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

Abstract

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.

Use of a mobile application for self-management of pancreatic enzyme replacement therapy is associated with improved gastro-intestinal related quality of life in children with Cystic Fibrosis

“…This was not achievable due to the expected small samples in each participating center. However, we did obtain data on natural variability of CF-PedsQL-GI before [15] , and the present increase of CF-PedsQL-GI exceeded this natural variability. Ideally, future research will include a control group, to confirm the findings that suggest an increase in QOL associated with using the app.…”

“…QOL was evaluated using -The shortened, CF specific version of the Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Module (CF-PedsQL-GI): a GI specific QOL questionnaire, consisting of 9 subdomains and 54 questions for use in children 5-18 years of age and in parents of children > 2 years. This shortened version of the Ped-sQL GI [12,13] was validated for use in children with CF [14,15] . Age-specific versions of the questionnaires were used in children and parents.…”

“…Change in CF-PedsQL-GI was assessed at M3 and M6. Based on the validation results and assuming a 15% drop-out, 200 patients would be needed to achieve a power of 80% and a significance level of 0.05 to see an improvement of 3.5 units in CF-PedsQL-GI, which was considered clinically meaningful, based on data from previous studies [15,22] .…”

“…We decided not to use CFQ-R, the most commonly used CF-specific QOL questionnaire, as primary outcome. Within the MyCyFAPP project the CF-PedsQL-GI was validated for use in children with CF focusing on GI symptoms [15] . The present results demonstrate its responsiveness to an intervention.…”

Dietary Aspects to Incorporate in the Creation of a Mobile Image-Based Dietary Assessment Tool to Manage and Improve Diabetes

“…Research has found that similar MIDAT for patients with cystic fibrosis was effective in supporting medical monitoring, adjusting medication and improving quality of life in patients. [69][70][71][72]. Other possibilities of this novel tool include, but are not limited to, analyzing dietary inputs to determine insulin requirements, linking and incorporating information from continuous glucose monitors, adjusting insulin dosing and completing delivery through insulin pumps to create a self-regulating, realtime responsive, "smart" system for PWD.…”