Researchers are developing a protocol that can be used to evaluate interventional trials on the management of pulmonary exacerbations of cystic fibrosis, according to details of a study published in Trials.
Pulmonary exacerbations are a hallmark of cystic fibrosis. Around 25% of patients do not fully recover after experiencing one such episode; hence, they are a key driver of lung damage. Although antibiotics and airway clearance therapies often offer symptomatic relief, significant knowledge gaps on the optimal management of these acute episodes remain.
Randomized controlled trials can provide researchers with a wealth of information regarding the efficacy of a therapeutic agent, but some trials exact a heavy toll on the participants or the clinicians, making it likely that some segments of the patient population are underrepresented. This is particularly a concern in a highly heterogeneous patient group, such as in the case of individuals with cystic fibrosis.
Thus, the authors of this study are developing a protocol to study cystic fibrosis pulmonary exacerbation management with the view that it will be useful in capturing data on the management and outcomes of any type of pulmonary exacerbation that requires intensive therapy.
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“The . . . protocol describes the establishment of a prospective, multi-site cohort that is intended to perpetually enroll children and adults with cystic fibrosis to serve as an adaptive platform for evaluating the (comparative) effectiveness of interventions for pulmonary exacerbations, including through cohort-nested sub-studies,” Schultz and colleagues wrote.
The research team has initially enrolled individuals with cystic fibrosis from various clinical centers across Australia to assess different clinical strategies for managing pulmonary exacerbations. The first phase of the study aims to capture the medical data of individuals with cystic fibrosis of all ages in a database within their digital infrastructure.
The ongoing study is currently operating based on protocol version 7.0, last updated on November 8, 2021. Recruitment for this trial commenced on October 14, 2020. Newly approved versions of the protocol are updated online when available.
Schultz A, McLeod C, Berry S, et al. BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis. Trials. Published online March 22, 2023. doi:10.1186/s13063-023-07076-8