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

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 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. Methods: A multicenter, prospective observational study was performed in 6 European centers to validate the PedsQL GI in children with CF during 3 months. Results: In 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). Conclusions: PedsQL 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

Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis

Background A method to adjust Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis is not currently available. Objectives To assess the in vivo efficacy of a method to adjust the dose of enzymatic supplement in CF extrapolated from previous in vitro digestion studies (theoretical optimal dose, TOD). Secondly, to assess how individual patient characteristics influence the expected coefficient of fat absorption (CFA) and thus to identify an individual correction factor to improve TOD. Methods A prospective interventional study in 43 paediatric patients with CF from 5 European centres. They followed a 24h fixed diet with the theoretical optimal dose for each meal. Faecal collection was carried out between colorimetric markers in order to include all the faeces corresponding to the fixed diet. Beta regression models were applied to assess the associations of individual patient characteristics with the CFA. Results Median CFA was 90% (84, 94% 1st, 3rd Q.) with no significant differences among centres. Intestinal transit time was positively associated with CFA (p = 0.007), but no statistical associations were found with and age, gender, phenotype or BMI. Regression model showed no improvement of the in vitro predicted theoretical optimal dose when taking individual patient characteristics into account. Conclusion Strict adherence to the theoretical optimal dose of enzymatic supplement for a prescribed meal, led to median CFA levels at the clinical target of 90% with a low variability between patients. The proposed method can be considered as a first approach for an evidencebased method in PERT dosing based on food characteristics. Results have to be confirmed in free dietary settings

The Relative Contribution of Food Groups to Macronutrient Intake in Children with Cystic Fibrosis : a European Multicenter Assessment

Background: Optimal nutrition for children with cystic fibrosis (CF) improves prognosis and survival, but an increased caloric intake recommendation for this population raises concerns about the nutrient profile of their diets. Objective: Our aim was to assess the relative contribution of food groups to the total macronutrient intake of European pediatric patients with CF. Design: We conducted a cross-sectional study in which the participants recorded dietary intake from 2016 to 2017. Specifically developed nutritional composition databases were used to obtain nutritional data, including macronutrients and food groups, according to previously standardized criteria. Participants/setting: Two hundred and seven pediatric patients with CF from six European centers were involved in the My App for Cystic Fibrosis self-management project. Main outcome measures: Participants reported dietary intake with a detailed 4-day food record. Statistical analysis performed: Descriptive analyses of nutrient intake, food group consumption, and dietary origin of macronutrients were conducted with R software. Results: Similar patterns were found in nutrient and food group intake; both sugar and saturated fatty acids contributed >10% each to the total daily energy intake in all the centers. Large mean and median percent differences were observed in the intake of other nutrient and food groups, because sweets and snacks were consumed once or twice a day, and fruit and vegetables were consumed two or three times a day. Milk, meat, sweets and snacks, and oils were the main sources of fat in all centers. Conclusions: Study findings indicated less than optimal nutrient profiles, especially for sugars and saturated fatty acids, resulting from the high consumption of meat, dairy, and processed products and low consumption of fish, nuts, and legumes. These results can serve as a basis for future tailored interventions that target improved adherence to nutritional recommendations for patients with CF

Nomograms for morbidity and mortality after oncologic colon resection in the enhanced recovery era: results from a multicentric prospective national study

Purpose: Predicting postoperative complications and mortality is important to plan the surgical strategy. Different scores have been proposed before to predict them but none of them have been yet implemented into the routine clinical practice because their difficulties and low accuracy with new surgical strategies and enhanced recovery. The main aim of this study is to identify risk factors for postoperative morbidity and mortality after colonic resection (CR) without protective stomas, in order to develop a comprehensive, up-to-date, simple, reliable, and applicable model for the preoperative assessment of patients with colon cancer. Methods: Multivariable analysis was performed to identify risk factors for 60-day morbidity and mortality. Coefficients derived from the regression model were used in the nomograms to predict morbidity and mortality. Results: Three thousand one hundred ninety-three patients from 52 hospitals were included into the analysis. Sixty-day postoperative complications rate was 28.3% and the mortality rate was 3%. In multivariable analysis the independent risk factors for postoperative complications were age, male gender, liver and pulmonary diseases, obesity, preoperative albumin, anticoagulant treatment, open surgery, intraoperative complications, and urgent surgery. Independent risk factors for mortality were age, preoperative albumin anticoagulant treatment, and intraoperative complications. Conclusions: Risk factors for morbidity and mortality after CR for cancer were identified and two easy predictive tools were developed. Both of them could provide important information for preoperative consultation and surgical planning in the time of enhance recovery