Nutritional outcomes in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor: A 24-month real-world study

Background The highly effective CFTR modulator elexacaftor/tezacaftor/ivacaftor therapy has revolutionized the care of cystic fibrosis (CF) leading to significant improvement in patient outcomes. However, data on long-term evolution of nutritional outcomes are limited. Methods We conducted a prospective study to evaluate changes in nutritional outcomes in people with CF (pwCF), including weight, body mass index (BMI), fat absorption and fat-soluble vitamin levels. Study outcomes were measured before treatment initiation and after 12 and 24 months. Mean changes and 95% confidence intervals (CI) were obtained using generalized estimating equations. Results A total of 84 patients with a median age of 19 years (range:12-32) were enrolled. At the 24-month follow-up visit, weight increased by 4.7 kg (95% CI: 3.5, 6.0) and BMI increased by 0.40 standard deviation scores (95% CI: 0.27, 0.53). The prevalence of overweight and obesity increased from 4.8% to 10.7%. After 24 months of ETI therapy, levels of vitamin A and E, as well as international normalized ratio (used as a marker of vitamin K status) were lower than baseline values, while vitamin D did not significantly change. Conclusions ETI has primarily led to significant improvements in the nutritional status of pwCF, resulting in increased BMI. However, a shift from the traditional hypercaloric and high fat diet to a balanced, healthy diet is essential to avoid excessive weight gain. Additionally, our findings underscore the need for ongoing monitoring of fat-soluble vitamin levels and adherence to supplementation.