WIP 46 - Chronic infection status, nutritonal status and lung function changes in patients with cystic fibrosis before and after starting Elexacaftor/Tezacaftor/Ivacaftor: multi-center experience
Second year Pediatric resident Nemours Children's Hospital Orlando, Florida, United States
Background: Cystic fibrosis (CF) mutations cause a niche for colonization and infection in the lungs by various microorganisms. As CFTR modulator therapies can improve CFTR function it can be expected that modulator therapy will improve patient’s lung function and decrease exacerbations decreasing morbidity and mortality. Filho et al (2021) showed different mortality mean ages in different countries which could be related to having or not having access to modulator therapies. In 2019, a triple modulator therapy (Elexacaftor/Ivacaftor/Tezacaftor) was approved by the FDA for use in over 90% of CF patients. By extrapolation from studies done with monotherapy or dual therapy, it is believed that the triple modulator therapy will help on infection and pathogenesis of microorganism colonization in CF patients. Objective: The primary research aim is to evaluate the treatment outcome for patients with cystic fibrosis at 1 year post Elexacaftor/Ivacaftor/Tezacaftor treatment. The changes of the following endpoints from before treatment to 1-year post treatment are of interest: 1) status of colonization 2) lung function, and 3) nutritional status. Design/Methods: Retrospective cohort study, from data of patients with cystic fibrosis receiving Elexacaftor/Ivacaftor/Tezacaftor that were been followed at least one year before and after starting treatment seen at Nemours Children’s Health hospitals located at Orlando, Jacksonville, and Pensacola, FL, and Wilmington, DE. Study was approved by IRB. Data to be collected: oropharyngeal or sputum cultures, weight and height during visits, liver enzymes, and spirometry results. The chronic infection status will be defined by having more than 50% of cultures positive for the same bacteria or fungus despite of treatment in a year. Categorical variables will be summarized as frequency (percentage) and continuous variables will be reported as median (range). Wilcoxon signed rank test will be used to evaluate the change in outcome variables between pre and post treatment. All tests were two-sided with p value < 0.05 considered statistically significant.
