Background: Most patients with cystic fibrosis (CF) suffer from pancreatic insufficiency (PI), leading to fat malabsorption, malnutrition, abdominal discomfort and impaired growth. Pancreatic enzyme replacement therapy (PERT) is effective, but evidence based guidelines for dose adjustment are lacking.A mobile app for self-management of PERT was developed in the context of the HORIZON 2020 project MyCyFAPP. It contains an algorithm to calculate individual PERT-doses for optimal fat digestion, based on in vitro and in vivo studies carried out in the same project.In addition, the app includes a symptoms diary, educational material, and it is linked to a web tool allowing health care professionals to evaluate patient's data and provide feedback. Methods: A 6-month open label prospective multicenter interventional clinical trial was performed to assess effects of using the app on gastro-intestinal related quality of life (GI QOL), measured by the CF-PedsQL-GI (shortened, CF specific version of the Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Module). Results: One hundred and seventy-one patients with CF and PI between 2 and 18 years were recruited at 6 European CF centers. Self-reported CF-PedsQL-GI improved significantly from month 0 (M0)

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Among the 6 centres, a large variability and inconsistency with new guidelines on nutrition and PERT-use was found. Our findings document the lack of a general criterion to adjust PERT and suggest the potential benefit of educational and self-managerial tools to ensure adherence to therapies, both for clinical staff and families. They will be taken into account when developing these new tools during the next stages of MyCyFAPP Project.

BackgroundExpressing anthropometric parameters (height, weight, BMI) as z-score is a key principle in the clinical assessment of children and adolescents. The Centre for Disease Control and Prevention (CDC) growth charts and the CDC-LMS method for z-score calculation are widely used to assess growth and nutritional status, though they can be imprecise in some percentiles.ObjectiveTo improve the accuracy of z-score calculation by revising the statistical method using the original data used to develop current z-score calculators.DesignA Gaussian Process Regressions (GPR) was designed and internally validated. Z-scores for weight-for-age (WFA), height-for-age (HFA) and BMI-for-age (BMIFA) were compared with WHO and CDC-LMS methods in 1) standard z-score cut-off points, 2) simulated population of 3000 children and 3) real observations 212 children aged 2 to 18 yo.ResultsGPR yielded more accurate calculation of z-scores for standard cut-off points (p<<0.001) with respect to CDC-LMS and WHO approaches. WFA, HFA and BMIFA z-score calculations based on the 3 different methods using simulated and real patients, showed a large variation irrespective of gender and age. Z-scores around 0 +/- 1 showed larger variation than the values above and below +/- 2.ConclusionThe revised z-score calculation method was more accurate than CDC-LMS and WHO methods for standard cut-off points. On simulated and real data, GPR based calculation provides more accurate z-score determinations, and thus, a better classification of patients below and above cut-off points. Statisticians and clinicians should consider the potential benefits of updating their calculation method for an accurate z-score determination.

BackgroundMost 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 patients with CF. As the Horizon2020 project MyCyFAPP aims to improve GI QOL by using a newly designed mobile application, a sensitive and reliable outcome measure was needed. We aimed to study the applicability of the existing child-specific Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Scales and Module (PedsQL GI) in children with CF.MethodsA multicenter, prospective observational study was performed in 6 European centers to validate the PedsQL GI in children with CF during 3 months.ResultsIn total, 248 children and their parents were included. Within-patient variability of PedsQL GI was low (24.11), and there was reasonable agreement between children and parents (ICC 0.681). Nine of 14 subscales were informative (no ceiling effect). The PedsQL GI and the median scores for 4 subscales were significantly lower in patients compared to healthy controls. Positive associations were found between PedsQL GI and age (OR = 1.044, p = 0.004) and between PedsQL GI and BMI z-score (OR = 1.127, p = 0.036). PedsQL GI correlated with most CFQ-R subscales (r 0.268 to 0.623) and with a Visual Analogue Scale (r = 0.20).ConclusionsPedsQL GI is a valid and applicable instrument to assess GI QOL in children with CF. Future research efforts should examine the responsiveness of the CF PedsQL GI to change in the context of clinical interventions and trials.