Assessing gastro-intestinal related quality of life in cystic fibrosis: Validation of PedsQL GI in children and their parents.

摘要: Background Most patients with cystic fibrosis (CF) suffer from pancreatic insufficiency, leading to fat malabsorption, malnutrition and abdominal discomfort. Until recently, no specific tool was available for assessing gastro-intestinal related quality of life (GI QOL) in CF. As the Horizon2020 project MyCyFAPP aims improve GI QOL by using a newly designed mobile application, sensitive reliable outcome measure needed. We aimed study applicability existing child-specific Pediatric Quality Life Inventory, Gastrointestinal Symptoms Scales Module (PedsQL GI) children CF. Methods A multicenter, prospective observational performed 6 European centers validate PedsQL CF during 3 months. Results In total, 248 their parents were included. Within-patient variability low (24.11), there reasonable agreement between (ICC 0.681). Nine 14 subscales informative (no ceiling effect). The median scores 4 significantly lower compared healthy controls. Positive associations found age (OR = 1.044, p 0.004) BMI z-score 1.127, 0.036). correlated most CFQ-R (r 0.268 0.623) Visual Analogue Scale 0.20). Conclusions PedsQL is valid applicable instrument assess Future research efforts should examine responsiveness change context clinical interventions trials.