Surveillance of adverse events following immunisation in Australia annual report, 2021.

This report summarises Australia's spontaneous surveillance data for adverse events following immunisation (AEFI) for 2021 reported to the Therapeutic Goods Administration (TGA) and describes reporting trends over the 22-year period 1 January 2000 to 31 December 2021. This report excludes AEFI reports featuring pandemic coronavirus disease 2019 (COVID-19) vaccines, which are reported separately. There were 3,452 AEFI reports for non-COVID-19 vaccines administered in 2021, an annual AEFI reporting rate of 13.

Patterns of recurrence after radiotherapy for high-risk neuroblastoma: Implications for radiation dose and field.

Background: Prognosis for patients with high-risk neuroblastoma (HR-NBL) is guarded despite aggressive therapy, and few studies have characterized outcomes after radiotherapy in relation to radiation treatment fields.

Methods: Multi-institutional retrospective cohort of 293 patients with HR-NBL who received autologous stem cell transplant (ASCT) and EBRT between 1997-2021. LRR was defined as recurrence at the primary site or within one nodal echelon beyond disease present at diagnosis.

Child Opportunity Index and Diagnosis of Developmental Dysplasia of the Hip: Insights From a Children's Hospital Serving Disadvantaged Communities.

Introduction: Initiation of Pavlik harness treatment for developmental dysplasia of the hip (DDH) by 6 to 7 weeks of age predicts a higher rate of success. Child Opportunity Index (COI) 2.0 is a single metric designed to measure resources and conditions affecting children's healthy development.

Developmental isoform diversity in the human neocortex informs neuropsychiatric risk mechanisms.

RNA splicing is highly prevalent in the brain and has strong links to neuropsychiatric disorders; yet, the role of cell type-specific splicing and transcript-isoform diversity during human brain development has not been systematically investigated. In this work, we leveraged single-molecule long-read sequencing to deeply profile the full-length transcriptome of the germinal zone and cortical plate regions of the developing human neocortex at tissue and single-cell resolution. We identified 214,516 distinct isoforms, of which 72.

Brain cell-type shifts in Alzheimer's disease, autism, and schizophrenia interrogated using methylomics and genetics.

Few neuropsychiatric disorders have replicable biomarkers, prompting high-resolution and large-scale molecular studies. However, we still lack consensus on a more foundational question: whether quantitative shifts in cell types-the functional unit of life-contribute to neuropsychiatric disorders. Leveraging advances in human brain single-cell methylomics, we deconvolve seven major cell types using bulk DNA methylation profiling across 1270 postmortem brains, including from individuals diagnosed with Alzheimer's disease, schizophrenia, and autism.

Foxp3 depends on Ikaros for control of regulatory T cell gene expression and function.

Ikaros is a transcriptional factor required for conventional T cell development, differentiation, and anergy. While the related factors Helios and Eos have defined roles in regulatory T cells (Treg), a role for Ikaros has not been established. To determine the function of Ikaros in the Treg lineage, we generated mice with Treg-specific deletion of the Ikaros gene ().

Confidentiality in the Care of Adolescents: Technical Report.

Confidentiality is a foundational element of high-quality, accessible, and equitable health care. Despite strong grounding in federal and state laws, professional guidelines, and ethical standards, health care professionals and adolescent patients face a range of complexities and barriers to seeking and providing confidential care to adolescents across different settings and circumstances. The dynamic needs of adolescents, the oftentimes competing interests of key stakeholders, the rapidly evolving technological context of care, and variable health care billing and claims requirements are all important considerations in understanding how to optimize care to focus on and meet the needs of the adolescent patient.

Confidentiality in the Care of Adolescents: Policy Statement.

Confidentiality is an essential component of high-quality health care for adolescents and young adults and can have an impact on the health care experiences and health outcomes of youth. Federal and state laws, professional guidelines, and ethical standards provide a core framework for guidance in the implementation of confidentiality protections in clinical practice. This policy statement provides recommendations for pediatricians and other pediatric health care professionals, clinics, health systems, payers, and electronic health record developers to optimize confidentiality practices and protections for adolescents and young adults across the spectrum of care.

Sofosbuvir-velpatasvir in children 3-17 years old with hepatitis C virus infection.

Background: The safety and efficacy of sofosbuvir-velpatasvir in children aged 3-17 years with chronic hepatitis C virus (HCV) infection of any genotype were evaluated.

Methods: In this Phase 2, multicenter, open-label study, patients received once daily for 12 weeks either sofosbuvir-velpatasvir 400/100 mg tablet (12-17 years), 200/50 mg low dose tablet or oral granules (3-11 years and ≥17 kg), or 150/37.5 mg oral granules (3-5 years and <17 kg).

Adjustment and homesickness in hospitalised children: A systematic review.

Children can experience significant distress during hospitalisation, as a result of the treatment process and due to psychosocial factors impacting their adjustment to the hospital environment. Such factors can contribute to negative outcomes for the child. Despite this, limited research focus has been placed on understanding the psychosocial factors that contribute to a child's distress to inform support strategies that can improve the experience of hospitalisation across paediatric conditions.

Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States.

Metachromatic leukodystrophy (MLD) is a fatal, progressive neurodegenerative disorder caused by biallelic pathogenic mutations in the ARSA (Arylsulfatase A) gene. With the advent of presymptomatic diagnosis and the availability of therapies with a narrow window for intervention, it is critical to define a standardized approach to diagnosis, presymptomatic monitoring, and clinical care. To meet the needs of the MLD community, a panel of MLD experts was established to develop disease-specific guidelines based on healthcare resources in the United States.

Transcatheter Pulmonary Valve Replacement With the Harmony Valve in Patients Who Do Not Meet Recommended Oversizing Criteria on the Screening Perimeter Plot.

Background: Anatomic selection for Harmony valve implant is determined with the aid of a screening report and perimeter plot (PP) that depicts the perimeter-derived radius along the right ventricular outflow tract (RVOT) and projects device oversizing. The PP provides an estimation of suitability for implant, but its sensitivity as a screening method is unknown. This study was performed to describe anatomic features and outcomes in patients who underwent Harmony TPV25 implant despite a PP that predicted inadequate oversizing.

Management of Hematochezia in Infants with Congenital Heart Disease Admitted to the Acute Care Cardiology Unit: A Multicenter Retrospective Pilot Study.

Objective: To assess the evaluation and prevalence of benign hematochezia (BH) vs necrotizing enterocolitis (NEC) in infants with congenital heart disease (CHD) <6 months old admitted to the acute care cardiology unit.

Study Design: This was a multicenter retrospective review of patient characteristics and evaluation of all hematochezia events in patients with CHD <6 months admitted to acute care cardiology unit at 3 high-volume tertiary care centers from February 2019 to January 2021. NEC was defined by the Bell staging criteria.

Feeding Practices in Infants with Hematochezia and Necrotizing Enterocolitis on Acute Care Cardiology Units.

Infants with congenital heart disease (CHD) are at risk for developing both benign hematochezia and necrotizing enterocolitis (NEC). Despite these risks there are very few studies that investigate modifiable risk factors such as feeding practices. It remains unclear what feeding practices should be avoided due to higher incidence of CHD-NEC.

A preclinical systematic review and meta-analysis assessing the effect of biological sex in lipopolysaccharide-induced acute lung injury.

It is unclear what effect biological sex has on outcomes of acute lung injury (ALI). Clinical studies are confounded by their observational design. We addressed this knowledge gap with a preclinical systematic review of ALI animal studies.

Multicenter Validation of the Charcot-Marie-Tooth Functional Outcome Measure.

Background And Objectives: Charcot-Marie-Tooth disease type 1A (CMT1A), caused by a duplication of , is the most common hereditary peripheral neuropathy. For participants with CMT1A, few clinical trials have been performed; however, multiple therapies have reached an advanced stage of preclinical development. In preparation for imminent clinical trials in participants with CMT1A, we have produced a Clinical Outcome Assessment (COA), known as the CMT-Functional Outcome Measure (CMT-FOM), in accordance with the FDA Roadmap to Patient-Focused Outcome Measurement to capture the key clinical end point of function.

Burden of paediatric hospitalisations to the health care system, child and family: a systematic review of Australian studies (1990-2022).

Background: Paediatric hospitalisations represent a significant cost to the health system and cause significant burden to children and their families. Understanding trends in hospitalisation costs can assist with health planning and support strategies across stakeholders. The objective of this systematic review is to examine the trends in costs and burden of paediatric hospitalisations in Australia to help inform policy and promote the well-being of children and their families.

Defining curative endpoints for transfusion-dependent β-thalassemia in the era of gene therapy and gene editing.

β-thalassemia is a monogenic disease that results in varying degrees of anemia. In the most severe form, known as transfusion-dependent β-thalassemia (TDT), the clinical hallmarks are ineffective erythropoiesis and a requirement of regular, life-long red blood cell transfusions, with the development of secondary clinical complications such as iron overload, end-organ damage, and a risk of early mortality. With the exception of allogeneic hematopoietic cell transplantation, current treatments for TDT address disease symptoms and not the underlying cause of disease.

Outcomes of Elastic Stable Intramedullary Nailing for Surgical Treatment of Pediatric Tibial Shaft Fractures.

Background: This retrospective study aimed to assess radiographic and clinical outcomes, characterize demographic, injury, and fracture characteristics, and elucidate the rate of postoperative complications and associated factors in a large cohort of children treated with elastic stable intramedullary nail for diaphyseal tibial fractures at two large pediatric referral centers.

Methods: Medical records were reviewed for demographic clinical and radiographic parameters at injury, surgery, and all subsequent clinical visits until radiographic healing was observed and/or for a minimum of 6 months postoperatively.

Results: A total of 146 patients (79.