, information about any intravenous antibiotics and other treatments like steroids or disease modulators will be captured, in order to describe the treatment of CF pulmonary exacerbations, their outcomes, as well as any clinical, demographic, and other lifestyle factors which might also influence these outcomes. All data relating to treatments and outcomes of exacerbations required to be entered into the BEAT CF platform will be sourced from participant's medical records. Participants in the BEAT CF platform will be requested to complete a second type of quality of life questionnaire at the time of any hospitalisation for treatment of pulmonary exacerbations. This questionnaire, called the CRISS, is validated for participants aged 12 years and over. There will be no experimental treatments administered as part of the BEAT CF platform. Across Australia, CF centres use a range of approaches and antibiotic regimens. All participants will be treated according to their centre’s standard of care. The BEAT CF platform provides the central infrastructure to simultaneously evaluate the range of treatment strategies used in CF across Australia. The BEAT CF platform will establish effective and accurate collection of data to enable the future nesting of platform clinical trial(s). The BEAT CF platform is intended to continue perpetually. Participants who enrol in the BEAT CF platform will continue to contribute data to the platform unless they specifically withdraw their consent to participation.