Journal of Pediatric Gastroenterology and Nutrition最新文献

{"title":"Acute pancreatitis gut dysbiosis persists at 1-year follow-up and is associated with clinical outcomes.","authors":"Chinenye R Dike, Qing Duan, Faizan Ahmed, Lee A Denson, David Haslam, Philip Minar, Nicholas J Ollberding, Georgios I Papachristou, Kenneth D R Setchell, Tyler Thompson, David S Vitale, Xueheng Zhao, Maisam Abu-El-Haija","doi":"10.1002/jpn3.70135","DOIUrl":"10.1002/jpn3.70135","url":null,"abstract":"<p><strong>Objectives: </strong>Pediatric acute pancreatitis (AP) is associated with gut dysbiosis. We aimed to determine if dysbiosis persisted during follow-up and whether it is associated with clinical outcomes.</p><p><strong>Methods: </strong>Prospective enrollment of participants <21 years with first AP. Stool samples were obtained at baseline (n = 41), 3 months (n = 19), and 12 months (n = 12) and in healthy controls (HC; n = 34). Evaluation for diabetes (DM) or prediabetes (pre-DM) was performed. At 12-month follow-up gastrointestinal (GI) symptom surveys were completed and AP recurrence-acute recurrent pancreatitis (ARP) recorded. Shotgun metagenomic sequencing was performed on extracted microbial DNA.</p><p><strong>Results: </strong>Microbial alpha diversity was lower for AP versus HC at all three time points (p < 0.008). Bray-Curtis ordinations showed the AP cohort did not cluster by time point, highlighting similarity in microbial composition over time. Within 12-month follow-up: 7/44 participants developed pre-DM/DM, 7/42 developed ARP, 16 had zero or one while 15 had multiple GI symptoms. Distinct clustering of samples was observed in the baseline samples of the group that developed ARP (p = 0.023) and in follow-up samples with multiple GI symptoms, p < 0.05. Relative abundance of most species was lower in AP samples when compared to HC at all time points with enrichment in Ruminococcus gnavus and Clostridium innocuum (AQ) (False Discovery Rate p < 0.05). Several pathways involved in protein biosynthesis were depleted in the AP cohort at all time points.</p><p><strong>Conclusions: </strong>Gut dysbiosis persisted following AP in children at 3 and 12 months follow-up compared to HC. Microbiome signatures differed in the ARP cohort and those with multiple GI symptoms.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"690-698"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408973/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540611","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Utility of a noninvasive urine-based test for diagnosing acute pancreatitis in children.","authors":"Tamar Orgad, Isra Abu-Rahma, David Rekhtman, Saar Hashavya, Peri Milman, Mordechai Slae, Zev Davidovics, Michael Wilschanski, Liron Birimberg-Schwartz","doi":"10.1002/jpn3.70119","DOIUrl":"10.1002/jpn3.70119","url":null,"abstract":"<p><strong>Objectives: </strong>Pediatric acute pancreatitis (AP) is a growing clinical concern with a wide spectrum of severity, from mild episodes to life-threatening conditions. Traditional diagnostic methods primarily rely on serum amylase and lipase measurements, which are invasive and can be challenging in children. This study is the first to evaluate the diagnostic accuracy of the urine trypsinogen-2 dipstick test (UTDT) as a noninvasive test for diagnosing pediatric AP.</p><p><strong>Methods: </strong>This prospective study included 28 pediatric patients (31 episodes) presenting with acute abdominal pain at a tertiary medical center from November 2022 to October 2024. AP was diagnosed based on the International Study Group of Pediatric Pancreatitis: In Search for a Cure (INSPPIRE) criteria. Urine samples were collected either within the first 24 hours (h) or later during hospitalization. UTDT sensitivity and specificity were calculated and compared to serum amylase and lipase levels.</p><p><strong>Results: </strong>Of the 31 episodes, 19 (61%) were confirmed as AP, and 12 (39%) were attributed to other causes. The UTDT had an overall sensitivity of 68% and specificity of 100%. Sensitivity increased to 87% when urine samples were collected within 24 h of admission. In non-AP cases, UTDT consistently produced negative results, with the high specificity supporting its reliability in distinguishing AP from other conditions.</p><p><strong>Conclusions: </strong>The UTDT demonstrates promise as a rapid, noninvasive diagnostic tool for pediatric AP, particularly when used early in the disease course. Its high specificity and ease of use suggest that it may serve as an alternative to invasive blood tests once validated through larger-scale studies. Further research is needed to confirm these findings and establish the role of UTDT in clinical practice.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"683-689"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408976/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144317174","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mast cells infiltrates are common in eosinophilic esophagitis and still elevated in histological remission: A digital evaluation in children.","authors":"Theresa Hironimus, Sabrina Degen, Anja Rappl, Ida Allabauer, Joachim Woelfle, Arndt Hartmann, Volker Bruns, Rosalie Kletzander, Carol Geppert, André Hoerning","doi":"10.1002/jpn3.70137","DOIUrl":"10.1002/jpn3.70137","url":null,"abstract":"<p><strong>Objectives: </strong>Eosinophilic esophagitis (EoE) is a type 2 inflammatory chronic allergic disease with several additional immune cell subsets being involved. The aim of this study was to assess the identification and quantification of mast cells (MC) infiltrates using an objective and examiner independent analysis via digital image analysis of digitized histochemically stained biopsies.</p><p><strong>Methods: </strong>Biopsies were taken from the esophagus of 24 children and adolescents diagnosed with EoE and stained for MC and eosinophilic granulocytes using anti-CD117 and Congo red, respectively. Samples were digitized, eosinophilic granulocytes and MCs were quantified using the MIKAIA® image analysis software.</p><p><strong>Results: </strong>At diagnosis MC infiltrations were regularly observed in active disease. MC numbers were 160 (cells/mm²) before therapy initiation and 33 (cells/mm²) in histological remission (<15 Eos/HPF). The number of mucosal MCs at the time of remission decreased less than that of eosinophilic granulocytes, regardless of the initiated therapy. Therefore, patients in histological remission with eosinophilic granulocytes showing on average 2,4 cells/mm² still exhibited MC infiltrations (average 33 cells/mm²). Furthermore, male patients displayed higher numbers of eosinophilic granulocytes at time of diagnosis. With regard to the site of biopsy sampling, an accumulation of the MC count in the distal direction can be observed.</p><p><strong>Conclusions: </strong>Mast cells are involved in EoE and persist after achieving histological remission. Both CD117 + MC and eosinophilic granulocytes can be quantified using MIKAIA® as a tool for objectifying the histological diagnosis of EoE.</p><p><strong>Trial registration: </strong>Identification number: DRKS-ID 00014688; https://drks.de/search/de/trial/DRKS00014688.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"618-625"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408972/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540614","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Specific phenotypes may drive an increased incidence of pediatric inflammatory bowel disease in South-Eastern Norway.","authors":"Svend Andersen, Tomm Bernklev, Ketil Størdal, Milada C Hagen, Vendel A Kristensen, Randi Opheim, Christine Olbjørn, Jon Rove, Emma E Løvlund, Hans K Holm, Batool Aballi, Marte L Høivik, Gøri Perminow","doi":"10.1002/jpn3.70148","DOIUrl":"https://doi.org/10.1002/jpn3.70148","url":null,"abstract":"<p><strong>Objectives: </strong>Pediatric inflammatory bowel disease (PIBD) incidence has increased in recent decades but may be stabilizing, prompting exploration of incidence changes, disease distribution, and severity.</p><p><strong>Methods: </strong>From 2017 to 2019, patients under 18 years with PIBD symptoms were recruited from nine hospitals in South-Eastern Norway for Inflammatory Bowel Disease in South-Eastern Norway III (IBSEN III), a population-based inception cohort study. The primary outcome was a diagnosis of any PIBD subtype as defined by revised Porto criteria. Paris classification system defined disease phenotypes, and descriptions of covariates were gathered from patients in IBSEN III.</p><p><strong>Results: </strong>We identified 324 PIBD patients, with 216 consenting to the IBSEN III study. The crude incidence rate was 17.8 per 100,000 person-years (PY) (95% confidence interval [CI]: 15.9-19.8). Crohn's disease (CD) was found in 118 patients (54.6%); 48% had ileocolonic distribution, 40% had upper gastrointestinal disease, and 12.8% had perianal disease. Complications (stricturing and/or penetrating) were noted in 18%. Ulcerative colitis (UC) was diagnosed in 78 patients (36.1%), predominantly pancolitis (41%), with 30% having proctitis. One in five suffered severe disease. IBD unclassified was found in 20 patients (9.3%). PIBD incidence in those under 16 was 13.6/100,000 PY, up from 4.7 in the IBSEN study 27 years ago. Terminal ileitis (11%-23%) and proctitis (14%-25%) rose from IBSEN (1990-1993) to IBSEN III, while stricturing/penetrating disease changed insignificantly (17%-16%).</p><p><strong>Conclusions: </strong>The incidence of PIBD has risen in South-Eastern Norway, with increased cases of terminal ileitis in CD, unchanged stricturing/penetrating disease, and increased proctitis in UC compared to the original IBSEN study.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier: NCT02727959.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144958217","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Microplate ingestion from E-cigarettes: A true risk for children? A case series.","authors":"Cristina Bucci, Maria Giovanna Puoti, Paolo Quitadamo, Rossella Turco, Sara Isoldi","doi":"10.1002/jpn3.70151","DOIUrl":"10.1002/jpn3.70151","url":null,"abstract":"","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"757-760"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144600829","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The prevalence and predictive factors of overlapping disorders of gut-brain interaction and celiac disease in children.","authors":"Andrew Krueger, Umangi Patel, Jennifer Hardy, Temara Hajjat, Daniel Mallon, Neha Santucci","doi":"10.1002/jpn3.70118","DOIUrl":"10.1002/jpn3.70118","url":null,"abstract":"<p><strong>Objectives: </strong>Disorders of gut-brain interaction (DGBI) are increasing in prevalence; however, diagnosis remains challenging in the setting of organic diseases. While adult studies have shown overlap between DGBI and celiac disease (CeD), no United States studies have assessed DGBI prevalence using Rome IV criteria in pediatric CeD. This study aims to report DGBI prevalence in pediatric CeD patients adherent to gluten-free diet (GFD) with declining serologies and identify common DGBI subtypes and predictive factors for developing DGBI.</p><p><strong>Methods: </strong>Single-center, retrospective study of children (4-21 years old) with biopsy-proven CeD who were evaluated for DGBI based on Rome IV criteria. Patients who were adherent to a GFD, demonstrated tissue transglutaminase immunoglobulin A (TTG IgA) decline, and had at least one visit 9-24-months after diagnosis with a pediatric gastroenterologist were assessed for the presence or absence of gastrointestinal symptoms at all subsequent follow-up visits. Predictive factors associated with DGBI diagnosis were evaluated.</p><p><strong>Results: </strong>Of the 191 pediatric patients included, 43% (n = 83) met Rome IV DGBI diagnostic criteria. Functional constipation (27/83, 33%) and functional abdominal pain (24/83, 29%) were the most common DGBI. Abdominal pain, constipation, and vomiting at initial presentation as well as comorbid joint hypermobility, headaches, and chronic musculoskeletal pain increased risk of developing DGBI after serological decline.</p><p><strong>Conclusions: </strong>DGBI are common in pediatric CeD patients adherent to a GFD with declining TTG IgA. Clinicians should have a high index of suspicion for DGBI in CeD patients with persistent symptoms despite strict GFD adherence to facilitate diagnosis and management.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"596-605"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12265883/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evolution of antibiotic resistance pattern of Helicobacter pylori in Spanish children: A 10-year multicenter study.","authors":"Gonzalo Botija, Josefa Barrio, Beatriz Martínez-Escribano, Miguel Gallardo, Pedro Urruzuno, Natalia Alonso, Julio-Alberto Vázquez, Alfonso Barrio, Aránzazu Recio, Carmen Miranda-Cid, Juana Rizo, María-José González-Abad, Elia Pérez-Fernández, María-Luz Cilleruelo","doi":"10.1002/jpn3.70123","DOIUrl":"10.1002/jpn3.70123","url":null,"abstract":"<p><strong>Objectives: </strong>To delineate the evolution of antibiotic resistance over the past decade in Spanish children diagnosed with Helicobacter pylori (H. pylori) infection.</p><p><strong>Methods: </strong>An observational, retrospective, multicenter study was conducted in Madrid, Spain. This study included children diagnosed with H. pylori infection via endoscopy with positive culture and antimicrobial susceptibility testing between 2011 and 2020.</p><p><strong>Results: </strong>A total of 1205 patients (56.7% female) were included in the study. Of these, 18.7% had previously undergone unsuccessful treatment. The resistance to the antibiotics tested was as follows: clarithromycin, 42.9% (n = 504, [95% confidence interval, CI: 40.1%-45.8%]); metronidazole, 24% (n = 280, [95% CI: 21.5%-26.5%]); rifampicin, 14.8% (n = 120, [95% CI: 12.4%-17.4%]); levofloxacin, 5.2% (n = 58, [95% CI: 3.9%-6.6%]); amoxicillin, 2.6% (n = 30, [95% CI: 1.7%-3.6%]); and tetracycline, 0.9% (n = 10, [95% CI: 0.4%-1.6%]). The double resistance rate was 12.2% (n = 143, [95% CI: 10.4%-14.2%]). During the study period, antibiotic resistance remained relatively stable, with a notable decrease in metronidazole (incidence rate ratio [IRR]: 0.941, [95% CI: 0.898-0.985], p = 0.01) and double resistance (IRR: 0.933, [95% CI: 0.875-0.995], p = 0.03) values. The overall eradication rate was 76.6% (n = 752, [95% CI: 73.8-79.2%]), which was significantly higher in patients without prior treatment. The temporal progression of eradication rates showed a substantial increase, with an average annual increase of 2.9% (IRR: 1.029, [95% CI: 1.015-1.043], p < 0.001).</p><p><strong>Conclusions: </strong>The prevalence of antibiotic resistance in our setting (Madrid, Spain) was remarkably high and remained stable throughout the study period, except for a notable decline in metronidazole and double resistance.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"514-522"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333348","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advances in prognostic biomarkers for biliary atresia: Current insights and future directions.","authors":"Ahmad Anouti, Pavithra Sudhakara, Chelsea Pratt, Reena Mourya, Lisa VanWagner, Pranavkumar Shivakumar, Sindhu Pandurangi","doi":"10.1002/jpn3.70131","DOIUrl":"10.1002/jpn3.70131","url":null,"abstract":"<p><p>Biliary atresia (BA) is a progressive, fibrosing cholangiopathy of infancy characterized by inflammatory obstruction of the bile ducts, ultimately leading to end-stage liver disease if untreated. Early diagnosis and timely surgical intervention via hepatoportoenterostomy (HPE) are critical for improving outcomes; however, prognostication remains challenging due to heterogeneous responses to surgery and variable clinical trajectories. This review provides a comprehensive synthesis of current research on prognostic biomarkers in BA, encompassing clinical indicators, routine laboratory parameters, novel serum biomarkers, histopathologic features, hepatic gene expression profiles, imaging modalities, and both in vitro and computational prognostic modeling systems. While traditional clinical factors, such as age at HPE and postoperative serum bilirubin levels, continue to serve as important predictors of outcome, they lack sufficient discriminatory power for individualized risk stratification. Recent advances have identified emerging biomarkers, including inflammatory cytokines, immune activation markers, and indicators of fibrosis and extracellular matrix remodeling, which show potential in correlating with disease progression and native liver survival. Imaging modalities such as ultrasound elastography have also demonstrated promise in noninvasively assessing liver stiffness and predicting clinical outcomes. Furthermore, the identification of hepatic gene expression signatures and multigene prognostic classifiers offers new avenues for precision risk assessment. However, most of these advancements have not translated into clinical practice due to small sample sizes and limited external validation. Future research efforts must focus on large-scale, multicenter studies to validate findings and establish robust, integrative prognostic models that can inform clinical decision-making and facilitate personalized therapeutic strategies in BA.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"497-506"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408957/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144506030","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Paediatric acute liver failure: A prospective, nationwide, population-based surveillance study in Germany.","authors":"Dominic Lenz, Muhammad Abdulaziz, Bianca Peters, Matias Wagner, Lea D Schlieben, Victor M Corman, Ulrich Baumann, Philip Bufler, Tal Dattner, Rainer Ganschow, Kristin Genzel, Nicole Hammann, Steffen Hartleif, Bianca Hegen, Stephan Henning, André Hoerning, Martin Jankofsky, Norman Junge, Simone Kathemann, Birgit Knoppke, Martina Kohl-Sobania, Martin Laass, Elke Lainka, Eberhard Lurz, Michael Melter, Hanna Müller, Denisa Pilic, Markus Ries, Lisa Schiefele, Tobias Schwerd, Ekkehard Sturm, Mechtild Wegner, Michael S Urschitz, Sven F Garbade, Daniel Wenning, Christian Drosten, Alexander Fichtner, Stefan Kölker, Georg F Hoffmann, Holger Prokisch, Christian Staufner","doi":"10.1002/jpn3.70149","DOIUrl":"10.1002/jpn3.70149","url":null,"abstract":"<p><strong>Objectives: </strong>Paediatric acute liver failure (PALF) is a rare but life-threatening condition, yet comprehensive epidemiological data in Germany are lacking. Our study aimed to systematically analyse incidence, aetiology, and outcome of PALF in Germany.</p><p><strong>Methods: </strong>In a nationwide, population-based surveillance study, cases of PALF (defined following the PALF study group inclusion criteria) were queried from 2016 to 2018 through the German Paediatric Surveillance Unit (ESPED). Demographic, clinical, laboratory, therapeutic, and outcome data were collected and analysed. In case of unexplained aetiology, whole exome and virus sequencing was offered as a complementary diagnostic.</p><p><strong>Results: </strong>Over the 3-year period, 148 cases were reported, yielding an estimated incidence of 3.7 per 1 million children per year. Neonates and infants were predominantly affected (45% of the cases); median age at PALF was 1.2 years (range: 0-17.9 years). Metabolic/genetic diseases were the most common cause (23%), followed by infectious causes (17%). The overall diagnostic yield was 73%, diagnosis remained unknown in 40 cases. Clinical outcome was age-dependent: new-borns showed a significant higher lethality (42%), followed by infants (29%), toddlers (15%), and school children (12%). Liver transplantation was reported in 22% of cases.</p><p><strong>Conclusions: </strong>This study provides comprehensive insights into PALF epidemiology in Germany. Metabolic/genetic causes and infectious diseases were most common. Advances in standardised diagnostic work-up and genetic analysis have enhanced diagnostic yield, yet mortality remains substantial, particularly among neonates. Further research is warranted to improve diagnostic accuracy, therapeutic outcomes, and overall management of PALF.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"653-662"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408955/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575712","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcome reporting in studies of paediatric achalasia: A systematic review.","authors":"Jonathan J Neville, Sierra Schaffer, Simon Eaton, Nigel J Hall","doi":"10.1002/jpn3.70128","DOIUrl":"10.1002/jpn3.70128","url":null,"abstract":"<p><strong>Objectives: </strong>Paediatric achalasia is a rare condition associated with significant morbidity. A core outcome set (COS) would standardise reporting, enable comparison of data sets, and focus research efforts; ultimately improving care for children with achalasia. We aimed to identify outcomes currently reported in studies of paediatric achalasia to inform outcomes for a COS.</p><p><strong>Methods: </strong>A systematic review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-analysis guidelines. Studies investigating children ≤18 years of age with a diagnosis of achalasia were included. Primary and secondary outcomes were recorded and assigned to OMERACT core areas. The study was pre-registered (PROSPERO: CRD42024509855).</p><p><strong>Results: </strong>Sixty-two studies were included in this review, consisting of 54 retrospective and 8 prospective studies. Median cohort size was 20 patients (inter-quartile range: 13-28). Forty-eight unique outcomes were reported. The most common outcomes reported were intra-operative complications (65%, 40 studies), post-operative complications (58%, 36 studies) and length of stay (58%, 36 studies). A primary outcome was specified in 12 studies (19%), the most common was the Eckardt score (13%) in 8 studies. Studies least frequently reported outcomes in the death (21%, 13 studies) and pathophysiological manifestations (35%, 22 studies) core areas.</p><p><strong>Conclusions: </strong>The studies included in this review were predominantly small and retrospective. Of the few studies that specified a primary outcome, the majority used the Eckardt score, which is unvalidated in children. Outcomes relevant to pathophysiological manifestations, life impact and survival were under-reported. A COS for paediatric achalasia, involving key stakeholders, would ensure that patient-relevant outcomes were reported, reduce heterogeneity and facilitate meta-analysis.</p>","PeriodicalId":16694,"journal":{"name":"Journal of Pediatric Gastroenterology and Nutrition","volume":" ","pages":"523-529"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12408982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368923","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}