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Liver transplantation (LT) for children with urea cycle disorders (UCDs) is capable of correcting the enzymatic defect and preventing progressive neurologic injury. We describe the characteristics and outcomes of pediatric LT recipients with UCDs. We identified all pediatric (

Congenital hepatic fibrosis (CHF) is a hereditary fibrocystic disease that can progress to portal hypertension and recurrent cholangitis requiring liver transplantation (LT). It can be associated with renal pathology and need for kidney transplantation (KT). We describe the clinical characteristics and outcomes of patients undergoing liver transplantation alone (LTA) and simultaneous liver-kidney transplantation (SLKT) for CHF using the Unites States Scientific Registry of Transplant Recipients. A total of 197 patients who received LT for CHF between 2002 and 2018 were identified - 87 (44.2%) received SLKT, 110 (55.8%) received LTA. The 1-, 3- and 5-year patient survival were 99.0%, 96.2% and 94.6%. The 1-, 3- and 5-year liver graft survival were 94.9%, 91.1% and 89.6%. No significant differences in patient or liver graft survival were observed between the SLKT and LTA groups, or between paediatric and adult recipients. 53.3% of patients with CHF necessitating LT also have significant renal disease requiring KT. Kidney graft survival for isolated KT prior to LT were poorer compared with KT performed simultaneously or after LT. Both LTA and SLKT for CHF are associated with excellent long-term outcomes in paediatric and adult patients.

Non-alcoholic fatty liver disease (NAFLD) is becoming an increasingly important healthcare issue along with the rising rates of obesity worldwide. It is the most common chronic liver disease in the paediatric population and the fastest growing indication for liver transplant in young adults. The pathogenesis is complex with contributions from multiple factors and genetic predisposition. While non-invasive laboratory tests and imaging modalities are being increasingly used, the liver biopsy continues to play a crucial role in the diagnosis and prognosis of NAFLD. Histologically, the assessment of paediatric fatty liver disease requires special considerations with respect to a periportal predominant pattern seen in prepubertal patients, as well as a different set of disease processes in the differential diagnosis. In this review, we provide a summary of current knowledge on the epidemiology, pathogenesis and clinical course of paediatric NAFLD as well as the clinical guidelines on diagnosis and management. We discuss the indications and limitations of liver biopsy, histological patterns seen in paediatric NAFLD, other entities to be considered in the differential diagnosis, and conclude with appropriate triaging of liver biopsies and essential elements of pathology reporting.

Liver transplantation (LT) is offered in cases of advanced disease for both pediatric patients with hepatoblastoma (HBL) and those with hepatocellular carcinoma (HCC). Current United States organ allocation priorities differ between the two groups.We retrospectively examined the waitlist and posttransplant outcomes of pediatric LT candidates with HBL and HCC using the United Network for Organ Sharing registry (February 2002 to September 2020).Six hundred sixty-eight children with HBL and 95 children with HCC listed for first LT were identified. Patients with HBL were younger (p .001), had lower laboratory Model for End-stage Liver Disease (MELD)/Pediatric End-stage Liver Disease (PELD) scores (p .001), and had lesser proportion with encephalopathy (p = .01). Patients with HCC had an increased risk of waitlist mortality in univariable (unadjusted subdistribution hazard ratio [sHR] = 4.37, 95% confidence interval [CI], 2.01-9.51, p .001) and multivariable competing risk regression (adjusted sHR = 3.08, 95% CI 1.13-8.37, p = .03) accounting for age and laboratory MELD/PELD score. Five hundred ninety-five children underwent LT for HBL and 76 for HCC. Patients transplanted for HBL had a significantly higher proportion with status 1B exception (71.3% vs. 7.9%, p .001). No difference was observed in patient (unadjusted log-rank test, p = .52; adjusted hazard ratio [HR] = 0.77, 95% CI, 0.40-1.48, p = .43) or graft survival (unadjusted log-rank test, p = .93; adjusted HR = 0.74, 95% CI 0.42-1.33, p = .32) between HCC and HBL recipients.Waitlist mortality for pediatric LT candidates with HCC is significantly higher than for HBL, while posttransplant patient and graft survival are similar. This highlights an opportunity to improve equitable prioritization for children with HCC who may have reduced access to size-appropriate deceased donor organs and less effective bridge-to-transplant therapies.

We compared the waitlist mortality of 114 adolescent (12–17 years) and 217 adult (≥ 18 years) biliary atresia candidates, and the post-transplant survival of 117 adolescent and 160 adult biliary atresia liver transplant recipients using Scientific Registry of Transplant Recipients data (2002-2018). After adjusting for Model for End-stage Liver Disease score (P = 0.001) and ascites, portal vein thrombosis, encephalopathy, life support at listing (all P ≥ 0.09), adults demonstrated 10.9 times higher risk of waitlist mortality vs. adolescents (95 per cent c.i. 1.55 to 76.46; P = 0.02). Adolescents exhibited superior patient (P = 0.007) and graft survival (P = 0.006).

Hepatoblastoma is the most common type of liver cancer in children. Refined therapeutic approaches combining risk-adapted chemotherapy along with complete tumor resection has led to improved survival. We aimed to evaluate the current state of management and outcomes for hepatoblastoma in the United States.We retrospectively reviewed 794 children (18 years) with hepatoblastoma from the National Cancer Database (2004-2015). We assessed overall survival by means of Kaplan-Meier method, log-rank tests, and multivariable Cox regression.Median age was 1 year (interquartile range: 0-2) and 170 (21.4%) presented with metastatic disease. Surgical resection was included in the treatment of 614 (77.3%) children (resection in 66.8% and liver transplantation in 10.6%). In the entire cohort, 95.1% of children received chemotherapy. In the surgical cohort, 575 (93.6%) received chemotherapy (34.5% neoadjuvant, 28.7% adjuvant, 30.5% both neoadjuvant and adjuvant). The 5-year overall survival was 76.6% for the entire cohort (no-surgery group: 55.3% vs surgery group: 82.8%). In multivariable analysis for all children, age ≥8 years (P = .009), metastasis (P.001), surgery only (P = .009), and chemotherapy only (P.001) were risk factors for mortality. In multivariable analysis for the surgical cohort, metastasis (P = .001), multifocality (P = .02), no chemotherapy (P = .03), and margin-positive resection (P = .02) were risk factors for mortality.Excellent long-term overall survival is achievable with a combination of chemotherapy and surgical resection when a negative resection margin is achieved. However, nearly a quarter of children never received surgical treatment, representing a potential opportunity for improvement in care.

Purpose This study evaluates overall survival (OS) between liver transplantation (LT) and liver resection (LR), while assessing the effect of margin status, in children with hepatocellular carcinoma (HCC). Methods The National Cancer Database was queried (2004–2015) for children ( Results One hundred six children with HCC treated surgically (LT 34, LR 72) were identified. For T1 stage, no difference in OS was observed for LT vs. margin-negative liver resection [LR(−)] (log-rank, p = 0.47). For T2/T3/T4 stage, no difference in OS was observed for LT vs. LR(−) (log-rank, p = 0.08); both subgroups exhibited superior OS vs. margin-positive liver resection [LR(+)] (log-rank, LT vs. LR(+): p = 0.001 and LR(−) vs. LR(+): p = 0.04). On multivariable Cox regression: (i) histology (fibrolamellar vs. not) and T stage (T1 vs. T2/T3/T4) were not associated with OS (both p = 0.06), (ii) chemotherapy and size > 5 cm were not associated with OS (both p ≥ 0.42), (iii) when compared to LT, both LR(−) (p = 0.03) and LR(+) (p = 0.001) were associated with increased likelihood of mortality. Conclusion Although margin-negative resection may be obtained with LT or LR, early LT consultation is warranted for children at high risk of LR(+) regardless of Milan criteria due to the negative impact of LR(+) on OS. Type of study Retrospective cohort study. Level of evidence III

There is a broad clinical spectrum of gastrointestinal bleeding in children, ranging from subtle laboratory findings to dramatic clinical presentations. This review provides a framework for the evaluation and management of gastrointestinal hemorrhage for pediatricians. It outlines strategies for obtaining a tailored patient history and conducting a thorough physical examination that can shed light on the location, severity, and likely etiology of bleeding. It appraises blood tests, radiologic tools, and endoscopic modalities frequently used to identify and control a source of bleeding.

OBJECTIVES Variceal hemorrhage (VH) is a serious complication of portal hypertension (PH). We evaluated the feasibility, safety, and clinical impact of esophageal capsule endoscopy (ECE) in pediatric and young adult patients with known or suspected PH. METHODS Children and young adults with PH at Boston Children's Hospital (2005-2017) were offered ECE for variceal screening or surveillance. Patient histories, ECE findings, and clinical outcomes were reviewed retrospectively. RESULTS One hundred and forty-nine ECE studies were performed in 98 patients (57.1% male patients) using 3 ECE devices for variceal screening (66.5%) or surveillance (33.5%). Three readers interpreted the studies (88.3%, 10.3%, and 1.4%, respectively). Median age was 16 years (IQR 13.7-18.5). One hundred and three ECE studies involved patients