Modeling cystic fibrosis patient prognosis: Nomograms to predict lung transplantation and survival prior to highly effective modular therapy

Annalisa V Piccorelli and Jerry A Nick

PLOS ONE, 2024, vol. 19, issue 12, 1-13

Abstract: Background: The duration of time a person with cystic fibrosis (pwCF) spends on the lung transplant waitlist is dependent on waitlist and post-transplant survival probabilities and can extend up to 2 years. Understanding the characteristics involved with lung transplant and survival prognoses may help guide decision making by the patient, the referring CF Center and the transplant team. Methods: This study seeks to identify clinical predictors of lung transplant and survival of individuals with CF using 29,847 subjects from 2003–2014 entered in the Cystic Fibrosis Foundation Patient Registry (CFFPR). Results: Predictors significant (p ≤ 0.05) in the final logistic regression model predicting probability of lung transplant/death were: FEV1 (% predicted), BMI, age of diagnosis, age, number of pulmonary exacerbations, race, sex, CF-related diabetes (CFRD), corticosteroid use, infections with B. cepacia, P. aeruginosa, S. aureus, MRSA, pancreatic enzyme use, insurance status, and consecutive ibuprofen use for at least 4 years. The final Cox regression model predicting time to lung transplant identified these predictors as significant FEV1 (% predicted), BMI, age of diagnosis, age, number of pulmonary exacerbations, race, sex, CF-related diabetes (CFRD), corticosteroid use, infections with B. cepacia, P. aeruginosa, S. aureus, MRSA, pancreatic enzyme use, and consecutive ibuprofen use for at least 4 years. The concordance indices were 0.89 and 0.92, respectively. Conclusions: The models are translated into nomograms to simplify investigation of how various characteristics relate to lung transplant and survival prognosis individuals with CF not receiving highly effective CFTR modulator therapy.

Date: 2024
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Persistent link: https://EconPapers.repec.org/RePEc:plo:pone00:0292568

DOI: 10.1371/journal.pone.0292568

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