Cholangioscopy in Children and Adolescents: Utilization, Outcomes, and Safety.

Background: Endoscopic retrograde cholangiopancreatography (ERCP) is increasingly utilized for management of biliary disorders in children and adolescents. Practice patterns surrounding cholangioscopy in pediatric patients, however, are largely uncharacterized.

Methods: We retrospectively analyzed all ERCPs in which cholangioscopy was performed on patients 18 and under at our tertiary care children's hospital from 2015 to 2020 using our institution's paper and electronic medical record system.

Nationwide Evolution of Pediatric Endoscopic Retrograde Cholangiopancreatography Indications, Utilization, and Readmissions over Time.

Objectives: To analyze outcome and utilization trends over time of pediatric endoscopic retrograde cholangiopancreatography (ERCP) in an all-capture US population-level study.

Study Design: Using the National Inpatient Sample (2005-2014) and National Readmission Database (2010-2014), we identified pediatric (age <20 years) hospitalizations during which ERCP was performed and assessed ERCP-associated readmissions. International Classification of Diseases, Ninth Revision, Clinical Modification codes were used to identify hospitalization diagnoses, comorbidities, and patient/hospital characteristics.

Fluoroscopy Time During Endoscopic Retrograde Cholangiopancreatography Performed for Children and Adolescents is Significantly Higher With Low-volume Endoscopists.

Background: Endoscopic retrograde cholangiopancreatography (ERCP) is a fluoroscopy and endoscopy-based procedure important for diagnosis and management of pediatric pancreaticobiliary disorders. Patient, procedure, endoscopist, and facility characteristics have been shown to influence ERCP complexity and procedure outcomes as well as fluoroscopy utilization in adults; however, the extent to which this is true in pediatric patients remains under-studied and there are minimal data regarding fluoroscopy utilization in pediatric ERCP.

Methods: We retrospectively analyzed ERCPs performed on patients <18 years of age at our tertiary care children's hospital from 2002 to 2017 using our institution's paper and electronic medical record system along with a prospectively maintained radiation exposure database.

Open-label prospective therapeutic clinical trials: oral vancomycin in children and adults with primary sclerosing cholangitis.

Background: Oral vancomycin (OV) in primary sclerosing cholangitis (PSC) has been evaluated as a potential therapeutic agent. We report the long-term biochemical course and outcomes of patients with PSC treated with OV.

Methods: Patients were enrolled in 2 open-label clinical trials (ClinicalTrials.

Successful liver transplantation in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE).

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a fatal disorder characterized by progressive gastrointestinal dysmotility, peripheral neuropathy, leukoencephalopathy, skeletal myopathy, ophthalmoparesis, and ptosis. MNGIE stems from deficient thymidine phosphorylase activity (TP) leading to toxic elevations of plasma thymidine. Hematopoietic stem cell transplant (HSCT) restores TP activity and halts disease progression but has high transplant-related morbidity and mortality.

Pediatric Endoscopy Practice Patterns in the United States, Canada, and Mexico.

Background And Aims: Endoscopic procedures are important for diagnosis and management of many gastrointestinal, liver, and biliary conditions in children. Therapeutic endoscopy procedures, including endoscopic retrograde cholangiopancreatography (ERCP), are performed less frequently in children relative to adults. A formal study to evaluate institutional volumes and practice patterns for advanced therapeutic pediatric endoscopy procedures has, however, not been previously undertaken.

Prenatal treatment of ornithine transcarbamylase deficiency.

Purpose Of Study: Patients with neonatal urea cycle defects (UCDs) typically experience severe hyperammonemia during the first days of life, which results in serious neurological injury or death. Long-term prognosis despite optimal pharmacological and dietary therapy is still poor. The combination of intravenous sodium phenylacetate and sodium benzoate (Ammonul®) can eliminate nitrogen waste independent of the urea cycle.

Initial experience with peroral endoscopic myotomy for treatment of achalasia in children.

Background: Achalasia is a primary esophageal motility disorder characterized by aperistalsis of the esophagus and failed relaxation of the lower esophageal sphincter that presents rarely in childhood. The peroral endoscopic myotomy (POEM) procedure is an emerging treatment for achalasia in adults that has recently been introduced into pediatric surgical practice.

Methods: This is a prospective case series of all children referred to Stanford University Lucile Packard Children's Hospital with manometry-confirmed achalasia who underwent a POEM procedure from 2014 to 2016.

Mass cytometry reveals a distinct immunoprofile of operational tolerance in pediatric liver transplantation.

Long-term IS in transplant patients has significant morbidity, poorer quality of life, and substantial economic costs. TOL, defined as graft acceptance without functional impairment in the absence of IS, has been achieved in some pediatric LT recipients. Using mass cytometry, peripheral blood immunotyping was performed to characterize differences between tolerant patients and patients who are stable on single-agent IS.

Protein and calorie intakes in adult and pediatric subjects with urea cycle disorders participating in clinical trials of glycerol phenylbutyrate.

Background: Little prospectively collected data are available comparing the dietary intake of urea cycle disorder (UCD) patients to UCD treatment guidelines or to healthy individuals.

Objective: To examine the protein and calorie intakes of UCD subjects who participated in clinical trials of glycerol phenylbutyrate (GPB) and compare these data to published UCD dietary guidelines and nutritional surveys.

Design: Dietary data were recorded for 45 adult and 49 pediatric UCD subjects in metabolic control during participation in clinical trials of GPB.

Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis.

Neonatal cholestasis is a potentially life-threatening condition requiring prompt diagnosis. Mutations in several different genes can cause progressive familial intrahepatic cholestasis, but known genes cannot account for all familial cases. Here we report four individuals from two unrelated families with neonatal cholestasis and mutations in NR1H4, which encodes the farnesoid X receptor (FXR), a bile acid-activated nuclear hormone receptor that regulates bile acid metabolism.

Glutamine and hyperammonemic crises in patients with urea cycle disorders.

Unlabelled: Blood ammonia and glutamine levels are used as biomarkers of control in patients with urea cycle disorders (UCDs). This study was undertaken to evaluate glutamine variability and utility as a predictor of hyperammonemic crises (HACs) in UCD patients.

Methods: The relationships between glutamine and ammonia levels and the incidence and timing of HACs were evaluated in over 100 adult and pediatric UCD patients who participated in clinical trials of glycerol phenylbutyrate.

Self-reported treatment-associated symptoms among patients with urea cycle disorders participating in glycerol phenylbutyrate clinical trials.

Background: Health care outcomes have been increasingly assessed through health-related quality of life (HRQoL) measures. While the introduction of nitrogen-scavenging medications has improved survival in patients with urea cycle disorders (UCDs), they are often associated with side effects that may affect patient compliance and outcomes.

Methods: Symptoms commonly associated with nitrogen-scavenging medications were evaluated in 100 adult and pediatric participants using a non-validated UCD-specific questionnaire.

Text Messaging Improves Participation in Laboratory Testing in Adolescent Liver Transplant Patients.

Background: In solid organ transplant patients, non-participation in all aspects of the medical regimen is a prevalent problem associated with adverse consequences particularly in the adolescent and young adult (AYA) age group. This study is the first to evaluate the feasibility, utility and impact of a text messaging (TM) intervention to improve participation in laboratory testing in adolescent liver transplant patients.

Methods: AYA patients, aged 12 to 21 years, were recruited for a prospective pilot trial evaluating a TM intervention delivered over a 1-year period.

Blood ammonia and glutamine as predictors of hyperammonemic crises in patients with urea cycle disorder.

Purpose: The aim of this study was to examine predictors of ammonia exposure and hyperammonemic crises in patients with urea cycle disorders.

Methods: The relationships between fasting ammonia, daily ammonia exposure, and hyperammonemic crises were analyzed in >100 patients with urea cycle disorders.

Results: Fasting ammonia correlated strongly with daily ammonia exposure (r = 0.

Novel protocol including liver biopsy to identify and treat CD8+ T-cell predominant acute hepatitis and liver failure.

In the majority of children with ALF, the etiology is unknown and liver transplantation is often needed for survival. A patient case prompted us to consider that immune dysregulation may be the cause of indeterminate acute hepatitis and liver failure in children. Our study includes nine pediatric patients treated under a multidisciplinary clinical protocol to identify and treat immune-mediated acute liver injury.

Elevated phenylacetic acid levels do not correlate with adverse events in patients with urea cycle disorders or hepatic encephalopathy and can be predicted based on the plasma PAA to PAGN ratio.

Background: Phenylacetic acid (PAA) is the active moiety in sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB, HPN-100). Both are approved for treatment of urea cycle disorders (UCDs) - rare genetic disorders characterized by hyperammonemia. PAA is conjugated with glutamine in the liver to form phenylacetyleglutamine (PAGN), which is excreted in urine.