Journal of Pediatric Gastroenterology and Nutrition最新文献

Objectives: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a prevalent disease in children. Vibration-controlled transient elastography (VCTE) offers a noninvasive alternative to liver biopsy, using controlled attenuation parameter (CAP) to estimate steatosis and liver stiffness measurement (LSM) for fibrosis. However, pediatric data with histological validation are limited. This prospective, multicenter study evaluated the accuracy of CAP and LSM in pediatric MASLD.

Methods: Children with histologically confirmed MASLD from the Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Database 3 underwent VCTE within 6 months of liver biopsy. CAP was evaluated for correlation with steatosis grades, and LSM for correlation with fibrosis stages. The diagnostic performance of LSM in distinguishing fibrosis stages was analyzed using histological findings as the reference standard.

Results: Among 92 children with MASLD (mean age 13 ± 3 years), CAP values were similar across steatosis grades (median 325, 310, and 323 dB/m for grades 1-3, respectively) and showed no significant correlation with histologic steatosis (p = 0.422). Median LSM values increased with fibrosis stage (6.0-8.8 kPa), but significant differences were detected only between stage 0 and stage 3 fibrosis (p = 0.037). For advanced fibrosis (stages 3-4), area under the receiver operating characteristic curve was 0.67, with sensitivity 67%, specificity 76%, positive predictive value 40%, and negative predictive value 90%.

Conclusion: In this prospective, multicenter cohort, VCTE showed modest accuracy for grading steatosis or staging fibrosis in pediatric MASLD. Improved noninvasive methods are urgently needed for evaluation and monitoring in this population.

Objectives: Noncirrhotic portal hypertension (NCPH) is a rare condition marked by elevated portal venous pressure in the absence of cirrhosis and is caused by intrahepatic and extrahepatic pathways. This study aimed to describe the characteristics, etiologies, clinical manifestations, and outcomes of NCPH in children.

Methods: This study was a retrospective chart review of 63 patients in a single tertiary center. Participants were children (≤18 years old) diagnosed with portal hypertension in the absence of cirrhosis.

Results: The three most common etiologies included extrahepatic portal vein obstruction (EHPVO) in 27 (42.8%), congenital hepatic fibrosis (CHF) in 17 (27%), and nodular regenerative hyperplasia (NRH) in 10 (15.9%). At diagnosis, most patients presented with incidental splenomegaly (20; 32%) or gastrointestinal (GI) bleeding (20; 32%); 20.5% of children had ongoing evidence of clinical GI bleeding despite endoscopic intervention or beta-blocker use. Children with GI bleeding had significantly lower platelet counts (97.4 vs. 178.5 × 10³/μL, p = 0.005). Esophagogastroduodenoscopy (EGD) was performed in 43/63 (68.3%), commonly revealing esophageal varices (EV; 37; 86%) with EV Grade 3 in two-thirds. Liver transplantation was performed in 9 (21%), surgical shunts in 7 (11%), and transjugular intrahepatic portosystemic shunt (TIPS) in 2 (3%). The overall mortality rate was 3/63 (5%), with two deaths due to hemorrhagic shock in children with oncologic comorbidities.

Conclusions: NCPH, with diverse underlying etiologies, often presents with splenomegaly and variceal bleeding. Most patients who undergo endoscopy have varices. Treatment options include endoscopic treatment, shunting, and liver transplant; mortality was seen in 5% of children in this series.

Objectives: Deficiency of serum 25-hydroxyvitamin D [25(OH)D] was associated with decreased short-term response to anti-tumor necrosis factor-alpha (TNF-α) agents in adults with inflammatory bowel disease (IBD). The aim of this study was to evaluate the association between serum 25(OH)D levels and the outcome of children with IBD undergoing anti-TNF-α therapy.

Methods: Children with IBD who were treated with anti-TNF-α agents and whose 25(OH)D levels had been measured at the initiation of therapy were included. Demographic, clinical, and laboratory data were collected retrospectively between 1/2012 and 1/2022. 25(OH)D levels above 30 ng/mL were considered sufficient.

Results: A total of 150 children with IBD were treated with anti-TNF-α agents, and 84 of them (58 Crohn's disease, 26 ulcerative colitis, median [interquartile range] age 15.2 [12.8-16.5] years) met the inclusion criteria. Sixty-five (77%) patients were 25(OH)D-deficient. Adequate 25(OH)D levels were associated with clinical response (hazard ratio [HR] = 4, 95% confidence interval [CI] 1.43-11.11, p = 0.008), and clinical remission (HR = 4.62, 95% CI 2.56-8.33, p < 0.001). While anti-TNF-α trough levels were comparable between 25(OH)d-deficient and non-deficient children, intensification of anti-TNF-α therapy was more prevalent among children with 25(OH)D deficiency (65% vs. 21%, p < 0.001).

Conclusions: Adequate serum 25(OH)D is an independent predictor of a favorable outcome of pediatric IBD under anti-TNF-α therapy.

Objective: Patient satisfaction (PS) is an important healthcare outcome and should be an integral part of quality improvement agendas. Between 2019 and 2021, the Italian Society of Pediatric Gastroenterology, Hepatology and Nutrition, Endoscopy Interest Group conducted a nationwide quality assessment survey, EndoPed. One of the study's aims was to identify areas for improvement based on the results of a PS questionnaire.

Methods: The smartphone app-based, prospective survey involved 24 Italian pediatric endoscopy centers. Procedure details and quality standards were recorded. The modified Group Health Association of America-9 (GHAA-9m) satisfaction questionnaire consisted of 7 Likert-scale questions that explored waiting times, communication, and staff evaluation. Two additional closed questions investigated the will to confirm the endoscopist and the facility. Dissatisfaction was defined with the arbitrary cut-off of < 7/10.

Results: Questionnaire results were available for 1974 (55%) procedures. The questionnaire's internal consistency was good (Cronbach's α 0.86). Overall satisfaction was high. Shorter operator experience (odds ratio [OR] 4 [95% confidence interval [CI] 1.6 to -10.3]; p < 0.003), prolonged waiting time (OR 2.7 [95%CI 1.6-4.5]; p < 0.000), prolonged procedure duration (OR 2.9 [95% CI 1.8-4.7]; p < 0.000) and conscious sedation (OR 5 [95% CI 3-6.2]; p < 0.000) were confirmed in a multivariate model to be associated to lower satisfaction rates.

Conclusions: Through a standardized methodology, the PS questionnaire is a valuable tool for clinical quality assessment. PS highlight improvement fields different from those identified by quality standards and items, adding elements for an exhaustive quality evaluation. Interventions derived from PS analysis could help to improve pediatric endoscopy.

Objective: Fatigue is highly prevalent in children with inflammatory bowel disease (IBD), even during clinical remission. This suggests a role for transdiagnostic factors-lifestyle, psychological, and social influences not specific to the disease. This study aimed to assess the prevalence of severe fatigue in pediatric IBD and evaluate its associations with both IBD-focused and transdiagnostic factors.

Methods: Children with IBD ages 8-18 from the PROactive cohort completed the Pediatric Quality of Life Inventory-Multidimensional Fatigue Scale. IBD-focused clinical data were extracted from electronic health records. Transdiagnostic factors were assessed using validated patient-reported outcome measures. Associations with fatigue were examined using linear regression.

Results: Among 127 patients (mean age 14.9 ± 2.7 years; 43% male), most were in clinical remission. One hundred six patients self-reported fatigue, and 99 parents reported their child's fatigue. Severe fatigue was self-reported by 29%. Of IBD-focused factors, only disease activity (β = -0.40) and comorbidity (β = -0.18) showed significant associations with fatigue. Of transdiagnostic factors, lower physical, emotional, and social functioning; poorer sleep quality; less physical activity; more pain, anxiety, and depressive symptoms; lower life satisfaction and self-rated health; and increased school absence and pressure were significantly associated with more fatigue. Backward selection retained only transdiagnostic factors in the multivariate model, which explained 78% of fatigue variance.

Conclusion: Fatigue is common in children with IBD and is more strongly associated with transdiagnostic than disease-focused factors. These findings highlight the importance of integrative care strategies that address modifiable psychosocial and lifestyle domains to reduce fatigue and improve functioning in children with IBD.

Objectives: There are no standardized criteria about stepping down from combination therapy (immunomodulator and tumor necrosis factor (TNF)-alpha-inhibitors) in children with inflammatory bowel disease (IBD) to reduce risk for side effects. Our aim was to describe how de-escalation has been performed in a large paediatric cohort and to find prognostic factors for therapy de-escalation without the need for therapy adjustments post-de-escalation.

Methods: Real-world data from CEDATA-GPGE, a German-Austrian registry for paediatric IBD patients, from 2004 to 2023, were analyzed. Patients not requiring therapy adjustments post-de-escalation and patients requiring therapy adjustments after de-escalation were compared, and prognostic factors were identified.

Results: Two hundred and thirty out of 6248 registered patients received combination therapy for at least 6 months. In 64 patients therapy adjustment was not required after de-escalation. Crohn's disease (CD) patients, younger patients, and patients with positive modified predictors of poor outcome were significantly more often on combination therapy. Regarding de-escalation, CD patients were more often successfully de-escalated than ulcerative colitis (UC) and IBD-unclassified patients. UC patients with a less severe disease manifestation (Paris classification E1 or E2) were de-escalated more successfully than those with more extensive disease (E3 or E4). De-escalation to monotherapy with a biologic led to a more successful de-escalation than de-escalation to immunomodulator monotherapy or stopping both biologic and immunomodulator.

Conclusions: De-escalation is more likely successful in patients with CD, and de-escalating combination therapy to monotherapy with a TNF-alpha-inhibitor is more advantageous.