Enhanced Donor Antigen Presentation by B Cells Predicts Acute Cellular Rejection and Late Outcomes After Transplantation.

Background: Enhanced B-cell presentation of donor alloantigen relative to presentation of HLA-mismatched reference alloantigen is associated with acute cellular rejection (ACR), when expressed as a ratio called the antigen presenting index (API) in an exploratory cohort of liver and intestine transplant (LT and IT) recipients.

Methods: To test clinical performance, we measured the API using the previously described 6-h assay in 84 LT and 54 IT recipients with median age 3.3 y (0.

Using social media for patient care, research, and professional development: A North American Society of Pediatric Gastroenterology, Hepatology, and Nutrition position paper.

The advent of social media has changed numerous aspects of modern life, with users developing and maintaining personal and professional relationships, following and sharing breaking news and importantly, searching for and disseminating health information and medical research. In the present paper, we reviewed available literature to outline the potential uses, pitfalls and impacts of social media for providers, scientists and institutions involved in digestive health in the domains of patient care, research and professional development. We recommend that these groups become more active participants on social media platforms to combat misinformation, advocate for patients, and curate and disseminate valuable research and educational materials.

Mutations in latent membrane protein 1 of Epstein-Barr virus are associated with increased risk of posttransplant lymphoproliferative disorder in children.

Epstein-Barr virus (EBV)-positive posttransplant lymphoproliferative disorder (PTLD) results in significant morbidity and mortality in pediatric transplant recipients. Identifying individuals at an increased risk of EBV-positive PTLD could influence clinical management of immunosuppression and other therapies, improving posttransplant outcomes. A 7-center prospective, observational clinical trial of 872 pediatric transplant recipients evaluated the presence of mutations at positions 212 and 366 of EBV latent membrane protein 1 (LMP1) as an indicator of risk of EBV-positive PTLD (clinical trials: NCT02182986).

Domino liver transplant from a donor with maple syrup urine disease into a recipient with phenylketonuria.

Classical phenylketonuria (PKU) presents a unique challenge for women of child-bearing age. In the context of pregnancy, poorly controlled hyperphenylalaninemia can result in a devastating constellation of outcomes for the baby referred to as the maternal PKU Syndrome. We present the case of a woman with classical PKU unable to maintain a restricted diet and refractory to pharmacological therapies.

Sarcopenia prevalence in pediatric intestinal transplant recipients: Implications on post-transplant outcomes.

Background: Sarcopenia has been associated with poor surgical outcomes but has not been studied in pediatric intestinal transplantation. We aimed to determine sarcopenia prevalence in intestinal transplant recipients and the association of sarcopenia with outcomes.

Methods: We performed a cross-sectional retrospective chart review of intestinal transplant recipients from 2000-present.

Addressing Adolescent Relationship Abuse in the Context of Reproductive Health Care.

Adolescent relationship abuse (ARA) is a significant public health issue that includes physical, sexual, psychological and cyber abuse, reproductive coercion, and/or sexual exploitation within an intimate relationship in which one or both partners is a minor. ARA is associated with numerous negative outcomes that include all domains of health. Many negative outcomes of ARA are related to reproductive and sexual health (RSH); thus, reproductive health care providers must be equipped to recognize and address ARA.

Domino transplantation for pediatric liver recipients: Obstacles, challenges, and successes.

Background: Domino liver transplantation aims to address the need to increase the liver donor supply. In a domino liver transplant, the domino recipient receives the explanted liver from the recipient of a traditional liver transplant. The domino donor typically requires liver transplant to correct a metabolic disorder; the explanted liver thus has a single gene defect but otherwise normal structure and function.

Distinct interferon signatures and cytokine patterns define additional systemic autoinflammatory diseases.

BACKGROUNDUndifferentiated systemic autoinflammatory diseases (USAIDs) present diagnostic and therapeutic challenges. Chronic interferon (IFN) signaling and cytokine dysregulation may identify diseases with available targeted treatments.METHODSSixty-six consecutively referred USAID patients underwent underwent screening for the presence of an interferon signature using a standardized type-I IFN-response-gene score (IRG-S), cytokine profiling, and genetic evaluation by next-generation sequencing.

Effect of Surgical Versus Medical Therapy on Diabetic Kidney Disease Over 5 Years in Severely Obese Adolescents With Type 2 Diabetes.

Objective: To compare diabetic kidney disease (DKD) rates over 5 years of follow-up in two cohorts of severely obese adolescents with type 2 diabetes (T2D) undergoing medical or surgical treatment for T2D.

Research Design And Methods: A secondary analysis was performed of data collected from obese participants of similar age and racial distribution enrolled in the Teen-Longitudinal Assessment of Bariatric Surgery (Teen-LABS) and the Treatment Options of Type 2 Diabetes in Adolescents and Youth (TODAY) studies. Teen-LABS participants underwent metabolic bariatric surgery (MBS).

[Percutaneous management of pulmonary atresia with intact ventricular septum and critical pulmonary stenosis].

Introduction: Pulmonary atresia with intact ventricular septum and critical pulmonary stenosis in newborns encompasses a wide spectrum of disease, including cases with significant right ventricular hypoplasia and coronary artery to right ventricle fistulae, which may be considered a contraindication for decompression of the right ventricle. The aim of this study was to review the middle- and long-term outcomes of these patients over 20 years and identify differential factors between both groups, including patients with coronary artery fistulae.

Patients And Methods: We performed a descriptive retrospective study by identifying all patients that received a diagnosis of pulmonary atresia with intact ventricular septum and critical pulmonary stenosis between January 1996 and January 2018.

Effective and safe tumor inhibition using vinblastine in medulloblastoma.

Most medulloblastoma protocols worldwide include vincristine during radiation and chemotherapy. A significant dose-limiting toxicity is peripheral neuropathy; however, there is a paucity of data to support the view that omission of vincristine does not impact survival. Herein we report two adolescent patients with Group 4 and SHH medulloblastoma, where vinblastine successfully replaced vincristine with resolution of their peripheral neuropathy.

Cytomegalovirus in Pediatric Hematopoietic Stem Cell Transplantation: A Case-Based Panel Discussion of Current Challenges.

Cytomegalovirus (CMV) remains a significant contributor to morbidity and death after pediatric solid and stem cell transplantation. Decisions regarding prevention and treatment often lack pediatric-specific data to drive decision making. We present here a case-based discussion around some of these specific topics and focus on approaches to CMV prevention, post-CMV secondary prophylaxis options, and identification and treatment of resistant CMV infection, including emerging antiviral agents and the use of cytotoxic CMV-specific T-cells, in the setting of pediatric hematopoietic stem cell transplantation.

Development of a newborn screening tool based on bivariate normal limits: using psychosine and galactocerebrosidase determination on dried blood spots to predict Krabbe disease.

Purpose: Newborn screening for Krabbe disease (KD) originated in New York State in 2006 but has proven to have a high false positive rate and low positive predictive value. To improve accuracy of presymptomatic prediction, we propose a screening tool based on two biomarkers, psychosine and galactocerebrosidase enzyme activity (GalC).

Methods: We developed the tool using measures from dried blood spots of 166 normal newborns and tested it on dried blood spot measures from 15 newborns who later developed KD, 8 newborns identified as "high risk" by the New York screening protocol but were disease-free at follow-up, and 3 symptomatic children with onset before 4 years of age.

Fetuses with single ventricle congenital heart disease manifest impairment of regional brain growth.

Objective: Anomalous neurological development associated with congenital heart disease (CHD) has been reported as early as third trimester of fetal development. While several studies have characterized variations in CHD neurodevelopmental outcomes in early childhood, these reports are often confounded by postnatal factors such as surgical outcome. Recent studies have focused on the comparing neurological variations between fetuses with CHD and normal controls.

Survival and Long-Term Functional Outcomes for Children With Cardiac Arrest Treated With Extracorporeal Cardiopulmonary Resuscitation.

Objectives: To identify patient- and disease-related factors related to survival and favorable outcomes for children who underwent extracorporeal cardiopulmonary resuscitation after a refractory cardiac arrest.

Design: Retrospective observational study with prospective assessment of long-term functional outcome.

Patients: Fifty-six consecutive children undergoing extracorporeal cardiopulmonary resuscitation at our institution from 2007 to 2015.

Survival of children after liver transplantation for hepatocellular carcinoma.

Hepatocellular carcinoma (HCC) in childhood differs from adult HCC because it is often associated with inherited liver disease. It is, however, unclear whether liver transplantation (LT) for HCC in childhood with or without associated inherited disease has a comparable outcome to adult HCC. On the basis of data from the European Liver Transplant Registry (ELTR), we aimed to investigate if there are differences in patient and graft survival after LT for HCC between children and adults and between patients with underlying inherited versus noninherited liver disease, respectively.

New surgical technique for creation of a continent appendicostomy: Invaginated appendicostomy.

Introduction: The main complications of appendicostomy are stenosis and stomal fecal leakage. Although many authors report that the appendix is naturally continent, it is recommended to perform a plication of the cecum with the appendix to prevent stomal fecal leakage. We present here the creation of a different continent mechanism.

Identification of intestinal ion transport defects in microvillus inclusion disease.

Loss of function mutations in the actin motor myosin Vb (Myo5b) lead to microvillus inclusion disease (MVID) and death in newborns and children. MVID results in secretory diarrhea, brush border (BB) defects, villus atrophy, and microvillus inclusions (MVIs) in enterocytes. How loss of Myo5b results in increased stool loss of chloride (Cl(-)) and sodium (Na(+)) is unknown.

Recommended Curriculum for Training in Pediatric Transplant Infectious Diseases.

A working group representing the American Society of Transplantation, Pediatric Infectious Diseases Society, and International Pediatric Transplant Association has developed a collaborative effort to identify and develop core knowledge in pediatric transplant infectious diseases. Guidance for patient care environments for training and core competencies is included to help facilitate training directed at improving the experience for pediatric infectious diseases trainees and practitioners in the area of pediatric transplant infectious diseases.

Unexpected megarectum: A potential hidden source of complications in patients with anorectal malformation.

Background: Primary posterior sagittal anorectoplasty is recommended to repair anorectal malformations with rectoperineal or rectovestibular fistula. The aim of this study was to identify the impact of the presence of megarectum on the relative frequency of complications related to posterior sagittal anorectoplasty.

Methods: We performed a cross-sectional retrospective study including patients with anorectal malformation, preoperative rectogram and surgically treated with primary or staged posterior sagittal anorectoplasty.

Medical status of 219 children with biliary atresia surviving long-term with their native livers: results from a North American multicenter consortium.

Objectives: To examine the medical status of children with biliary atresia (BA) with their native livers after hepato- portoenterostomy (HPE) surgery.

Study Design: The Childhood Liver Disease Research and Education Network database was utilized to examine subjects with BA living with their native livers 5 or more years after HPE and to describe the prevalence of subjects with BA with an "ideal" outcome, defined as no clinical evidence of chronic liver disease, normal liver biochemical indices (aspartate aminotransferase, alanine aminotransferase, γ-glutamyl transpeptidase, platelet count, total bilirubin, international normalized ratio, and albumin), and normal health-related quality of life 5 or more years after HPE.

Results: Children with BA (n = 219; 43% male) with median age 9.

Genetic deletion of toll-like receptor 4 on platelets attenuates experimental pulmonary hypertension.

Rationale: Recent studies demonstrate a role for toll-like receptor 4 (TLR4) in the pathogenesis of pulmonary hypertension (PH); however, the cell types involved in mediating the effects of TLR4 remain unknown.

Objectives: The objective of this study was to determine the contribution of TLR4 expressed on nonparenchymal cells to the pathogenesis of PH.

Methods And Results: TLR4 bone marrow chimeric mice revealed an equal contribution of TLR4 on nonparenchymal and parenchymal cells in the pathogenesis of PH as determined by measuring right ventricular (RV) systolic pressure and RV hypertrophy.

Portal hypertension in children and young adults with biliary atresia.

Objective: Biliary atresia (BA) frequently results in portal hypertension (PHT), complications of which lead to significant morbidity and mortality. The Childhood Liver Disease Research and Education Network was used to perform a cross-sectional multicentered analysis of PHT in children with BA.

Methods: Subjects with BA receiving medical management at a Childhood Liver Disease Research and Education Network site were enrolled.

Efficacy of fat-soluble vitamin supplementation in infants with biliary atresia.

Objective: Cholestasis predisposes to fat-soluble vitamin (FSV) deficiencies. A liquid multiple FSV preparation made with tocopheryl polyethylene glycol-1000 succinate (TPGS) is frequently used in infants with biliary atresia (BA) because of ease of administration and presumed efficacy. In this prospective multicenter study, we assessed the prevalence of FSV deficiency in infants with BA who received this FSV/TPGS preparation.