Safety and Efficacy of Deutetrabenazine at High versus Lower Daily Dosages in the ARC-HD Study to Treat Chorea in Huntington Disease.

Background: Huntington disease (HD) is a progressive neurodegenerative disease that causes psychiatric and neurological symptoms, including involuntary and irregular muscle movements (chorea). Chorea can disrupt activities of daily living, pose safety issues, and may lead to social withdrawal. The vesicular monoamine transporter 2 inhibitors tetrabenazine, deutetrabenazine, and valbenazine are approved treatments that can reduce chorea.

Neutralizing antibody immune correlates in COVAIL trial recipients of an mRNA second COVID-19 vaccine boost.

Neutralizing antibody titer has been a surrogate endpoint for guiding COVID-19 vaccine approval and use, although the pandemic's evolution and the introduction of variant-adapted vaccine boosters raise questions as to this surrogate's contemporary performance. For 985 recipients of an mRNA second bivalent or monovalent booster containing various Spike inserts [Prototype (Ancestral), Beta, Delta, and/or Omicron BA.1 or BA.

How Pediatric Readiness can Impact Pediatric Trauma From Every Day to Mass Events.

Disaster events such as weather events and mass casualty events are increasing in frequency and severity. Caring for children during a surge requires a regional approach given limited pediatric inpatient capacity and expertise. During the 2024 American Academy of Pediatrics National Convention and Exhibition, the Section on Surgery and Council on Children and Disasters (COCD) partnered to present a joint symposium emphasizing importance of pediatric readiness and disaster preparedness and role of pediatric trauma surgeons in disaster preparedness and response in all communities.

T follicular helper cell expansion and hyperimmunoglobulinemia with spontaneous IgE production to dietary antigens in IgA-deficient mice.

Immunoglobulin A (IgA), the most abundantly produced antibody at mucosal surfaces, is thought to play key roles in immune responses to respiratory and enteric pathogens and in the regulation of commensal colonization. Low IgA levels have been associated with recurrent infections and immune dysregulation, including inflammatory bowel disease and autoimmunity. Levels of IgA in maternal breast milk and infant stool are both inversely associated with the emergence of immune responses to food antigens in infants and, in naturally resolving food sensitivity and immunotherapy protocols, the induction of IgA antibodies to dietary antigens has been associated with the acquisition of food tolerance.

Development and Evaluation of a Presenter Coaching Program at an Academic Surgical Conference.

Objective: Presenting at academic conferences is an important means of disseminating research, networking, and building a professional reputation, but the quality of presentations at conferences is often suboptimal. This project describes the design, implementation, and evaluation of a presentation coaching program offered by an academic surgical society to presenters at its annual meeting.

Design: Oral presenters were paired with a coach and encouraged to meet independently, yet coaching was unstructured.

Effectiveness of behavioural and psychological interventions for managing obesity in children and adolescents: A systematic review and meta-analysis framed using minimal important difference estimates based on GRADE guidance to inform a clinical practice guideline.

Objective: Conduct a systematic review and meta-analysis of randomized controlled trials (RCTs) of behavioural and psychological interventions for managing paediatric obesity.

Methods: Eligible studies, published between 1985 and 2022, included 0 to 18 year olds with outcomes reported ≥3 months post-baseline, including patient-reported outcome measures (PROMs), cardiometabolic and anthropometric outcomes, and adverse events (AEs). We pooled data using a random effects model and assessed certainty of evidence (CoE) related to minimally important difference estimates for outcomes using GRADE.

Effects of HMG CoA reductase (HMGCR) deficiency on skeletal muscle development.

Pathogenic variants in HMGCR were recently linked to a limb-girdle muscular dystrophy (LGMD) phenotype. The protein product HMG CoA reductase (HMGCR) catalyzes a key component of the cholesterol synthesis pathway. The two other muscle diseases associated with HMGCR, statin-associated myopathy (SAM) and autoimmune anti-HMGCR myopathy, are not inherited in a Mendelian pattern.

Pain management after pediatric minimally invasive repair of pectus excavatum: a narrative review.

Background And Objective: Pectus excavatum is a common congenital chest wall abnormality characterized by a concave appearance of the chest, and minimally invasive repair of pectus excavatum (MIRPE) is the surgical treatment of choice. A rapidly growing field of research is pain management in children undergoing MIRPE, with many shifts in practice occurring over the last decade. The primary objectives of this narrative review are to describe current methods of perioperative pain management and the development of enhanced recovery after surgery (ERAS) to improve the experience of patients undergoing MIRPE.

Response to induction chemotherapy modifies the effect of conventional prognostic factors in high-risk neuroblastoma: A report from the Children's Oncology Group.

Background: Response to induction chemotherapy has been shown to predict outcome in patients with high-risk neuroblastoma (HR-NB), with those achieving a complete response (CR) having superior outcomes.

Methods: We evaluated whether conventional prognostic factors remain prognostic in subsets of patients defined by response to induction. 1244 Patients from four COG high-risk trials were included.

Hybrid care model: Combining telemedicine and office visits for diabetes management in older adults with type 1 diabetes.

Aims: To evaluate the use of hybrid (telehealth and in-person) care on visitation and glycaemia in older adults with type 1 diabetes (T1D).

Methods: In this retrospective study, we examined clinical characteristics, number of visits (telehealth and in-person) and continuous glucose monitoring (CGM) metrics for older adults (≥65 years) with T1D from electronic health records during the pre-COVID-19 pandemic (March 1, 2019-March 1, 2020; in-person) and pandemic (September 1, 2020-August 31, 2021; hybrid) periods. Main outcomes were the number of visits and changes in glycaemic control (HbA1c), and in a sub-group of older adults using CGM, changes in CGM metrics between in-person and hybrid care.

Emergency department utilization by youth before and after firearm injury.

Background: Emergency department (ED) visits may serve as opportunities for firearm injury prevention and intervention efforts. Our objective was to determine ED utilization by youth before and after firearm injury.

Methods: We performed a retrospective cohort study of ED encounters by youth (0-18 years old) with firearm injury from eight states using the 2019 State ED and Inpatient Databases.

Pediatric erythromelalgia from multidisciplinary perspectives: a scoping review.

Erythromelalgia is a rare, chronic pain disorder characterized by the triad of intense burning sensation, warmth, and redness, primarily involving the hands and feet, and usually alleviated by cold and worsened by heat. The objective of this scoping review was to: 1) map the existing literature on erythromelalgia in youth, 2) identify knowledge gaps, and 3) inform directions for future research in pediatric erythromelalgia. One hundred and sixty-seven studies reporting 411 cases of childhood-onset erythromelalgia were identified.

Recommendations for mitochondria transfer and transplantation nomenclature and characterization.

Intercellular mitochondria transfer is an evolutionarily conserved process in which one cell delivers some of their mitochondria to another cell in the absence of cell division. This process has diverse functions depending on the cell types involved and physiological or disease context. Although mitochondria transfer was first shown to provide metabolic support to acceptor cells, recent studies have revealed diverse functions of mitochondria transfer, including, but not limited to, the maintenance of mitochondria quality of the donor cell and the regulation of tissue homeostasis and remodelling.

Trial of Labor After Cesarean Delivery in Individuals With Twin Pregnancies and Two Prior Cesarean Deliveries.

Objective: To investigate short-term maternal and neonatal outcomes in individuals with twin pregnancies and two prior cesarean deliveries who underwent trial of labor after cesarean (TOLAC).

Methods: A cross-sectional study of live-birth data was conducted between 2014 and 2021 in the United States. Individuals with more than two prior cesarean deliveries and multiple gestations higher than twins were excluded.

Imaging Demyelinated Axons After Spinal Cord Injuries with PET Tracer [F]3F4AP.

Spinal cord injuries (SCIs) often lead to lifelong disability. Among the various types of injuries, incomplete and discomplete injuries, where some axons remain intact, offer potential for recovery. However, demyelination of these spared axons can worsen disability.

US and UK college students' experiences of living with chronic pain: A qualitative analysis.

: To understand the lived experiences and needs of college students with chronic pain living in the US and the UK. College students ( = 115), mean age 21.4 years (SD = 1.

Age-Related Ventricular Tachycardia Substrate Characteristics for Repaired Tetralogy of Fallot Before Transcatheter Pulmonary Valve Placement.

Background: Ventricular tachycardia (VT) substrate characteristics before transcatheter pulmonary valve replacement (TPVR) in repaired tetralogy of Fallot (rTOF) are unknown.

Objectives: In this study, the authors sought to evaluate substrates for sustained monomorphic VT before TPVR in rTOF.

Methods: Retrospective (2017 to 2021) and prospective (commencing 2021) rTOF patients with native right ventricular outflow tract referred for electrophysiology study (EPS) before TPVR were included.

A Retrospective Cohort Study of Transgender Adolescents' Gender-Affirming Hormone Discontinuation.

Purpose: To understand the rate of, and reasons for, discontinuation of gender-affirming hormones (GAH) in transgender adolescents.

Methods: Retrospective cohort study of individuals starting GAH between January 2007 and December 2022. Individuals were included if they were diagnosed with gender dysphoria, were prescribed GAH, and took GAH continuously for a minimum of 6 months.

Emotional Health of Transgender Youth 24 Months After Initiating Gender-Affirming Hormone Therapy.

Purpose: Limited data exist about the emotional health of transgender youth, either before or after initiation of gender-affirming hormone (GAH). The objectives were: (1) Investigate and verify the factor structure of the National Institutes of Health Toolbox Emotional Battery (NIHTB-EB) among trans and non-binary (TNB) youth; (2) Examine changes in emotional health over 24 months of GAH treatment; and (3) Examine the extent to which changes in emotional health were associated with improved appearance congruence (AC).

Methods: Study respondents were from Trans Youth Care - United States (TYCUS) study, an observational, prospective, longitudinal study of adolescents initiating GAH enrolled between 2016 and 2019.

The role of the hippocampus in working memory and word reading: Novel neural correlates of reading among youth living in the context of economic disadvantage.

A left-lateralized cortical reading circuit underlies successful reading and fails to engage in individuals with reading problems. Studies identifying this circuit included youth from economically advantaged backgrounds and focused on cortical, not subcortical, structures. However, among youth with low scores on reading tests who are living in the context of economic disadvantage, this brain network is actively engaged during reading, despite persistent reading problems.