Royal Children's Hospital

Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership

Summary Co‐designed research in paediatric HSCT is limited. We sought to determine research priorities which represent the shared priorities of patients, parents, carers and healthcare professionals (HCP) within Australia, New Zealand, the Netherlands and the United Kingdom. An i...

Multidomain Post-Concussive Symptom Trajectories and Biopsychosocial Correlates Following Pediatric Concussion

IMPORTANCE: Understanding how post-concussive symptoms (PCS) evolve across multiple domains and how these patterns relate to biopsychosocial factors is critical for identifying children at risk of prolonged recovery following concussion. OBJECTIVE: To identify clinically meaningf...

Heterochronicity shapes brain network formation

Brain network formation unfolds on a nonuniform developmental timetable, with different cortical regions generating connections at different developmental phases. Generative network models (GNMs) aim to uncover the principles underpinning the organization of connectomes by creati...

An international multicenter analysis of the outcome of pediatric first variceal hemorrhage

Background & Aims: Management of portal hypertension in children including primary prophylaxis of variceal bleeding is controversial due to limited understanding of mortality risk after first variceal hemorrhage (1VH). This international multicenter study was designed to advance...

Anesthesia as a Partner in Public Health: Enabling National Pediatric SARS-CoV-2 Serosurveillance in Australia

Anesthetic departments played a critical role in Australia’s COVID-19 response by facilitating the collection of residual blood samples from children undergoing elective procedures. Through a partnership with the Pediatric Active Enhanced Disease Surveillance network, this approa...

Corticosteroid use in children with community acquired sepsis: a multicentre prospective cohort study

Objectives Corticosteroids in paediatric sepsis have the potential to improve outcomes, yet there is little supporting evidence regarding efficacy, choice of agents, dose or timing of administration. We describe the patterns of corticosteroid use in children hospitalised with sus...

Tension pneumothorax – a rare complication of ventriculoperitoneal shunt

Background Ventriculoperitoneal shunt placement is a standard treatment for hydrocephalus. Common complications include shunt infection, obstruction, malfunction and abdominal complications. Thoracic complications, however, are rare. Observations We report on a five-year-old Maur...

Genome-wide analysis of social behaviour across social domains, reporters and developmental stages: a meta-regression approach

Abstract Social behaviour is a heritable trait linked to well-being and mental health, yet it varies across social context, including social domains, informants and development, which may shape its underlying genomic architecture. Here, we conducted a genome-wide meta-regression...

Implementing Sequential Analysis for Vaccine Safety Surveillance in Victoria, Australia

This preprint presents an end-to-end implementation framework for MaxSPRT-style sequential analysis in vaccine safety surveillance, using the Surveillance of Adverse Events Following Vaccination in the Community (SAEFVIC) system in Victoria, Australia, as the operational setting....

Adenovirus infection after cellular therapy: diagnostic and therapeutic challenges

Purpose of review To review recent advances in the epidemiology, diagnosis, and management of human adenovirus (HAdV) infection following haematopoietic cell transplantation (HCT) and other cellular therapies, with emphasis on risk stratification, molecular surveillance, and emer...

Neuroradiologic Findings in Patients With S CN2A Disease

SCN2A disease spans a broad clinical spectrum from self-limited (familial) neonatal-infantile epilepsy to severe developmental and epileptic encephalopathies. Systematic characterization of the spectrum of neuroradiologic abnormalities in SCN2A disease has not been performed. Thi...

Promotion of long-term health in early life

Noncommunicable chronic diseases (NCDs) are on the rise in high-income countries, leading to substantial morbidity and mortality and contributing to increased resource demands and healthcare costs. Early-life exposures can initiate trajectories that culminate in NCDs later in lif...

Implementing a patient decision aid about selective dorsal rhizotomy in clinical practice: pre-post pilot study

PURPOSE: To evaluate whether implementing a patient decision aid about Selective Dorsal Rhizotomy (SDR) for carers of children with bilateral spastic cerebral palsy (CP) improves carer decision-making outcomes and is acceptable to healthcare providers, and to explore carers' expe...

Quality and concordance of international paediatric sepsis guidelines: a cross-sectional appraisal from the SENTINEL International study

Objectives To evaluate the methodological quality of hospital-specific paediatric sepsis clinical practice guidelines (CPGs) from sites participating in an international study using the Appraisal of Guidelines for Research and Evaluation II (AGREE II) tool and to assess the consi...

Diabetes and Driving: How Predictive Technologies Will Alter Ethical and Legal Responsibilities

In 2023, a 67-year-old man with long-standing type 1 diabetes experienced a severe hypoglycemic episode and drove into an outdoor dining area in Victoria, Australia, killing 5 people and injuring several others. Although the charges against the driver were dismissed, future cases...

Human-inherited RORγT deficiency encompasses genetic heterogeneity, T cell deficiency, and clinical homogeneity

Abstract We previously reported inherited RORγT deficiency in seven patients from three ancestries (Chilean, Palestinian, Saudi Arabian) with mycobacterial disease and chronic mucocutaneous candidiasis (CMC). We report here five additional patients from different ancestries (Afgh...

A Modified Delphi Study on the Ethics and Governance of Live Injecting at Aesthetic Conferences. Is There a Better and Safer Alternative?

BACKGROUND: Live clinical demonstrations at aesthetic medicine conferences is common, yet concerns regarding patient safety, commercial influence, and ethical governance. This study established a consensus-based governance framework for live injecting and compared expert and atte...

Disruptions in speech, language and social communication in lysosomal storage diseases

Communication enables social connection, autonomy and identity, making it a fundamental domain of human functioning. In lysosomal storage diseases (LSDs), progressive neurological dysfunction can severely disrupt speech, language and social communication, resulting in profound is...

A necroptotic-to-apoptotic signaling axis underlies inflammatory bowel disease

ABSTRACT Inflammatory bowel disease (IBD) is a chronic condition caused by altered cytokine signaling, maladaptive immunity, dysbiosis, and intestinal barrier dysfunction. Patients with IBD receive therapy to correct these imbalances and achieve remission. However, most patients...

Efficacy and safety of fenfluramine in Dravet syndrome: The impact of patient clinical characteristics

Abstract Objective To assess the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) stratified by age, number of previously attempted antiseizure medications (ASMs), and SCN1A pathogenic variant status. Methods In this post hoc analysis, data from three ran...