Results of a National Delphi consensus on the outpatient management of pediatric psychogenic nonepileptic seizures in the United States.

Background And Objectives: The purpose of this study was to develop national consensus based on expert opinion on the optimal outpatient care model of pediatric psychogenic nonepileptic seizures (PNES).

Methods: A core working group (CWG) within the PNES special interest group of the Pediatric Epilepsy Research Consortium was established. The CWG developed a rigorous scoring rubric to select experts in pediatric PNES within the United States of America and a three-round Delphi study was conducted to assess consensus on key components of the management of pediatric PNES in the outpatient setting.

DROSHA Regulates Mesenchymal Gene Expression in Wilms Tumor.

Wilms tumor, the most common pediatric kidney cancer, resembles embryonic renal progenitors. Currently, there are no ways to therapeutically target Wilms tumor driver mutations, such as in the microRNA processing gene DROSHA. In this study, we used a "multiomics" approach to define the effects of DROSHA mutation in Wilms tumor.

Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.

Background 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.

Exploring the Barriers to Social Support Interactions: A Qualitative Study of Young Adult Cancer Patients and Young Adult Supporters.

This study explores the reasons why young adults (ages 18-39) struggle to provide or avoid providing support to young adult cancer patients. The study also explores young adult cancer patients' perceptions of why they have not received support from individuals who did not provide support. A total of 722 reasons were collected through online surveys and analyzed: 438 were provided by young adults who knew a young adult with cancer ( = 131), and young adult cancer patients ( = 111) provided 284 reasons why they believe they did not receive support.

Comprehensive characterization of patient-derived xenograft models of pediatric leukemia.

Patient-derived xenografts (PDX) remain valuable models for understanding the biology and for developing novel therapeutics. To expand current PDX models of childhood leukemia, we have developed new PDX models from Hispanic patients, a subgroup with a poorer overall outcome. Of 117 primary leukemia samples obtained, successful engraftment and serial passage in mice were achieved in 82 samples (70%).

Evaluation of the Use of Appetite Stimulants in Pediatric Patients with Cystic Fibrosis.

Objective: Poor nutrition in patients with cystic fibrosis (CF) has been associated with lower lung function and increased morbidity and mortality. Conversely, better nutritional status has been associated with improved pulmonary function and fewer CF-associated complications. There is no consensus regarding appetite stimulant therapy in patients with CF (pwCF).

Psychosocial Outcomes in Children with Cleft Lip and/or Palate: Associations of Demographic, Cleft Morphologic, and Treatment-Related Variables.

Objective: To determine associations of demographic, morphologic, and treatment protocol parameters with quality of life (QoL), appearance/speech satisfaction, and psychological adjustment.

Design: Observational study utilizing retrospective report of protocol variables and current outcome variables.

Setting: Six North American cleft treatment clinics.

Ectopic PLAG1 induces muscular dystrophy in the mouse.

Even though various genetic mutations have been identified in muscular dystrophies (MD), there is still a need to understand the biology of MD in the absence of known mutations. Here we reported a new mouse model of MD driven by ectopic expression of PLAG1. This gene encodes a developmentally regulated transcription factor known to be expressed in developing skeletal muscle, and implicated as an oncogene in certain cancers including rhabdomyosarcoma (RMS), an aggressive soft tissue sarcoma composed of myoblast-like cells.

A Multicenter Survey of Pediatric-Neonatal Transport Teams in the United States to Assess the Impact of the Coronavirus Disease 2019 Pandemic on Staffing.

Objective: The coronavirus disease 2019 pandemic has resulted in unprecedented burnout in frontline health care providers. However, the impact of the pandemic on interfacility pediatric and neonatal transport team members has not been studied. The current study uses a survey design to document the impact of the pandemic on pediatric and neonatal transport team members with a focus on staffing and resilience promotion strategies.

Pediatric ERCP in the Setting of Acute Pancreatitis: A Secondary Analysis of an International Multicenter Cohort Study.

Previous studies have demonstrated the safety of performing endoscopic retrograde cholangiopancreatography (ERCP) in the pediatric population; however, few have addressed the outcomes of children undergoing ERCP during acute pancreatitis (AP). We hypothesize that ERCP performed in the setting of AP can be executed with similar technical success and adverse event profiles to those in pediatric patients without pancreatitis. Using the Pediatric ERCP Database Initiative, a multi-national and multi-institutional prospectively collected dataset, we analyzed 1124 ERCPs.

The cost of hospitalizations for treatment of hemodialysis catheter-associated blood stream infections in children: a retrospective cohort study.

Background: Hospitalization costs for treatment of hemodialysis (HD) catheter-associated blood stream infections (CA-BSI) in adults are high. No studies have evaluated hospitalization costs for HD CA-BSI in children or identified factors associated with high-cost hospitalizations.

Methods: We analyzed 160 HD CA-BSIs from the Standardizing Care to Improve Outcomes in Pediatric End-stage Kidney Disease (SCOPE) collaborative database linked to hospitalization encounters in the Pediatric Health Information System (PHIS) database.

Technical Outcomes in Pediatric Endoscopic Retrograde Cholangiopancreatography: Data from an International Collaborative.

Objectives: Endoscopic retrograde cholangiopancreatography (ERCP) in adults has been extensively studied through multicenter prospective studies. Similar pediatric studies are lacking. The Pediatric ERCP Database Initiative (PEDI) is a multicenter collaborative aiming to evaluate the indications and technical outcomes in pediatric ERCPs.

Screening for Academic Risk Among Students With Cleft Lip and/or Palate: Patterns of Risk and Qualities of Effective Tools.

This study evaluated the effectiveness of academic screening measures in relation to parent-reported diagnoses. Multicenter, retrospective cohort study including structured interviews, questionnaires, and chart reviews. Six North American cleft centers.

A Multisite Study Investigating Child and Parent Proxy Reported Quality of Life in Children With Cleft Lip and/or Palate.

This observational, multisite cohort study explored health-related quality of life (HRQoL) in children with cleft lip and/or palate (CL/P), including interrater agreement and ratings for this group relative to clinical cutoff scores and published means for healthy and chronically ill children. Participants (338 children ages 8-10 years, 45.9% male and their parents, 82.

Predictors of Prolonged Fluoroscopy Exposure in Pediatric Endoscopic Retrograde Cholangiopancreatography: Results From the Large Pediatric Endoscopic Retrograde Cholangiopancreatography Database Initiative Multicenter Cohort.

Background And Aims: Ionizing radiation exposure during endoscopic retrograde cholangiopancreatography (ERCP) is an important quality issue especially in children. We aim to identify factors associated with extended fluoroscopy time (FT) in children undergoing ERCP.

Methods: ERCP on children <18 years from 15 centers were entered prospectively into a REDCap database from May 2014 until May 2018.

A Prospective Study of Family Engagement for Prevention of Central Line-associated Blood Stream Infections.

Introduction: We sought to determine if a family-centered care (FCC) handout intervention designed to encourage family engagement (FE) in the prevention of central line-associated bloodstream infections (CLABSIs) would alter parental perceptions of FCC and improve staff compliance with CLABSI bundle components.

Methods: A prospective quasiexperimental study of 121 legal guardians of children with a central venous catheter (CVC) admitted to the pediatric intensive care unit (PICU). Baseline (n = 59) and intervention (n = 62) groups of parents completed an 18-question online survey assessing basic CLABSI care practices and FCC principles.

Interdisciplinary Approach for the Treatment of Complex Bilateral Cleft Lip and Palate With Missing Premaxilla.

Complex craniofacial deformities such as complete bilateral cleft lip and palate require interdisciplinary approach for proper diagnosis and treatment. A severe skeletal discrepancy caused by bilateral cleft lip and palate and missing premaxilla was successfully managed with orthodontic preparation and distraction osteogenesis. Conventional prosthodontic treatment combined with orthodontic preparation was proven to be a viable option to manage multiple missing teeth in cleft lip and palate.

Examining the association of medical complications and posttraumatic stress symptoms in pediatric solid organ transplant patients and their caregivers.

Background: Post-traumatic stress symptoms (PTSS) have been reported by pediatric solid organ transplant (SOT) patients and their caregivers well after transplantation. This study examined the relationship between PTSS, medication adherence, and medical complications in SOT patients and their caregivers. A secondary aim examined the association between patient and caregiver-reported PTSS.