PP085 - EFFECTS OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR ON PANCREATIC, AND NUTRITIONAL OUTCOMES IN ADULTS WITH CYSTIC FIBROSIS : A SINGLE-CENTER STUDY

PP085

EFFECTS OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR ON PANCREATIC, AND NUTRITIONAL OUTCOMES IN ADULTS WITH CYSTIC FIBROSIS : A SINGLE-CENTER STUDY

F. Vandamme¹, C. Audousset², H. Bernard¹, T. Perez², M. Lemaitre¹, D. Seguy^1,*

¹Diabetology/nutrition, ²CRCM (cystic fibrosis reference center), CHU de Lille, Lille, France

Rationale: Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a highly effective CFTR modulator for patients with cystic fibrosis (CF) carrying the F508del variant. However, data on extra-pulmonary outcomes in patients without this variant remain limited. This study aimed to confirm the beneficial effects of ETI in our cohort, which included 30% of patients without the F508del variant.

Methods: We conducted a single-center observational study in adult CF patients treated with ETI for at least 12 months at Lille University Hospital, France. All patients carried at least one ETI-responsive mutation. Data of annual report on respiratory function, nutritional status, body composition (assessed by bioimpedance analysis) and pancreatic function were collected at baseline and 12 months later.

Results: 49 patients (mean age ± SD, 32.5±10.5 years; 26.5% without the F508del variant) were included. After one year of ETI therapy, sweat chloride levels decreased by a median [Q1 – Q3] of -43 [-73.5 – -22.5] mEq/L (p<0.001) and amylase levels by -13 [-30.0 – -2.5] U/L (<0.001). FEV1 (force expired volume in 1 second) improved by +14.3 ± 10.7% of the predicted value. The number of pulmonary exacerbations decreased (p < 0.001), and body weight increased by +4.8±4.1 kg, with a BMI increase of +1.7±1.4 kg/m² (p<0.001). Hand grip strength improved by +2 [-2 – 6] kg (p=0.008), as did fat-free mass, albumin and transthyretin levels but also fat mass, and total cholesterol levels (p<0.001). These benefits were comparable between patients with and without F508del variant

Conclusion: ETI therapy should not be limited to patients with the F508del variant. Although effective across all disease aspects, patients should be aware of the impact of ETI on body fat mass, cholesterol levels and its potential long-term consequences especially in these population where cardiovascular risk is not yet well known

Disclosure of Interest: None declared