Extracorporeal Membrane Oxygenation in Children with Pulmonary Atresia and Intact Ventricular Septum: Mortality and Associated Outcomes.

Data on outcomes of extracorporeal membrane oxygenation (ECMO) are limited in patients with pulmonary atresia intact ventricular septum (PAIVS). The objective of this study was to describe the use of ECMO and the associated outcomes in patients with PAIVS. We retrospectively reviewed neonates with PAIVS who received ECMO between 2009 and 2019 in 19 US hospitals affiliated with the Collaborative Research for the Pediatric Cardiac Intensive Care Society (CoRe-PCICS).

Perfluorodecanoic acid (PFDA) increases oxidative stress through inhibition of mitochondrial β-oxidation.

PER: and polyfluoroalkyl substances (PFAS) are a large group of synthetic organic chemicals that are ubiquitous environmental pollutants. Among PFAS, perfluorodecanoic acid (PFDA) is one of the most toxic compounds, but the molecular basis behind its toxicity is not fully understood. In an interspecies comparison with placental cells (HTR-8/SVneo) and zebrafish embryos, we demonstrate that PFDA induces mitochondrial dysfunction and impairs fatty acid β-oxidation.

Imaging of common coronary artery anomalies and imaging features important for clinical decision-making.

Anomalous coronary arteries are associated with sudden cardiac death, although only in a relatively small percentage. Although this has been a field of study for years and there are many ongoing studies of larger patient cohorts and longer term follow-up, important questions remain at present in clinical decision-making, particularly whether or not to have surgery. Advanced imaging techniques including coronary computed tomography (CT) and cardiac magnetic resonance (MR) play an important role in the workup of patients with known or suspected anomalies including delineating origin and course as well as defining presence of high-risk imaging features.

Skin Graftless Dorsal Metacarpal Island VY Advancement Flap vs. Dorsal Rectangular Flap with Skin Grafting in 213 Simple Syndactyly Releases.

Background: Simple syndactyly is a common congenital upper extremity difference. Traditional reconstructive approaches utilize skin grafts; however, recent studies suggest skin graftless techniques may improve outcomes. We hypothesized that patients who underwent reconstruction with a skin graftless dorsal metacarpal island VY advancement flap would have fewer postoperative complications compared to those released with a dorsal rectangular flap with skin grafts.

Bladder exstrophy-epispadias-cloacal exstrophy complex: characteristics, aetiologies, and epidemiologic findings.

Bladder exstrophy-epispadias-cloacal exstrophy complex (BEEC) is a spectrum of congenital urologic anomalies that involve the bladder, urethra, genitalia, and pelvic musculoskeletal system, and can affect urinary continence, sexual health, and fertility. BEEC includes a wide spectrum of anatomical abnormalities with different levels of severity: epispadias represents the mildest phenotype, classic bladder exstrophy (CBE) is the most common defect, and cloacal exstrophy (CE) - often referred to as omphalocele, exstrophy, imperforate anus, and spinal defects (OEIS) complex - is the most severe form. BEEC disorders cause significant health problems and affect the health-related quality of life (QoL) of affected individuals.

Artificial intelligence-based morphologic classification and molecular characterization of neuroblastic tumors from digital histopathology.

A deep learning model using attention-based multiple instance learning (aMIL) and self-supervised learning (SSL) was developed to perform pathologic classification of neuroblastic tumors and assess MYCN-amplification status using H&E-stained whole slide images from the largest reported cohort to date. The model showed promising performance in identifying diagnostic category, grade, mitosis-karyorrhexis index (MKI), and MYCN-amplification with validation on an external test dataset, suggesting potential for AI-assisted neuroblastoma classification.

Neighborhood-Level Deprivation Impacts Graft and Patient Outcomes Among Pediatric Liver Transplant Recipients.

Background: The impact of social determinants of health (SDOH) on pediatric liver transplant outcomes has not been extensively investigated. The purpose of this study was to examine the effect of neighborhood-level deprivation on graft and patient survival among pediatric liver transplant recipients.

Methods: This is a single-center observational study among children who received a primary liver transplant from March 1996 to March 2023.

Regenerative Variability of Human Juvenile Chondrocyte Sheets From Different Cell Donors in an Athymic Rat Knee Chondral Defect Model.

Purpose: This study aimed to establish a combined histological assessment system of neo-cartilage outcomes and to evaluate variations in an established rat defect model treated with human juvenile cartilage-derived chondrocyte (JCC) sheets fabricated from various donors.

Methods: JCCs were isolated from the polydactylous digits of eight patients. Passage 2 (P2) JCC sheets from all donors were transplanted into nude rat chondral defects for 4 weeks (27 nude rats in total).

Feasibility of biliary atresia newborn screening in an integrated health network.

Unlabelled: Diagnostic delay remains a barrier to improving biliary atresia (BA) outcomes. We tested the implementation feasibility of a two stage BA newborn screening program in an integrated healthcare system.

Methods: Under a waiver of consent, we measured direct bilirubin (DB) levels in well newborns undergoing standard of care hyperbilirubinemia screening at four hospitals.

Modification and scoring of a transition tool to understand transition readiness among pediatric kidney transplant recipients.

Background: A successful transition from pediatric to adult healthcare for adolescent and young adult kidney transplant recipients is essential for maintaining graft and overall health. Readiness for transition is multifactorial and can be challenging to assess. The purpose of this study is to describe the development of a scoring system for a transition readiness assessment for pediatric kidney transplant recipients and assess overall and domain-specific readiness for transition.

Training the Next Generation of Transplant Surgeons With a 3-Dimensional Trainer: A Pilot Study.

Background: In the United States, no published guidelines promote exposure to technical variants (ie, living donor or split liver) during transplant fellowship. Simulation with hands-on liver models may improve training in transplantation. This pilot study addressed 3 overall goals (material and model creation tools, recruitment rates and assessment of workload, and protocol adherence).

Technical Recommendations for Computed Tomography Guidance of Intervention in the Right Ventricular Outflow Tract: Native RVOT, Conduits and Bioprosthetic Valves: A White Paper of the Society of Cardiovascular Computed Tomography (SCCT), Congenital Heart Surgeons' Society (CHSS), and Society for Cardiovascular Angiography & Interventions (SCAI).

This consensus document for the performance of cardiovascular computed tomography (CCT) to guide intervention in the right ventricular outflow tract (RVOT) in patients with congenital disease (CHD) was developed collaboratively by pediatric and adult interventionalists, surgeons and cardiac imagers with expertise specific to this patient subset. The document summarizes definitions of RVOT dysfunction as assessed by multi-modality imaging techniques and reviews existing consensus statements and guideline documents pertaining to indications for intervention. In the context of this background information, recommendations for CCT scan acquisition and a standardized approach for reporting prior to surgical or transcatheter pulmonary valve replacement are proposed and presented.

Comparing Penile Problems in Circumcised vs. Uncircumcised Boys: Insights From a Large Commercial Claims Database With a Focus on Provider Type Performing Circumcision.

Objectives: To compare penile problems in circumcised relative to uncircumcised boys, and to determine which providers performing the circumcision have fewer post-circumcision problems.

Methods: CPT codes in the 2011-2020 MarketScan database were used to identify boys who had a circumcision. Uncircumcised control subjects of the same age, state of residence, and insurance type were selected.

Effect of rabbit ATG PK on outcomes after TCR-αβ/CD19-depleted pediatric haploidentical HCT for hematologic malignancy.

We hypothesized that the inferior disease-free survival (DFS) seen in older patients who underwent αβ-T-cell/CD19-depleted (AB-TCD) haploidentical hematopoietic cell transplantation (HCT) for hematologic malignancies is caused by excessive exposure to rabbit antithymocyte globulin (rATG; Thymoglobulin). Between 2015 and 2023, 163 patients with a median age of 13 years (range, 0.4-27.

Prioritization of Randomized Clinical Trial Questions for Children Hospitalized With Common Conditions: A Consensus Statement.

Importance: There is a lack of randomized clinical trial (RCT) data to guide many routine decisions in the care of children hospitalized for common conditions. A first step in addressing the shortage of RCTs for this population is to identify the most pressing RCT questions for children hospitalized with common conditions.

Objective: To identify the most important and feasible RCT questions for children hospitalized with common conditions.

Emergency department pediatric readiness of United States trauma centers in 2021: Trauma center facility characteristics and opportunities for improvement.

Background: Emergency department (ED) pediatric readiness has been associated with lower mortality for injured children but has historically been suboptimal in nonpediatric trauma centers. Over the past decade, the National Pediatric Readiness Project (NPRP) has invested resources in improving ED pediatric readiness. This study aimed to quantify current trauma center pediatric readiness and identify associations with center-level characteristics to target further efforts to guide improvement.

How I use next-generation sequencing-MRD to plan approach and prevent relapse after HCT for children and adults with ALL.

Measurable residual disease (MRD) evaluation by multiparameter flow cytometry (MFC) or quantitative polymerase chain reaction methods is an established standard of care for assessing risk of relapse before or after hematopoietic cell transplantation (HCT) for acute lymphoblastic leukemia (ALL). Next-generation sequencing (NGS)-MRD has emerged as a highly effective approach that allows for the detection of lymphoblasts at a level of <1 in 106 nucleated cells, increasing sensitivity of ALL detection by 2 to 3 logs. Early studies have shown superior results compared with MFC and suggest that NGS-MRD may allow for the determination of patients in whom reduced toxicity transplant preparative approaches could be deployed without sacrificing outcomes.

Long-term outcomes of peripheral blood stem cell unrelated donors mobilized with filgrastim.

Allogeneic hematopoietic cell transplantation is a life-saving procedure used to treat a variety of devastating diseases. It requires hematopoietic stem cells collected via filgrastim-mobilized peripheral blood stem cells (PBSCs) or bone marrow (BM) harvest from volunteer unrelated donors (URDs). There is a paucity of safety data regarding donors' long-term adverse events.

Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment.

Viral infections remain a major risk in immunocompromised pediatric patients, and virus-specific T cell (VST) therapy has been successful for treatment of refractory viral infections in prior studies. We performed a phase II multicenter study (NCT03475212) for the treatment of pediatric patients with inborn errors of immunity and/or post allogeneic hematopoietic stem cell transplant with refractory viral infections using partially-HLA matched VSTs targeting cytomegalovirus, Epstein-Barr virus, or adenovirus. Primary endpoints were feasibility, safety, and clinical responses (>1 log reduction in viremia at 28 days).