The positive nutritional and gastrointestinal effects of elexacaftor/tezacaftor/ivacaftor in the treatment of cystic fibrosis



Year of publication 2023
Type Conference abstract
MU Faculty or unit

Faculty of Medicine

Description Background: Highly effective modulator therapy improves both nutritional status and quality of life. Clinical trials have even shown pancreatic insufficiency conversion, but mostly in paediatric patients treated with ivacaftor. Studies with elexacaftor/tezacaftor/ivacaftor (ETI) in older patients have not suggested the restoration of exocrine pancreas function, but quality data in adults are still lacking. Our aim was to show the effect of ETI therapy on the nutritional status and digestive function in adult patients with cystic fibrosis (CF). We hypothesized the improvement of nutritional parameters and gastrointestinal symptoms, and the reduction of pancreatic enzyme replacement therapy, but uncertain improvement in exocrine pancreatic function. Methods: This prospective study enrolled adult patients with CF treated with ETI from August 2021 to June 2022. We measured anthropometric parameters, laboratory nutritional markers, change of faecal elastase, changes in pancreatic enzymes replacement therapy needs, and gastrointestinal symptoms. Results: In the cohort of 29 adult patients with CF (mean age 29.1 years), 82.8% suffered exocrine pancreatic insufficiency. After ETI therapy, the mean BMI increased by 1.20 kg/m2 (p < 0.001), mean body weight by 3.51 kg (p < 0.001), albumin by 2.81 g/L, and prealbumin by 0.06 (both p < 0.001). One patient (4.5%, p < 0.001) developed pancreatic sufficiency, indicated by faecal elastase > 200 µg/g. The mean change in lipase substitution decreased by 1,969 units/kg/day (p < 0.001) and stool frequency by 1.18 per day (p < 0.001). No acute pancreatitis was observed. Conclusion: Our data suggests increased nutritional parameters, a restoration of exocrine pancreatic function, lower pancreatic substitution requirements, and improved defecation in adult CF patients on ETI therapy. Improvement in exocrine pancreatic function might be mutation-specific and requires further study.

You are running an old browser version. We recommend updating your browser to its latest version.

More info