Developing a Framework for Studying Lung Function Decline in Cystic Fibrosis
Research By: Rhonda Szczesniak, PhD
Post Date: July 1, 2023 | Publish Date: July 2023
Biostatistics and Epidemiology | Top Scientific Achievement
Lung function data is crucial for tracking the progression of cystic fibrosis (CF). Although this data has become more widely available through CF patient registries, variability in data collection and study methods has led to inconsistencies in estimates of lung function decline.
To address this, the Cystic Fibrosis Foundation convened a group of experts across several institutions to establish an analytic framework for assessing lung function decline in CF patients over time. Rhonda Szczesniak, PhD, with the divisions of Biostatistics and Epidemiology and Pulmonary Medicine at Cincinnati Children’s, served as first author for the group’s findings.
The researchers used data from over 35,000 patients in the Cystic Fibrosis Foundation Patient Registry to simulate various study scenarios. Scenarios varied by sample size, data reporting frequency, follow-up length and the inclusion of data collected during pulmonary exacerbations. Applying several linear and nonlinear statistical models, they systematically tested how these factors impacted results and published the code to implement all models.
“While outcomes differed based on statistical approach, results were generally consistent across most data availability scenarios,” Szczesniak says.
The findings highlight how methodological choices can influence study outcomes. Using these insights, the team developed guidelines for selecting the most suitable modeling approach to study lung function decline in CF, depending on study goals and data characteristics.
The findings offer a valuable resource for guiding future research on longitudinal CF outcomes, enhancing our ability to understand disease progression and assess the effectiveness of novel treatments and interventions, the co-authors say.
Other Cincinnati Children’s co-authors included Weiji Su, Emrah Gecili, Enas Ghulam and Grace Zhou. Collaborators included experts from the University of Cincinnati and 15 other research organizations.
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| Original title: | Lung Function Decline in Cystic Fibrosis: Impact of Data Availability and Modeling Strategies on Clinical Interpretations |
| Published in: | Annals of the American Thoracic Society |
| Publish date: | July 2023 |
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The Research Horizons blog features news and insights about the latest discoveries and innovations developed by the scientists of Cincinnati Children's. This blog does not provide medical advice, diagnosis, or treatment.
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