The natural history of primary sclerosing cholangitis in 781 children. A multicenter, international collaboration

There are limited data on the natural history of primary sclerosing cholangitis (PSC) in children. We aimed to describe the disease characteristics and long-term outcomes of pediatric PSC. We retrospectively collected all pediatric PSC cases from 36 participating institutions and conducted a survival analysis from the date of PSC diagnosis to dates of diagnosis of portal hypertensive or biliary complications, cholangiocarcinoma, liver transplantation, or death. We analyzed patients grouped by disease phenotype and laboratory studies at diagnosis to identify objective predictors of long-term outcome. We identified 781 patients, median age 12 years, with 4,277 person-years of follow-up; 33% with autoimmune hepatitis, 76% with inflammatory bowel disease, and 13% with small duct PSC. Portal hypertensive and biliary complications developed in 38% and 25%, respectively, after 10 years of disease. Once these complications developed, median survival with native liver was 2.8 and 3.5 years, respectively. Cholangiocarcinoma occurred in 1%. Overall event-free survival was 70% at 5 years and 53% at 10 years. Patient groups with the most elevated total bilirubin, gamma-glutamyltransferase, and aspartate aminotransferase-to-platelet ratio index at diagnosis had the worst outcomes. In multivariate analysis PSC-inflammatory bowel disease and small duct phenotypes were associated with favorable prognosis (hazard ratios 0.6, 95% confidence interval 0.5-0.9, and 0.7, 95% confidence interval 0.5-0.96, respectively). Age, gender, and autoimmune hepatitis overlap did not impact long-term outcome. CONCLUSION: PSC has a chronic, progressive course in children, and nearly half of patients develop an adverse liver outcome after 10 years of disease; elevations in bilirubin, gamma-glutamyltransferase, and aspartate aminotransferase-to-platelet ratio index at diagnosis can identify patients at highest risk; small duct PSC and PSC-inflammatory bowel disease are more favorable disease phenotypes

Navigating global collaboration:challenges faced by the international network on esophageal atresia

The International Network on Esophageal Atresia (INoEA) stands as a beacon of collaboration in addressing the complexities of this congenital condition on a global scale. The eleven board members, from various countries (USA, Canada, France, Australia, Italy, Sweden, Germany, and The Netherlands) and backgrounds (pediatric gastroenterology, pediatric surgery, pediatric pulmonology, nursing, and parents) met in a face-to-face symposium in Lille in November 2023, to identify challenges and solutions for improving global collaboration of the network.</p

The Case for Thoughtful Prescribing of Proton Pump Inhibitors in Infants.

International audienc

Association between dietary inflammatory index and inflammatory markers in the HELENA study

Background: Previous research has shown that diet is associated with low-grade systemic inflammation among adults. However, no study has yet been conducted to explore the association between inflammatory potential of diet and low-grade systemic inflammation among adolescents whose dietary behavior may be different from adults. Methods and Results: We examine the predictive ability of 24-h recall-derived dietary inflammatory index (DII) scores on inflammation among 532 European adolescents in the HELENA cross-sectional study. The DII is a literature-derived dietary index developed to predict inflammation. The DII was calculated per 1000 calories and was tested against C-reactive protein, ILs-1,2,4,10, TNF-alpha, ICAM, vascular cell adhesion molecule (VCAM), and IFN-gamma. All inflammatory markers had nonnormal distributions and therefore were log transformed. Analyses were performed using multivariable linear regression, adjusting for age, sex, city, BMI, smoking, and physical activity. Pro-inflammatory diet (higher DII scores) was associated with increased levels of various inflammatory markers: TNF-alpha, IL-1, 2, IFN-gamma, and vascular cell adhesion molecule (b(DIIt3vs1) = 0.13, 95% CI: 0.001, 0.25; 0.13, 95% CI 0.001, 0.25; 0.40, 95% CI: 0.03, 0.77; 0.53, 95% CI: 0.05, 1.01; 0.07, 95% CI: 0.01, 0.13, respectively). Conclusion: These results reinforce the fact that diet, as a whole, plays an important role in modifying inflammation in adolescents

Development of a core outcome set for paediatric achalasia: a joint ERNICA, ESPGHAN and EUPSA study protocol

Introduction: Achalasia is a rare disease in children. Studies investigating the efficacy of interventions and disease outcomes in paediatric achalasia are predominantly retrospective, consist of small cohorts and report heterogeneous outcomes. The variation in the use and definition of reported outcomes impedes meta-analysis, which is problematic in a rare paediatric condition. Similarly, there is a risk of under-reporting patient-relevant outcomes, such as quality of life. To overcome these issues, a minimum set of important and patient-relevant outcomes should be reported in all studies of paediatric achalasia. Core outcome sets (COS) are a standardised set of outcomes that can guide further research and facilitate data pooling and meta-analysis. The development of a COS in rare paediatric disease is essential, prior to conducting efficacy studies or creating a disease registry, to ensure that the most important outcomes are reported. Currently, no COS exists for children with achalasia. In this study, we aim to define a COS for paediatric achalasia for use in clinical research.// Methods and analysis: This study will consist of three parts. The first will be a systematic review of the literature, evaluating the outcomes and outcome definitions reported in published clinical research studies investigating paediatric achalasia. Second, a three-stage Delphi consensus process will be undertaken to identify and prioritise outcomes. This process will involve healthcare professionals, patients and parent representatives. Third, a consensus meeting will be held, during which the final COS will be defined.// Dissemination: The results of this study will be disseminated to stakeholders via the European Reference Network for Rare Inherited Congenital Anomalies, European Society for Pediatric Gastroenterology Hepatology and Nutrition, European Paediatric Surgeons’ Association, and patient groups. The COS will be published in a peer-reviewed journal and uploaded to the Core Outcome Measures in Effectiveness Trials (COMET) initiative website.// Trial registration number The study was pre-registered with the COMET initiative in July 2024 (https://www.comet-initiative.org/Studies/Details/2568). The systematic review component of the study was pre-registered on PROSPERO (CRD42024509855)

Associations between macronutrient intake and serum lipid profile depend on body fat in European adolescents: the Healthy Lifestyle in Europe by Nutrition in Adolescence (HELENA) study

The present study aimed to investigate the relationships between macronutrient intake and serum lipid profile in adolescents from eight European cities participating in the HELENA (Healthy Lifestyle in Europe by Nutrition in Adolescence) cross-sectional study (2006–7), and to assess the role of body fat-related variables in these associations. Weight, height, waist circumference, skinfold thicknesses, total choles- terol, HDL-cholesterol (HDL-C), LDL-cholesterol, TAG, apoB and apoA1 were measured in 454 adolescents (44 % boys) aged 12·5–17·5 years. Macronutrient intake (g/4180 kJ per d (1000 kcal per d)) was assessed using two non-consecutive 24 h dietary recalls. Associations were evaluated by multi-level analysis and adjusted for sex, age, maternal education, centre, sum of four skinfolds, moderate-to-vigorous

Clustering patterns of physical activity, sedentary and dietary behavior among European adolescents: The HELENA study.

Evidence suggests possible synergetic effects of multiple lifestyle behaviors on health risks like obesity and other health outcomes. A better insight in the clustering of those behaviors, could help to identify groups who are at risk in developing chronic diseases. This study examines the prevalence and clustering of physical activity, sedentary and dietary patterns among European adolescents and investigates if the identified clusters could be characterized by socio-demographic factors. METHODS: The study comprised a total of 2084 adolescents (45.6% male), from eight European cities participating in the HELENA (Healthy Lifestyle in Europe by Nutrition in Adolescence) study. Physical activity and sedentary behavior were measured using self-reported questionnaires and diet quality was assessed based on dietary recall. Based on the results of those three indices, cluster analyses were performed. To identify gender differences and associations with socio-demographic variables, chi-square tests were executed. RESULTS: Five stable and meaningful clusters were found. Only 18% of the adolescents showed healthy and 21% unhealthy scores on all three included indices. Males were highly presented in the cluster with high levels of moderate to vigorous physical activity (MVPA) and low quality diets. The clusters with low levels of MVPA and high quality diets comprised more female adolescents. Adolescents with low educated parents had diets of lower quality and spent more time in sedentary activities. In addition, the clusters with high levels of MVPA comprised more adolescents of the younger age category. CONCLUSION: In order to develop effective primary prevention strategies, it would be important to consider multiple health indices when identifying high risk groups.Peer Reviewe

Correlates of dietary energy misreporting among European adolescents : the Healthy Lifestyle in Europe by Nutrition in Adolescence (HELENA) study

This study examined the correlates of dietary energy under-reporting (UR) and over-reporting (OV) in European adolescents. Two selfadministered computerised 24-h dietary recalls and physical activity data using accelerometry were collected from 1512 adolescents aged 12.5-17.5 years from eight European countries. Objective measurements of height and weight were obtained. BMI was categorised according to Cole/International Obesity Task Force (IOTF) cut-off points. Diet-related attitudes were assessed via self-administered questionnaires.Reported energy intake (EI) was compared with predicted total energy expenditure to identify UR and OV using individual physical activity objective measures. Associations between misreporting and covariates were examined by multilevel logistic regression analyses. Among all, 33.3% of the adolescents were UR and 15.6% were OV when considering mean EI. Overweight (OR 3.25; 95% CI 2.01, 5.27) and obese (OR 4.31; 95% CI 1.92, 9.65) adolescents had higher odds for UR, whereas underweight individuals were more likely to over-report (OR 1.67; 95% CI 1.01, 2.76). Being content with their own figures (OR 0.61; 95% CI 0.41, 0.89) decreased the odds for UR, whereas frequently skipping breakfast (OR 2.14; 95% CI 1.53, 2.99) was linked with higher odds for UR. Those being worried about gaining weight (OR 0.55; 95% CI 0.33, 0.92) were less likely to OV. Weight status and psychosocial weight-related factors were found to be the major correlates of misreporting. Misreporting may reflect socially desirable answers and low ability to report own dietary intakes, but also may reflect real under-eating in an attempt to lose weight or real over-eating to reflect higher intakes due to growth spurts. Factors influencing misreporting should be identified in youths to clarify or better understand diet-disease associations

An international survey on anastomotic stricture management after esophageal atresia repair:considerations and advisory statements

BACKGROUND: Endoscopic dilatation is the first-line treatment of stricture formation after esophageal atresia (EA) repair. However, there is no consensus on how to perform these dilatation procedures which may lead to a large variation between centers, countries and doctor’s experience. This is the first cross-sectional study to provide an overview on differences in endoscopic dilatation treatment of pediatric anastomotic strictures worldwide. METHODS: An online questionnaire was sent to members of five pediatric medical networks, experienced in treating anastomotic strictures in children with EA. The main outcome was the difference in endoscopic dilatation procedures in various centers worldwide, including technical details, dilatation approach (routine or only in symptomatic patients), and adjuvant treatment options. Descriptive statistics were performed with SPSS. RESULTS: Responses from 115 centers from 32 countries worldwide were analyzed. The preferred approach was balloon dilatation (68%) with a guidewire (66%), performed by a pediatric gastroenterologist (n = 103) or pediatric surgeon (n = 48) in symptomatic patients (68%). In most centers, hydrostatic pressure was used for balloon dilatation. The insufflation duration was standardized in 59 centers with a median duration of 60 (range 5–300) seconds. The preferred first-line adjunctive treatments in case of recurrent strictures were intralesional steroids and topical mitomycin C, in respectively 47% and 31% of the centers. CONCLUSIONS: We found a large variation in stricture management in children with EA, which confirms the current lack of consensus. International networks for rare diseases are required for harmonizing and comparing the procedures, for which we give several suggestions. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s00464-020-07844-6) contains supplementary material, which is available to authorized users