Single ventricle palliation of truncus arteriosus with mitral atresia, interrupted aortic arch, and aberrant right subclavian artery.

The association of truncus arteriosus communis with interrupted aortic arch and mitral atresia is an exceptionally rare congenital defect. We describe the initial decision-making and management of this lesion, which eventually achieved a Fontan palliation.

Trastuzumab Deruxtecan, Antibody-Drug Conjugate Targeting HER2, Is Effective in Pediatric Malignancies: A Report by the Pediatric Preclinical Testing Consortium.

HER2 is expressed in many pediatric solid tumors and is a target for innovative immune therapies including CAR-T cells and antibody-drug conjugates (ADC). We evaluated the preclinical efficacy of trastuzumab deruxtecan (T-DXd, DS-8201a), a humanized monoclonal HER2-targeting antibody conjugated to a topoisomerase 1 inhibitor, DXd, in patient- and cell line-derived xenograft (PDX/CDX) models. HER2 mRNA expression was determined using RNA-seq and protein expression via IHC across multiple pediatric tumor PDX models.

Mammalian Target of Rapamycin Pathway Assessment in Antiphospholipid Antibody-Positive Patients with Livedo.

Objective: In antiphospholipid antibody (aPL) nephropathy, activation of the mammalian target of rapamycin (mTOR) contributes to endothelial cell proliferation, a key finding of aPL microvascular disease. Here, we examined mTOR activation in the skin of aPL-positive patients with livedo.

Methods: Three patient groups with livedo were studied: (1) persistently aPL-positive with systemic lupus erythematosus (SLE); (2) persistently aPL-positive without SLE; and (3) aPL-negative SLE (control).

Association Between the First-Hour Intravenous Fluid Volume and Mortality in Pediatric Septic Shock.

Study Objective: To determine whether the receipt of more than or equal to 30 mL/kg of intravenous fluid in the first hour after emergency department (ED) arrival is associated with sepsis-attributable mortality among children with hypotensive septic shock.

Methods: This is a retrospective cohort study set in 57 EDs in the Improving Pediatric Sepsis Outcomes quality improvement collaborative. Patients less than 18 years of age with hypotensive septic shock who received their first intravenous fluid bolus within 1 hour of arrival at the ED were propensity-score matched for probability of receiving more than or equal to 30 mL/kg in the first hour.

Characterizing Pain Among Adolescents and Young Adults With Arthrogryposis Multiplex Congenita.

Purpose: Primary study objectives were to ( a ) characterize pain and explore differences between adolescents and adults with arthrogryposis multiplex congenita (AMC) and ( b ) evaluate associations between pain-related outcomes and mobility.

Methods: People who can walk and with AMC completed pain-related questionnaires.

Results: Sixty-three participants (28 adolescents and 35 young adults) were recruited.

Assessment of foot alignment and function for ambulatory children with cerebral palsy: Results of a modified Delphi technique consensus study.

Purpose: The purpose of this study was to establish consensus for the assessment of foot alignment and function in ambulatory children with cerebral palsy, using expert surgeon's opinion through a modified Delphi technique.

Methods: The panel used a five-level Likert-type scale to record agreement or disagreement with 33 statements regarding the assessment of foot alignment and function. Consensus was defined as at least 80% of responses being in the highest or lowest of two of the five Likert-type ratings.

Anterior distal femoral hemiepiphysiodesis in children with cerebral palsy: Establishing surgical indications and techniques using the modified Delphi method and literature review.

Purpose: The purpose of this study was to develop consensus for the surgical indications of anterior distal femur hemiepiphysiodesis in children with cerebral palsy using expert surgeon opinion through a modified Delphi technique.

Methods: The panel used a 5-level Likert-type scale to record agreement or disagreement with 27 statements regarding anterior distal femur hemiepiphysiodesis. Consensus was defined as at least 80% of responses being in the highest or lowest 2 of the Likert-type ratings.

Finding consensus for hamstring surgery in ambulatory children with cerebral palsy using the Delphi method.

Purpose: There is marked variation in indications and techniques for hamstring surgery in children with cerebral palsy. There is particular uncertainty regarding the indications for hamstring transfer compared to traditional hamstring lengthening. The purpose of this study was for an international panel of experts to use the Delphi method to establish consensus indications for hamstring surgery in ambulatory children with cerebral palsy.

Body image and disordered eating behaviors in Hispanic/Latino Youth: Findings from the Hispanic Community Health Study/Study of Latino Youth.

Background: Prevalence of certain disordered eating behaviors is higher among Hispanic youth compared to non-Hispanics. Understanding the role of body image and social attitudes towards weight in disordered eating may inform treatment in Hispanic youth.

Methods: We analyzed data from the Hispanic Community Health Study/Study of Latino Youth (SOL Youth).

Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation.

Autologous hematopoietic stem cell transplantation using genome-edited cells can become a definitive therapy for hematological and non-hematological disorders with neurological involvement. Proof-of-concept studies using human genome-edited hematopoietic stem cells have been hindered by the low efficiency of engraftment of the edited cells in the bone marrow and their modest efficacy in the CNS. To address these challenges, we tested a myeloablative conditioning regimen based on Busulfan in an immunocompromised model of mucopolysaccharidosis type 1.

COVID-19 in pediatric kidney transplantation: a follow-up report of the Improving Renal Outcomes Collaborative.

Background: We report follow-up data from an ongoing prospective cohort study of COVID-19 in pediatric kidney transplantation through the Improving Renal Outcomes Collaborative (IROC).

Methods: Patient-level data from the IROC registry were combined with testing, indication, and outcomes data collected to describe the epidemiology of COVID testing, treatment, and clinical outcomes; determine the incidence of a positive COVID-19 test; describe rates of COVID-19 testing; and assess for clinical predictors of a positive COVID-19 test.

Results: From September 2020 to February 2021, 21 centers that care for 2690 patients submitted data from 648 COVID-19 tests on 465 patients.

Idiopathic midaortic syndrome.

Midaortic syndrome (MAS) is characterized by narrowing of the descending aorta between the distal aortic arch and the aortic bifurcation. We present the case of a 4-year-old male presenting with a murmur and diagnosed with MAS. He was treated with a thoracoabdominal bypass graft.

Personalized Research on Diet in Ulcerative Colitis and Crohn's Disease: A Series of N-of-1 Diet Trials.

Introduction: Evidence about specific carbohydrate diet (SCD) for inflammatory bowel disease (IBD) is limited. We conducted 54 single-subject, double-crossover N-of-1 trials comparing SCD with a modified SCD (MSCD) and comparing each with the participant's baseline, usual diet (UD).

Methods: Across 19 sites, we recruited patients aged 7-18 years with IBD and active inflammation.

Omphalitis and Concurrent Serious Bacterial Infection.

Objective: Describe the clinical presentation, prevalence of concurrent serious bacterial infection (SBI), and outcomes among infants with omphalitis.

Methods: Within the Pediatric Emergency Medicine Collaborative Research Committee, 28 sites reviewed records of infants ≤90 days of age with omphalitis seen in the emergency department from January 1, 2008, to December 31, 2017. Demographic, clinical, laboratory, treatment, and outcome data were summarized.

Leveraging Telehealth: Utilizing Digital Health Technology to Improve Patient Care in Orthopaedics.

The past decade has seen a shift in health care delivery models to be more value-based: patient-centered, accessible, and cost-effective. One of the primary modes of addressing these needs has been through the implementation of telemedicine-digital health care technology that streamlines and enhances traditional health care delivery. In the orthopaedic setting, there are various methods of telemedicine utilization, each uniquely optimized for different clinical scenarios.

Implementation of a Provider's Asthma-specific Note to Optimize Billing in the Pediatric Emergency Department.

Unlabelled: There is broad variability in provider documentation for asthma encounters within the pediatric emergency department. Inadequate provider documentation leads to discrepancies between the ideal current procedural terminology (CPT) code and the assigned CPT code based on the care provided. Multiple studies demonstrate improvement in medical provider documentation after implementing standardized documentation templates and educational programs.

Clinical characterization of familial hypercholesterolemia due to an amish founder mutation in Apolipoprotein B.

Background: Familial hypercholesterolemia (FH) due to a founder variant in Apolipoprotein B (ApoB) is reported in 12% of the Pennsylvania Amish community. By studying a cohort of ApoB heterozygotes and homozygotes, we aimed to characterize the biochemical and cardiac imaging features in children and young adults with a common genetic background and similar lifestyle.

Methods: We employed advanced lipid profile testing, carotid intima media thickness (CIMT), pulse wave velocity (PWV), and peripheral artery tonometry (PAT) to assess atherosclerosis in a cohort of Amish ApoB heterozygotes (n = 13), homozygotes (n = 3), and their unaffected, age-matched siblings (n = 9).

COVID-19 Infection and Guillain-Barre Syndrome: A Case Series.

The coronavirus disease 2019 (COVID-19) pandemic brought about an unprecedented time. Multiple systemic complications have been recognized with the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) as it can do much more than affect the respiratory system. One of the intriguing neurological complications is Guillain-Barre syndrome (GBS).

Effect of Physiologic Point-of-Care Cardiopulmonary Resuscitation Training on Survival With Favorable Neurologic Outcome in Cardiac Arrest in Pediatric ICUs: A Randomized Clinical Trial.

Importance: Approximately 40% of children who experience an in-hospital cardiac arrest survive to hospital discharge. Achieving threshold intra-arrest diastolic blood pressure (BP) targets during cardiopulmonary resuscitation (CPR) and systolic BP targets after the return of circulation may be associated with improved outcomes.

Objective: To evaluate the effectiveness of a bundled intervention comprising physiologically focused CPR training at the point of care and structured clinical event debriefings.

Incidence of Deep Vein Thrombosis in Cerebral Palsy Following an Orthopaedic Surgical Event.

Purpose: The purpose of this study was to identify the incidence of venous thromboembolism (VTE) and characterize the demographics, comorbidities, and risk factors for patients with cerebral palsy (CP) having orthopaedic surgery.

Methods: All patients diagnosed with CP who underwent an orthopaedic surgical procedure at one institution between 2008 and 2017 were identified. Diagnosis codes and associated patient events were recovered from the electronic medical record.

Dynamic Supination in Congenital Clubfoot: A Modified Delphi Panel Approach to Standardizing Definitions and Indications for Treatment.

Background: Dynamic supination is a well-recognized cause of congenital clubfoot deformity relapse. However, there is no consensus on how to diagnose it and there are varied approaches in its management. This study aims to define dynamic supination and indications for treatment by presenting consensus from an international panel of experts using a modified Delphi panel approach.