Understanding the Burden of Pediatric Traumatic Injury in Uganda: A Multicenter, Prospective Study.

Introduction: Traumatic injury is responsible for eight million childhood deaths annually. In Uganda, there is a paucity of comprehensive data describing the burden of pediatric trauma, which is essential for resource allocation and surgical workforce planning. This study aimed to ascertain the burden of non-adolescent pediatric trauma across four Ugandan hospitals.

Racial and ethnic disparities in acuity of presentation among children with newly diagnosed acute leukemia.

We evaluated disparities in disease burden, organ dysfunction, vital signs, and timing of therapy in children newly presenting with acute leukemia. Among 899 patients with acute leukemia diagnosed at two large children's hospitals, a priori lab-based definitions of high disease burden, infection risk, renal dysfunction, and coagulopathy were applied to electronic health record data. Black patients with acute myeloid leukemia had increased prevalence of elevated white blood cell count and uric acid; Black patients with acute lymphoblastic leukemia demonstrated increased prevalence of coagulopathy.

Reducing Volatile Anesthetic Waste Using a Commercial Electronic Health Record Clinical Decision Support Tool to Lower Fresh Gas Flows.

Background: Volatile anesthetic consumption can be reduced by minimizing excessive fresh gas flows (FGFs). Currently, it is unknown whether decision support tools embedded within commercial electronic health record systems can be successfully adopted to achieve long-term reductions in FGF rates. The authors describe the implementation of an electronic health record-based clinical decision support tool aimed at reducing FGF and evaluate the effectiveness of this intervention in achieving sustained reductions in FGF rates and volatile anesthetic consumption.

In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.

Patients with early-onset lysosomal storage diseases are ideal candidates for prenatal therapy because organ damage starts in utero. We report the safety and efficacy results of in utero enzyme-replacement therapy (ERT) in a fetus with CRIM (cross-reactive immunologic material)-negative infantile-onset Pompe's disease. The family history was positive for infantile-onset Pompe's disease with cardiomyopathy in two previously affected deceased siblings.

Sustained Within-season Vaccine Effectiveness Against Influenza-associated Hospitalization in Children: Evidence From the New Vaccine Surveillance Network, 2015-2016 Through 2019-2020.

Background: Adult studies have demonstrated within-season declines in influenza vaccine effectiveness (VE); data in children are limited.

Methods: We conducted a prospective, test-negative study of children 6 months through 17 years hospitalized with acute respiratory illness at 7 pediatric medical centers during the 2015-2016 through 2019-2020 influenza seasons. Case-patients were children with an influenza-positive molecular test matched by illness onset to influenza-negative control-patients.

What Factors Correlate With Length of Stay and Readmission After Limb Lengthening Procedures? A Large-database Study.

Background: Indications and techniques for limb lengthening procedures have evolved over the past two decades. Although there are several case series reporting on the complications and efficacy of these techniques, limited data are available on length of stay and hospital readmission rates after these procedures.

Questions/purposes: (1) What is the median length of stay after lower limb lengthening procedures, and is variability in patient demographics, preoperative diagnosis, and surgical technique associated with length of stay? (2) What is the 1-year readmission rate after lower limb lengthening procedures? (3) Is variability in patient demographics, preoperative diagnosis, and surgical technique associated with varying rates of hospital readmission?

Methods: Patients who underwent femoral or tibial lengthening from 2005 to 2015 in seven states were identified using the Healthcare Cost and Utilization Project (HCUP) State Inpatient Databases.

Stress and corticotropin releasing factor (CRF) promote necrotizing enterocolitis in a formula-fed neonatal rat model.

The etiology of necrotizing enterocolitis (NEC) is not known. Alterations in gut microbiome, mucosal barrier function, immune cell activation, and blood flow are characterized events in its development, with stress as a contributing factor. The hormone corticotropin-releasing factor (CRF) is a key mediator of stress responses and influences these aforementioned processes.

Bile acid biosynthesis in Smith-Lemli-Opitz syndrome bypassing cholesterol: Potential importance of pathway intermediates.

Bile acids are the end products of cholesterol metabolism secreted into bile. They are essential for the absorption of lipids and lipid soluble compounds from the intestine. Here we have identified a series of unusual Δ-unsaturated bile acids in plasma and urine of patients with Smith-Lemli-Opitz syndrome (SLOS), a defect in cholesterol biosynthesis resulting in elevated levels of 7-dehydrocholesterol (7-DHC), an immediate precursor of cholesterol.

Impact of Dependent Coverage Provision of the Affordable Care Act on Insurance Continuity for Adolescents and Young Adults With Cancer.

Purpose: The 2010 Dependent Coverage Provision (DCP) of the Affordable Care Act (ACA) allowed enrollees to remain on their parents' health insurance until 26 years of age. We compared rates of insurance disenrollment among patients with cancer who were DCP-eligible at age 19 to those who were not eligible at age 19.

Methods: Using OptumLabs Data Warehouse, which contains longitudinal, real-world, de-identified administrative claims for commercial enrollees, we examined patients born between 1982 and 1993 and diagnosed with cancer between 2000 and 2015.

The role of computed tomography and magnetic resonance imaging for preoperative pediatric cochlear implantation work-up in academic institutions.

Objective: The goal of the study is to investigate the association of pertinent preoperative temporal bone computed tomography (CT) and brain magnetic resonance imaging (MRI) results and intraoperative surgical findings and complications of pediatric cochlear implantation reported in academic settings.

Methods: This is a retrospective review of cochlear implant patients who received a pre-operative temporal bone CT and MRI of the brain between 2005 and 2012 at academic pediatric otolaryngology practices within children's hospitals in the United States and France. Scans were reviewed in a double-blind fashion and compared to intraoperative findings.

A Meningococcal Outer Membrane Vesicle Vaccine with Overexpressed Mutant FHbp Elicits Higher Protective Antibody Responses in Infant Rhesus Macaques than a Licensed Serogroup B Vaccine.

MenB-4C (Bexsero; GlaxoSmithKline Biologicals) is a licensed meningococcal vaccine for capsular B strains. The vaccine contains detergent-extracted outer membrane vesicles (dOMV) and three recombinant proteins, of which one is factor H binding protein (FHbp). In previous studies, overexpression of FHbp in native OMV (NOMV) with genetically attenuated endotoxin (LpxL1) and/or by the use of mutant FHbp antigens with low factor H (FH) binding increased serum bactericidal antibody (SBA) responses.

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.

Background: Deoxygenated sickle hemoglobin (HbS) polymerization drives the pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has potential to favorably modify disease outcomes. Voxelotor is an HbS polymerization inhibitor.

Disparities in Survival and Health Outcomes in Childhood Leukemia.

Purpose Of Review: The aim of this review is to summarize the current literature on pediatric leukemia disparities with attention to not only racial and ethnic disparities, but also socioeconomic disparities. We focus on disparities in survival as well as other health-related outcomes, including end-of-life care and late effects.

Recent Findings: While progress has been made in decreasing some disparities, most notably in pediatric acute lymphoblastic leukemia, disparities along many axes persist.

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.

Background: Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK) cells when matched sibling donors are unavailable unless high-dose chemotherapy is given. In previous studies, autologous gene therapy with γ-retroviral vectors failed to reconstitute B-cell and NK-cell immunity and was complicated by vector-related leukemia.

Methods: We performed a dual-center, phase 1-2 safety and efficacy study of a lentiviral vector to transfer complementary DNA to bone marrow stem cells after low-exposure, targeted busulfan conditioning in eight infants with newly diagnosed SCID-X1.

Molecular profiling and targeted therapy in pediatric gliomas: review and consensus recommendations.

As the field of neuro-oncology makes headway in uncovering the key oncogenic drivers in pediatric glioma, the role of precision diagnostics and therapies continues to rapidly evolve with important implications for the standard of care for clinical management of these patients. Four studies at major academic centers were published in the last year outlining the clinically integrated molecular profiling and targeting of pediatric brain tumors; all 4 demonstrated the feasibility and utility of incorporating sequencing into the care of children with brain tumors, in particular for children and young adults with glioma. Based on synthesis of the data from these studies and others, we provide consensus recommendations for the integration of precision diagnostics and therapeutics into the practice of pediatric neuro-oncology.

Disparities in pediatric acute myeloid leukemia (AML) clinical trial enrollment.

Equal access to clinical trial enrollment is important to ensure that findings are generalizable to the broader population. This study aimed to evaluate disparities in enrollment on pediatric oncology clinical trials. We assessed the relationship between patient characteristics and enrollment on COG trial AAML1031 in a cohort of pediatric patients with AML in the Pediatric Health Information System.

Use of test weights for breastfeeding infants with congenital heart disease in a cardiac transitional care unit: a best practice implementation project.

Objectives: The goal of this project was to promote breastfeeding among infants with congenital heart disease in a quaternary care academic children's hospital. We aimed to increase the total number of breastfeeding episodes among all patients in the Pediatric Cardiac Transitional Care Unit with implementation of pre- and post-breastfeeding (test) weights.

Introduction: Infants with congenital heart disease are able to breastfeed, but are often not encouraged to do so by healthcare providers.

A Meningococcal Native Outer Membrane Vesicle Vaccine With Attenuated Endotoxin and Overexpressed Factor H Binding Protein Elicits Gonococcal Bactericidal Antibodies.

Background: Meningococcal outer membrane vesicle (OMV) vaccines are prepared with detergents to remove endotoxin, which also remove desirable antigens such as factor H binding protein (FHbp). Native OMV (NOMV) vaccines with genetically attenuated endotoxin do not require detergent treatment and elicit broader serum bactericidal antibody (SBA) responses than OMV or recombinant FHbp (rFHbp) vaccines.

Methods: We measured human complement-mediated SBA responses in mice immunized with NOMV with overexpressed FHbp subfamily B (NOMV-FHbp), NOMV with FHbp genetically inactivated (NOMV-KO), and/or a control rFHbp vaccine against meningococcal and gonococcal strains.

Evaluation of a Magnetic Tracking and Electrocardiogram-based Tip Confirmation System for Peripherally Inserted Central Catheters in Pediatric Patients.

The purpose of this study was to investigate whether a magnetic tracking and electrocardiogram-based tip confirmation system (TCS) (Sherlock 3CG Tip Confirmation System; Bard, Covington, GA) permits safe and correct placement of a peripherally inserted central catheter (PICC) in the pediatric population. A total of 144 PICCs were placed using the TCS. After excluding participants for various reasons, 112/121 (92.

A novel nutritional intervention improves lung function in overweight/obese adolescents with poorly controlled asthma: the Supplemental Nutrition in Asthma Control (SNAC) pilot study.

Asthma in the obese is often severe, difficult to treat, and characterized by less eosinophilic inflammation than asthma in the nonobese. Obesity-associated metabolic dysregulation may be a causal factor. We previously reported that a nutrient- and fiber-dense bar [Children's Hospital Oakland Research Institute (CHORI)-bar], which was designed to fill gaps in poor diets, improved metabolism in healthy overweight/obese (OW/OB) adults.

Transitioning Patients With Iron Overload From Exjade to Jadenu.

Iron overload is a concern for patients who require chronic transfusions as a result of inherited or acquired anemias, including sickle cell disease, thalassemia, and myelodysplastic syndromes. Iron chelation therapy (ICT) is the primary treatment for iron overload in these patients. The ICT deferasirox, which has been available as an oral dispersible tablet for liquid suspension, is now also available as a once-daily, film-coated tablet (FCT).

Peripheral Blood Biomarkers Associated With Toxicity and Treatment Characteristics After I- Metaiodobenzylguanidine Therapy in Patients With Neuroblastoma.

Purpose: Few tools exist to predict clinical outcomes after radiopharmaceutical therapy. Our goal was to identify associations between blood-based biomarkers of radiation effect and clinical outcomes after I-metaiodobenzylguanidine (I-MIBG) therapy in patients with neuroblastoma.

Methods And Materials: We conducted a prospective, single-center cohort study in children with advanced neuroblastoma treated with I-MIBG as monotherapy or in combination with systemic putative radiation sensitizers.