Anterior deflected urinary stream in female children: description of a unique clinical entity and surgical management.

Objectives: To investigate the clinical presentation and outcomes for a series of female pediatric patients with severe anterior deflection of the urinary stream (ADUS) who were managed via urethromeatoplasty.

Methods: This single institution retrospective cohort study used the institutional billing database to identify female patients ≤18 years who received a urethromeatoplasty (CPT 53450) from 2007 to 2022. Patients were included if a substantial anterior deflection of their urinary stream was the primary indication for surgery.

Removal of the Metaphyseal Screw From Tension Band Constructs After Hemiepiphysiodesis: High Rates of Physeal Tethering and Subsequent Implant Removal.

Background: Removal of the metaphyseal screw from tension band plate constructs after correction of angular deformity in patients treated with hemiepiphysiodesis has been suggested as an alternative to removing the plate and both screws. While this has the potential benefit of easier implant removal and reinsertion in the event of rebound, there is debate in the literature regarding the benefits and risks of leaving the epiphyseal screw and plate in place.

Methods: Patients treated with hemiepiphysiodesis at the distal femur and/or proximal tibia with tension band plates and screws who underwent subsequent removal of the metaphyseal screw after correction were included.

Prevalence and Impact of Recurrent Rejection on Pediatric Heart Transplant Recipients: A PHTS Multi-Institutional Analysis.

Background: Studies evaluating the prevalence and impact of recurrent rejection (RR) in pediatric heart transplant (HT) are sparse.

Objectives: The purpose of this study was to describe prevalence and impact of RR on cardiac allograft vasculopathy (CAV) and graft loss after pediatric HT.

Methods: Data on HT from January 1, 2000, to June 30, 2020, in the Pediatric Heart Transplant Society database were included.

Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B.

Background: Little information regarding the management of invasive procedures in people with hemophilia B (HB) after undergoing gene therapy is available. Here, we report the management of invasive procedures in people with severe or moderately severe HB who had previously been treated with etranacogene dezaparvovec in the phase 2b and phase 3 Health Outcomes with Padua Gene; Evaluation in Hemophilia B clinical trials (NCT03489291 and NCT03569891).

Objectives: The objective of this study was to describe the use of exogenous FIX, endogenous FIX activity prior to invasive procedures, and peri- and postoperative bleeds in participants who underwent invasive procedures after receiving etranacogene dezaparvovec gene therapy.

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).

Protocol-Stipulated Dose Modification to Manage Chemotherapy-Induced Peripheral Neuropathy in Children, Adolescents, and Young Adults With High-Risk Hodgkin Lymphoma.

Purpose: Brentuximab vedotin (BV) incorporation into frontline chemotherapy regimens improved outcomes for classic Hodgkin lymphoma (cHL). The shared mechanism of action of BV and vinca alkaloids as microtubulin inhibitors increased the potential risk of chemotherapy-induced peripheral neuropathy (CIPN). Rates of CIPN and use of protocol-stipulated dose modifications of a microtubulin inhibitor were examined on the Children's Oncology Group AHOD1331 study, which compared BV, doxorubicin, vincristine (VCR), etoposide, prednisone, cyclophosphamide (BV-AVE-PC; BV arm) with bleomycin containing doxorubicin, bleomycin, vincristine, etoposide, prednisone, cyclophosphamide (ABVE-PC; standard arm) in patients with high-risk cHL ages 2-21 years.

Development and validation of an open-source pipeline for automatic population of case report forms from electronic health records: a pediatric multi-center prospective study.

Background: Clinical trials and registry studies are essential for advancing research and developing novel treatments. However, these studies rely on manual entry of thousands of variables for each patient. Repurposing real-world data can significantly simplify the data collection, reduce transcription errors, and make the data entry process more efficient, consistent, and cost-effective.

Pediatric Epilepsy Genetic Testing Results and Long-term Seizure Freedom.

To determine whether there is a correlation of genetic diagnosis/result with long-term seizure freedom in pediatric epilepsy patients. This was a prospective and retrospective cohort study of children with epilepsy referred for genetic testing at a single center. The primary outcomes were presence and type of genetic diagnosis (pathogenic, benign, or variant of uncertain significance) and patient epilepsy status (seizure free, treatment failure, uncertain).

Sustained Performance of Cardiac Arrest Prevention in Pediatric Cardiac Intensive Care Units.

Importance: The Pediatric Cardiac Critical Care Consortium (PC4) cardiac arrest prevention (CAP) quality improvement (QI) project facilitated a decreased in-hospital cardiac arrest (IHCA) incidence rate across multiple hospitals. The sustainability of this outcome has not been determined.

Objective: To examine the IHCA incidence rate at participating hospitals after the QI project ended and discern which factors best aligned with sustained improvement.

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.

Interventions to Improve Health Among Refugees in the United States: A Systematic Review.

Refugees arriving to the U.S. experience a high burden of both communicable and non-communicable diseases.

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.

Value of Imaging Measurements in Micrognathia-Related Fetal Airway Obstruction Within a Fetal Center.

Objective: Fetal imaging often identifies signs of upper airway obstruction due to micrognathia that may require airway intervention at delivery. This study investigated the role of quantitative fetal imaging measurements in predicting the need for otolaryngology consultation and intervention within a multidisciplinary Fetal Center.

Methods: Data were retrospectively collected from expectant mothers attending a multidisciplinary Fetal Center from January 2017 to October 2023.