Changes in Gastrointestinal Symptoms Among Children With Cystic Fibrosis During 12 Months of Elexacaftor/Tezacaftor/Ivacaftor Therapy: A Swedish Multicentre Study
Marcus Svedberg, Ellen Karlge Nilsson, Per Ekman, Henrik ImbergABSTRACT
Aim
Elexacaftor/Tezacaftor/Ivacaftor (ETI) has markedly improved clinical outcomes in cystic fibrosis (CF). However, its effects on gastrointestinal (GI) function, nutrition and pancreatic enzyme therapy in children remain insufficiently described. This study aimed to characterise GI‐related changes during the first year of ETI treatment in Swedish children with CF.
Methods
In this prospective, multicentre study at three paediatric CF centres, children aged 6–17 years who initiated ETI were assessed at baseline, 6 and 12 months. Outcomes included GI symptoms and the use of GI‐related medication, health‐related quality of life (CFQ‐R), nutritional status, PERT use and faecal elastase. Longitudinal changes were analysed using generalised least squares regression.
Results
Sixty‐two participants (mean age 11.7 years) completed follow‐up. The total mean combined number of reported GI symptoms declined from 1.5 to 0.9 ( p = 0.005), although no individual symptom changed significantly after 12 months of ETI treatment ( p > 0.05). CFQ‐R Digestive scores improved by 7.8 points ( p = 0.002). PERT dose decreased by 1.3 × 10 3 units/kg/day ( p < 0.001), while BMI z‐scores and faecal elastase remained unchanged.
Conclusion
ETI therapy reduced gastrointestinal symptoms and improved digestive quality of life in children with CF, accompanied by lower PERT use but unchanged pancreatic function and growth.