J Pediatr Gastroenterol Nutr
January 2025
Pediatric cholestatic liver diseases are rare conditions that can result from multiple specific underlying etiologies. Among the most common etiologies of pediatric cholestatic liver diseases are biliary atresia, Alagille syndrome (ALGS), and inherited disorders of bile acid transport. These diseases are characterized by episodic or chronic unremitting cholestasis.
View Article and Find Full Text PDFBackground: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a common disease in children. Lifestyle modification is the primary treatment but difficult to achieve and maintain. Topiramate is a component of an approved weight loss medication (topiramate-phentermine) in children aged 12 years and older but is more commonly used as a single agent, off-label, for pediatric obesity.
View Article and Find Full Text PDFJ Pediatr Gastroenterol Nutr
January 2025
Pediatric obesity, characterized by a body mass index (BMI) at or above the 95th percentile for age, affects a substantial number of children and adolescents worldwide. Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as nonalcoholic fatty liver disease, represents a prominent hepatic manifestation of obesity and metabolic syndrome, emerging as the most prevalent hepatic disorder among pediatric patients and a significant contributor to liver transplantation in adults. The escalating prevalence of pediatric MASLD mirrors the alarming rise in childhood obesity rates over recent decades.
View Article and Find Full Text PDFBackground And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.
View Article and Find Full Text PDFObjective: To determine the association between food insecurity and pediatric nonalcoholic fatty liver disease (NAFLD).
Methods: Cross-sectional study of patients < 21 years of age with histologically confirmed NAFLD. The Household Food Security Survey Module was administered to determine food insecurity status.
Importance: Live vaccines (measles-mumps-rubella [MMR] and varicella-zoster virus [VZV]) have not been recommended after solid organ transplant due to concern for inciting vaccine strain infection in an immunocompromised host. However, the rates of measles, mumps, and varicella are rising nationally and internationally, leaving susceptible immunocompromised children at risk for life-threating conditions.
Objective: To determine the safety and immunogenicity of live vaccines in pediatric liver and kidney transplant recipients.
Background: Chronic kidney disease (CKD) impacts long-term morbidity in pediatric liver transplant (LT) recipients. The prevalence of estimated glomerular filtration rate (eGFR) < 90 mL/min/1.73 m (eGFR < 90) at our institution was 25% at 1 year post-LT; thus, quality improvement (QI) project was initiated, aiming to decrease the prevalence of eGFR < 90 by at least 20% at 1 year-post LT.
View Article and Find Full Text PDFJ Clin Transl Hepatol
June 2022
Recent reports of acute hepatitis of unknown origin in previously healthy children have been increasing worldwide. The main characteristics of the affected children were jaundice and gastrointestinal symptoms. Their serum aminotransaminase levels were above 500 IU/L, with negative tests for hepatitis viruses A-E.
View Article and Find Full Text PDFIn this cross-sectional study, serum matrix metalloproteinase-7 levels were significantly lower in infants with jaundice and parenteral nutrition-associated liver disease compared with those with confirmed biliary atresia. Serum metalloproteinase-7 may aid in excluding biliary atresia and thus may minimize invasive testing in infants with a history of parenteral nutrition.
View Article and Find Full Text PDFNonalcoholic fatty liver disease (NAFLD) is the most common type of chronic liver disease in children. The mechanisms that drive NAFLD disease progression in this specific patient population remain poorly defined. In this study, we obtained liver biopsy samples from a multiethnic cohort of pediatric patients with NAFLD (n = 52, mean age = 13.
View Article and Find Full Text PDFBackground: This study aimed to characterize features present at the time of diagnosis and describe outcomes in patients with post-transplant lymphoproliferative disorder (PTLD) following pediatric solid organ transplantation.
Methods: We performed a retrospective review of solid organ transplant patients who developed pathologically confirmed PTLD at our center from 2006 to 2016.
Results: Of 594 patients included in this study, 41(6.
D-bifunctional protein (DBP) deficiency is a rare, autosomal recessive peroxisomal enzyme deficiency resulting in a high burden of morbidity and early mortality. Patients with DBP deficiency resemble those with a severe Zellweger phenotype, with neonatal hypotonia, seizures, craniofacial dysmorphisms, psychomotor delay, deafness, blindness, and death typically within the first 2 years of life, although patients with residual enzyme function can survive longer. The clinical severity of the disease depends on the degree of enzyme deficiency.
View Article and Find Full Text PDFCongenital tufting enteropathy (CTE) is a rare heritable cause of intractable diarrhea due to EPCAM mutation. Pathologic findings include intestinal villous atrophy, tufted discohesive tear-drop-shaped epithelium, and a normal brush border. In affected patients, absent intestinal epithelial cell adhesion molecule (EpCAM) expression results in loss of MOC31 immunostaining.
View Article and Find Full Text PDFTE measures liver stiffness to assess fibrosis. Its use in post-transplant patients was reported in few small pediatric studies. We evaluated TE ability to predict liver graft fibrosis in a large cohort while comparing it to the performance of APRI and FIB-4.
View Article and Find Full Text PDFA 20-year-old male presented 3.5 years after intestinal transplantation with rapidly progressive sensorineural hearing loss. Initial brain imaging was consistent with inflammation and/or demyelination.
View Article and Find Full Text PDFIn areas of the world where human herpesvirus 8 (HHV-8) is endemic, Kaposi sarcoma (KS) is a common SOT-associated cancer. In the United States, where the virus is not prevalent, PTKS is rare, and there is little literature on pediatric PTKS. We present a North American female who underwent deceased donor, left lateral segment liver transplant for biliary atresia at age 11 months.
View Article and Find Full Text PDFStandard management of intra-abdominal pediatric solid tumors requires complete resection. However, tumors with multiple organ and vascular involvement present a unique surgical challenge. We conducted a retrospective chart review of four patients, aged 2-14 years, undergoing MVT for intra-abdominal tumors with significant involvement of the visceral arteries and/or portomesenteric venous system at our institution.
View Article and Find Full Text PDFJ Pediatr Gastroenterol Nutr
April 2017
Objectives: The aim of the study was to evaluate whether liver stiffness measurement (LSM), determined by transient elastography, correlates with presence and severity of liver disease in children and young adults with cystic fibrosis (CF).
Methods: Subjects underwent LSM at routine CF visits. Presence and severity of cystic fibrosis liver disease (CFLD) was determined by clinical parameters.
J Pediatr Gastroenterol Nutr
January 2017
Cholestatic jaundice in infancy affects approximately 1 in every 2500 term infants and is infrequently recognized by primary providers in the setting of physiologic jaundice. Cholestatic jaundice is always pathologic and indicates hepatobiliary dysfunction. Early detection by the primary care physician and timely referrals to the pediatric gastroenterologist/hepatologist are important contributors to optimal treatment and prognosis.
View Article and Find Full Text PDFWe present a case of a three-yr-old child with a history of multisystem Langerhans cell histiocytosis treated with systemic chemotherapy, who developed progressive liver failure and received an orthotopic split liver transplant while continuing on chemotherapy. One month following transplant, he developed acute graft-vs.-host disease of the skin and gastrointestinal tract.
View Article and Find Full Text PDFJ Pediatr Gastroenterol Nutr
August 2006
Introduction: Chronic hepatitis C virus (HCV) infection affects 0.3% of children in the United States, and the general impression is that it has a benign course in childhood. We analyzed a pediatric population with chronic HCV in a quaternary referral center.
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