Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass

• DOI: 10.3389/fphar.2023.1157459
• Publication Date: 2023-07-13T10:40:52Z
• Authors: Marialena Mouzaki; Annie Dupuis; Julie Avolio; Katherine Griffin; Felix Ratjen; Elizabeth Tullis; Tanja Gonska
• Keywords: Ivacaftor; Weight change

Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. The mechanism as well impact of ivacaftor on weight, resting energy expenditure (REE) body composition remains be explored. Methods: This prospective observational study included 18 people with CF (pwCF) (age: median (range) 20 (6-58) years) carrying at least one gating mutation commencing ivacaftor. Assessments composition, REE laboratory investigations were performed baseline 6, 12 24 months after treatment initiation. Results: Treatment was associated a significantly positive change BMI z-score months. Fat mass (mean (95% CL) 6.5 kg (4.0; 9.0) from baseline, p = 0.0001), but not fat-free changed under treatment. There correlation between fat change. Overall, there no measured 108 kcal/d (-12; 228), 0.07) our cohort. Pancreatic other nutritional markers did treatment, exception an increase serum vitamin A levels (p 0.006). Conclusion: gain observed treated pwCF is predominantly secondary increases warranting early counseling starting CFTR-modulating respect healthy diet physical exercise.