Advisory Committee Chair
Advisory Committee Members
Date of Award
Degree Name by School
Doctor of Philosophy (PhD) School of Education
Background: Since malnutrition is a main clinical consequence of CF, a high-calorie, high-fat diet is recommended. Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators promote improved growth and weight gain in subsets of the CF population. Recently Elexacaftor/Tezacaftor/Ivacaftor (ETI) was approved for use in up to 90% of people with CF. Our single center open-label observational cohort study aimed to explore the changes in dietary and metabolic outcomes with use of ETI. Methods: Participants underwent baseline (V1) measurements prior to taking their first dose of ETI. Follow-up measurements were obtained at 28 days (V2) on ETI and > 6 months on drug (V3). Measurements at each time point included: weight, BMI, lung function, resting energy expenditure (REE%), hand grip strength (HGS), dietary intake, and enzyme dosage. Wilcoxon sign rank tests were used to compare changes in outcomes at each time point. Results: A total of 22 participants enrolled and completed baseline assessments. V2 was completed by 20 participants and 17 participants completed V3. Participants were 16-54 years of age (mean 26 years), and 50% were not previously taking CFTR modulators. Mean (± SD) BMI increased by 0.46 ± 0.93 kg/m² (p < 0.05) at V2 and 0.92 ± 0.88 kg/m² at V3 compared to V1 (p < 0.0001). REE% decreased by 6.6 ± 15.3 from V1 to V3 (p < iv 0.05). Total caloric intake increased by 297 ± 766 kcal/day (p < 0.05) and total fat intake increased by 19 ± 37 grams/day between V1 and V3 (p < 0.05). Conclusions: ETI increased BMI rapidly and this was sustained through at least 6 months. Decreased REE% combined with increased caloric intake are potential mechanisms of weight gain on ETI. Findings highlight the need for individualized nutritional counseling to improve diet quality and manage weight changes on ETI in the clinical setting.
Bailey, Julianna, "Nutritional and Metabolic Effects of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis" (2021). All ETDs from UAB. 543.