Journal of Pediatric Gastroenterology and Nutrition最新文献

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.

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: Experience with subcutaneous infliximab (SC-IFX) use in pediatric inflammatory bowel disease (IBD) remains limited. We aimed to evaluate the clinical efficacy, tolerability, and cost-benefit of SC-IFX as a treatment for children with IBD in a real-world setting.

Methods: A prospective, single-center cohort study was conducted at the Children's Hospital at Westmead, Australia, from February to June 2024. Thirty-three children aged 8-18 years with established IBD commenced SC-IFX (120 mg fortnightly). Assessments included clinical disease activity indices (Pediatric Crohn's Disease Activity Index [PCDAI]/Pediatric Ulcerative Colitis Activity Index [PUCAI]), biochemical markers, and infliximab levels. Patient experience was assessed via questionnaires on quality-of-life (QOL) and treatment satisfaction.

Results: Thirty-one (31/33) children completed treatment over a 12-week follow-up period. Median infliximab levels increased from 7.8 ug/mL (interquartile range [IQR]: 5.7-11) at baseline to 19.6 ug/mL (IQR: 13.2-21.8) at Week 12 (p < 0.001). Serum infliximab level >20 ug/mL was seen in 48% of children at Week 12. Minimal change was observed in fecal calprotectin and clinical disease activity indices. High satisfaction rates with improved patient and caregiver QOL are described. Cost analyses indicated a potential for net institutional savings. Drug continuation was planned in 85% (28/33) following the 12-week study period.

Conclusions: Our experience suggests SC-IFX may be an effective and well-tolerated alternative to intravenous infliximab (IV-IFX) in pediatric IBD. Median serum infliximab levels increased across over the study period. Use of SC-IFX may offer a positive impact on patient and caregiver QOL, along with resource and cost saving benefits for healthcare systems. Larger-scale studies are needed to validate these findings and optimize treatment protocols.

Objective: Data on the relationship between body composition and strength in children with intestinal failure (IF) is lacking. The objectives were to (1) assess strength in children with IF compared to published norms; (2) examine the relationship between body composition and strength.

Methods: Children aged 4-18 years with IF, excluding those with significant developmental delay or mobility challenges, were included. Functional strength was assessed using the Bruininks-Oseretsky Test of Motor Proficiency-2 (BOT-2) and muscle strength was measured with a dynamometer for handgrip strength (HGS) and knee flexion/extension. Body composition was assessed using dual-energy X-ray absorptiometry. Two-sample t-test compared strength in IF patients to published norms, and the relationship between body composition and strength was analyzed using linear regression.

Results: Thirty-one children (71% male, mean age 9.9 years) were included. Twelve (39%) received parenteral nutrition (PN). Children with IF had significantly lower HGS (p = 0.004), knee flexion (p = 0.002), and extension (p < 0.001) compared to published norms. Specifically, 80.6% scored below the mean for HGS, 71.0% for knee flexion, and 74.2% for extension. BOT-2 revealed 26.9% had scores less than -1 SD. No significant differences in strength were found between those on enteral nutrition or PN. A significant positive relationship between functional strength and increased fat-free mass, and decreased fat mass was found (r² = 0.36, p = 0.002), with male sex strengthening the relationship (r² = 0.62, p < 0.001).

Conclusion: Children with IF, both on enteral nutrition and PN, are weaker than healthy literature controls. Decreased strength was associated with lower fat-free mass. This relationship was stronger in males than in females, highlighting the need to further investigate the mechanisms behind body composition and strength in this population.

Trial registration: ClinicalTrials.gov identifier: NCT04610918.

Objectives: To understand the clinical utility of small bowel video capsule endoscopy (VCE) based on indications, diagnostic yield, and management for pediatric patients suffering from disorders of the small bowel.

Methods: A single-center retrospective chart review was performed at Children's Health in Dallas, Texas between 2004 and 2022. Pediatric patients who underwent small bowel VCE were identified by Medtronic's Rapid PillCam Reader Software, and studies were organized by their primary indication. Chi-squared analysis was used to compare the rates of positive studies and studies leading to therapy or diagnostic plan changes by indication.

Results: Our analysis included 478 completed VCE studies of which 245 (51.3%) were positive and 169 (35.4%) led to therapy or diagnostic plan changes. The median age of patients was 12 years (range: 10 months to 18 years; interquartile range 8, 15) with 58.8% male patients. Compared to other indications, VCE studies performed for abdominal pain are less likely to be positive (p = 0.0003) and lead to fewer therapy and diagnostic plan changes (p = 0.0037).

Conclusions: VCE is useful in diverse clinical settings to diagnose and monitor small bowel pathologies. It leads to fewer changes in clinical management when the primary indication is abdominal pain.

Objectives: Bioelectric devices such as gastric electrical stimulation (GES) are used to treat severe upper gastrointestinal motility disorders in both younger and older patients. We compared clinical, physiologic, quality of life, and full-thickness gastric biopsy results between younger and older patients undergoing GES.

Methods: We analyzed 245 patients (45 males, 200 females; median age 42 [range 2-80 years] 47.6% with delayed solid gastric emptying and 52.4% nondelayed). All patients underwent temporary GES trials, followed by full-thickness gastric biopsies and physiologic assessments during permanent GES placement. Histologic evaluation used standardized cellular markers. Validated patient-reported outcomes were assessed at baseline and 1 year, with a 1-point improvement as clinical success.

Results: Younger patients (n = 37; 9 males, 28 females; median age 17 [range 2-25 years]); reported less severe anorexia (3.0 vs. 3.5, p = 0.005) and bloating (2.0 vs. 3.5, p < 0.001) at baseline, while nausea and vomiting severity were similar to 208 older adults. Biopsy analysis showed fewer CD3+ and CD8+ cells in younger patients (p = 0.032 and p = 0.002) and fibrosis was more common in older adults (41.2% vs. 16.1%, p = 0.008).

Conclusion: Younger patients with gastroparesis (Gp) syndromes have full-thickness gastric biopsy abnormalities and clinical measures that are similar, but not identical to older patients. These findings indicate the need for broader use of tissue analysis and other clinical measures in severely symptomatic patients with Gp symptoms undergoing the bio-electric therapy of gastric electrical stimulation to optimize perioperative counseling and postoperative symptom management.

Objectives: We aim to categorize the mechanical properties of the esophagus in children with eosinophilic esophagitis (EoE) in different disease stages and based on different management modalities, using endoscopic functional luminal imaging probe (EndoFLIP) which has been established to objectively evaluate mechanical properties of the esophagus in patients with EoE.

Methods: We performed a retrospective analysis of children who completed EndoFLIP during sedated endoscopy over 2 years. Patients with EoE were categorized into EoE remission or active EoE based on published guidelines. Control subjects were those with normal endoscopy, esophageal biopsies, and EndoFLIP parameters. EndoFLIP studies were analyzed for distensibility index (DI) and maximum diameter (MD) at the esophagogastric junction (EGJ), as well as contractile response (CR).

Results: We included 130 subjects, 60 controls, and 70 with EoE (34 [49%] had active EoE and 36 [51%] were in remission). DI and MD were significantly lower in active EoE compared to controls (p < 0.001). DI and MD were significantly lower in initial (baseline) EoE compared to active EoE on therapy (p < 0.05) and EoE remission (p < 0.001). There was no statistically significant difference in DI and MD between EoE subgroups on different therapies.

Conclusion: Assessment of esophageal mechanical properties using EndoFLIP provides valuable information on disease severity in pediatric EoE. In our cohort, histological remission achieved with therapies such as proton pump inhibitors, topical steroids, and dupilumab was associated with improved distensibility and diameter at the EGJ. However, a subset of patients continued to demonstrate abnormal EndoFLIP findings despite histologic improvement.

Objective: Extensively hydrolyzed formulas (eHF) are the first-line approach for cow's milk protein allergy (CMPA). This study evaluated the effects of eHF with synbiotic eHF (SeHF) on the general condition and symptoms of presumed CMPA infants during the initial 4-week exclusion diet period.

Methods: This observational prospective study (NCT04893330) recruited 114 patients (intention-to-treat [ITT] population; aged ≤8 months) with presumed CMPA. Patients were followed up for 28 days (D28) (per-protocol [PP], n = 80). Changes in general condition and presumed allergic symptoms were assessed using a 4-level Likert scale. Secondary endpoints included the time to symptom improvement, formula acceptability and consumption, and the parent's quality of life (QoL).

Results: The presumed allergic symptoms had improved/resolved for 86.3% of the PP population in 7-10 days on average. 86.8% of ITT cases had digestive symptoms at baseline and those reduced/disappeared in 93.2% of the PP cases at D28. The majority of parents agreed that the formula was suitable for the baby (78.8%). The QoL significantly improved from 60 ± 28 to 42 ± 24 (p < 0.001).

Conclusion: This study reflects the real-world management of presumed CMPA in our private practices using SeHF, which effectively improved CMPA symptoms. It also highlights the importance of waiting at least 2 weeks before reconsidering CMPA diagnosis.