Journal of Pediatric Gastroenterology and Nutrition最新文献

Objectives: People with cystic fibrosis (CF) are at risk of iron deficiency (ID), and although well established in adults, data in children are limited. We assessed iron status in a cohort of children with CF and evaluated the effectiveness of ID management.

Methods: We retrospectively analysed iron status and treatment data from medical records of 190 children with CF (aged 1-16 years) assessed routinely in 2021 and 2022 at the Royal Brompton Hospital (RBH), London. Ferritin, mean corpuscular volume (MCV), and haemoglobin were used to determine the prevalence of ID, ID erythropoiesis (IDE) and ID anaemia (IDA) using RBH definitions.

Results: The proportion of children with abnormal iron status indices decreased between the assessments (2021 - 63% vs. 2022 - 54%, p = 0.03). Prevalence of ID without anaemia (51% vs. 44%, p = 0.12), IDE (8% vs. 6%, p = 0.63), and IDA (4% vs. 3%, p = 1.00) did not differ between assessments. Sixty children received dietary advice for ID without anaemia, and iron supplements were prescribed for six and seven children with IDE and IDA, respectively. Change in iron status indices between assessments did not differ between treated and untreated children, although ferritin increased in the seven children treated for IDA (p = 0.04). There was a positive association between highly effective modulator therapy and change in MCV.

Conclusions: ID remains an issue in children with CF, warranting annual monitoring of iron status. Management with dietary advice and/or iron supplementation showed limited effectiveness using our criteria for abnormal iron status.

Objective: The latest European Society of Gastroenterology Hepatology and Nutrition (ESPGHAN) criteria for celiac disease (CD) diagnosis reduced the requirement for a small intestinal biopsy but still, for most of the cases a small intestinal biopsy is required for a safe diagnosis: hence the attempt to identify serum biomarkers that could replace, in most of these latter cases, the requirement of the biopsy (what is called a "liquid biopsy"). The aim of this study is to identify a set of serum biomarkers able to differentiate celiac patients from age and human leukocyte antigen (HLA)-matched healthy controls.

Methods: Relative concentration of 92 inflammation-linked proteins was examined in the sera of 50 children with CD compared with 50 HLA DQ2/8 and age-matched healthy controls born in genetically at-risk families, using proximity extension immunoassay technology (Olink Proteomics®) with the ProSeek Multiplex Inflammation panel.

Results: Three different multivariate analysis (Univariate and multivariate distribution analysis, random forest classification and linear discriminant analysis) localized a cluster of seven molecules (CASP8, CXCL9, NT-3, SIRT2, STAMBP, ST1A1, and TNFSF14) with a remarkable diagnostic potential, able to differentiate around 90% (95% confidence interval [CI]; 0.7-0.99) of CD patients from controls.

Conclusion: Patients with CD, compared to age- and HLA-matched healthy controls, show in their sera an increased expression of inflammatory molecules, involved in NF-κB cytokine signaling, cell apoptosis, and crypt proliferation pathways. A set of seven of these proteins can differentiate cases from controls with an accuracy higher than 90%. The implementation of this approach in clinical setting could in future facilitate a noninvasive and individualized approach for CD diagnosis.

Objectives: To assess whether low-volume transanal irrigation (L-TAI) is effective as add-on to oral laxative therapy for children with functional constipation and retentive fecal incontinence.

Methods: Two-arm randomized controlled trial, including children aged 4-14 suffering from retentive fecal incontinence. All included children were refractory to at least 2 months treatment with stool softening oral laxatives. Children were included across three pediatric departments in Denmark and randomized into two treatment groups. Treatment duration was 6 weeks. The control group continued oral laxative therapy. The intervention group received L-TAI as add-on. The primary objective was evaluating reduction in fecal incontinence episodes. Secondary objectives included assessment of constipation symptoms, rectal diameter, and well-being based on the WHO-5 questionnaire. Participants were classified as nonresponders (0%-49% reduction) or responders (partial response = 50%-99% reduction, or full response = 100% reduction).

Results: Fifty children were included. The respective median ages were 7 (interquartile range [IQR] = 3) in the intervention group and 6 years (IQR = 1) in the control group. In the intervention group, 75% were responders with 35% experiencing full response, while 33% in the control group were responders, with 4.8% experiencing full response (p = 0.007 and p = 0.020 for response and full response respectively). At follow-up, 55% of the intervention group and 90.5% of the control group still met ROME-IV criteria for constipation (p = 0.010).

Conclusions: L-TAI is effective as add-on to oral laxatives in treating fecal incontinence and constipation. Further studies with longer follow-up periods are needed to assess long-term effects. Clinical Trial identification number: NCT05570318 (https://clinicaltrials.gov/study/NCT05570318).

Extraintestinal manifestations (EIMs) manifest in 6%-47% of patients with inflammatory bowel disease (IBD). Here, we characterize the course of EIMs in pediatric patients receiving vedolizumab included in the VedoKids cohort study. This was a subgroup analysis of the pediatric VedoKids cohort, a multicenter, prospective study of children (aged 0-18 years) with IBD treated with vedolizumab and followed through 54 weeks. EIMs were identified in 18/142 (12.6%) children at baseline; 56% of these cases were articular EIMs; the EIMs resolved in 89% within 18 months. Concomitant medications were administered in 72% of EIM cases, most of which were ongoing at the time of vedolizumab initiation. Of the 124 children without EIMs, five (4%) developed EIMs during follow up: three arthritis, two cutaneous manifestations. The presence of EIMs did not affect the durability of vedolizumab treatment. In conclusion, most EIMs in children with IBD resolved with vedolizumab treatment, but almost half received concomitant medications. Clinical Trial Registration: ClinicalTrials.gov Identifier: NCT02862132. https://clinicaltrials.gov/study/NCT02862132?term=NCT02862132&rank=1.

Objectives: Nonesophageal eosinophilic gastrointestinal disorders (non-EoE EGIDs) are rare, underrecognized inflammatory diseases of the gastrointestinal (GI) tract, especially in children. Their clinical heterogeneity and lack of specific biomarkers contribute to diagnostic delays and therapeutic challenges.

Methods: This retrospective multicenter study included pediatric patients (<18 years) diagnosed with non-EoE EGIDs across 12 Italian centers affiliated with the Italian Society of Pediatric Gastroenterology Hepatology and Nutrition (SIGENP). The data were retrospectively collected from January 2012 to December 2022. Diagnosis was based on ESPGHAN histological criteria. Clinical, laboratory, endoscopic, and histological data were collected at baseline and during follow-up. Treatment modalities and outcomes were analyzed.

Results: A total of 86 patients (71% male; median age 10.5 years) were included. The stomach was the most commonly involved site (45.4%), followed by the small bowel (40.7%) and colon (24.4%). Multiple segment involvement occurred in 45.4% of cases. Abdominal pain (64.9%) and diarrhea (38.2%) were the most common symptoms, with diarrhea significantly associated with colonic involvement. Laboratory findings showed peripheral eosinophilia in 50% of patients and hypoalbuminemia in those with multisegment involvement. Very early-onset EGIDs (>1 and <2 years) were associated with features such as predominant colonic involvement and anemia. Treatment responses varied: 50% achieved clinical remission after first-line therapy (proton pump inhibitors, topical/systemic steroids, or elimination diets). Among 61/86 patients who underwent endoscopic follow-up, 49.2% achieved macroscopic remission and 40.9% histological remission.

Conclusions: Pediatric non-EoE EGIDs present with heterogeneous symptoms and variable tract involvement. Younger children show distinct phenotypes. Laboratory findings are nonspecific and have limited diagnostic utility. Treatment remains challenging, with suboptimal response rates. These findings underscore the need for prospective studies.

Objectives: The impact of elexacaftor/tezacaftor/ivacaftor (ETI) on cystic fibrosis (CF) hepatobiliary involvement (CFHBI) is uncertain. The goal of this study was to investigate the changes in key liver parameters in PUSH study participants who started ETI compared to those who did not (noETI).

Methods: PUSH was a prospective observational study of persons with CF (pwCF) (3-12 yo at PUSH entry). Linear mixed effect model (LMEM) with one knot tested if ETI changed the slope of trajectories of clinical parameters by comparing ETI to noETI. Index time (IT) for ETI was ETI start and time of ETI availability for noETI. Time zero in the LMEM was index time.

Results: One hundred forty-seven participants (104 ETI, 43 noETI). At IT, the groups were similar, for age, and liver parameters. Mean duration of ETI use was 22 months. The annual rate of change over time after IT in ETI compared to noETI was significantly improved for FEV₁% pre (+3.4/yr p = 0.02) and wt z score (+0.06/yr, p = 0.006). There was improvement for ETI vs noETI for GGT (-15%/yr, p = 0.01) and ALT (-12%/yr, p = 0.02). Participants with CFHBI on ETI demonstrated similar trends and also had improvements in GGT and GGT to platelet ratio (GPR), but there were no differences in liver stiffness or US classification changes.

Conclusions: GGT and GPR improve in pwCF and CFHBI who received ETI compared to noETI. There were no changes in other liver parameters. This suggests an early signal for positive impact on CFHBI, but no early improvement in fibrosis.