BEAT-CF (Bayesian Evidence Adaptive Treatment for people with Cystic Fibrosis): description of a prospective cohort for nested studies in cystic fibrosis.

Abstract

Background: Despite recent improvements in treatment modalities for cystic fibrosis (CF), there is currently limited evidence and a lack of consensus regarding optimal treatment strategies for the different aspects of CF, including pulmonary exacerbations (PEx). We aimed to establish a prospective cohort of people with CF (pwCF) to evaluate alternative approaches to managing CF in the era of modulator therapies.

Methods: We prospectively enrolled children and adults with CF receiving care at specialist CF centres across Australia. Participant data were systematically collected on demography, clinical signs and symptoms, comorbidities, spirometry, participant reported outcomes, microbiology and treatments received. Here we describe the demographic, microbiological and clinical characteristics of the participants at enrolment, to understand the representativeness of the cohort for planning future nested studies.

Results: Between 14 October 2020 and 31 December 2023, 927 pwCF were enrolled across eleven Australian CF centres. Of these, 51% (n=472) were male, 77% (n=709) were <18 years old, 88% (n=811) had a highest ppFEV1 (percent predicted forced expiratory volume exhaled in the first second) of ≥70% in the preceding year, and 35% (n=322) reported detection of Pseudomonas aeruginosa in their airway specimens.

Conclusions: We have established a contemporary cohort of pwCF with granular clinical and treatment data for PEx. This cohort will enable future nested studies focused on PEx management and other aspects of CF care. Understanding the baseline characteristics of these participants, as presented here, is critical for interpreting subsequent outcomes and for identifying factors that may influence disease progression and response to therapies.