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Research

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA

The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Research

Assessing the Use and Acceptability of Virtual Reality to Assist Coping in Children Undergoing Clinical Procedures

Virtual reality is used as a distraction tool during medical procedures that can cause anxiety and pain. We assessed the usefulness, engagement, value and feasibility of virtual reality to help children cope with routine clinical procedures.

Research

A roadmap for understanding sulfadoxine-pyrimethamine in malaria chemoprevention

Melissa Penny PhD, PD, BSc (Hons) Professor Fiona Stanley Chair in Child Health Research melissa.penny@thekids.org.au Professor Fiona Stanley Chair

Research

Machine learning techniques to predict diabetic ketoacidosis and HbA1c above 7% among individuals with type 1 diabetes — A large multi-centre study in Australia and New Zealand

Type 1 diabetes and diabetic ketoacidosis (DKA) have a significant impact on individuals and society across a wide spectrum. Our objective was to utilize machine learning techniques to predict DKA and HbA1c>7 %.

Research

Cause-Specific Secular Trends and Prevention Measures of Post-Neonatally Acquired Cerebral Palsy in Victoria and Western Australia 1975–2014: A Population-Based Observational Study

To describe the timing and causes of post-neonatally acquired cerebral palsy (PNN-CP) and map the implementation of relevant preventive strategies against cause-specific temporal trends in prevalence.

Research

Barriers and facilitators to mental health care access and engagement for LGBTQA+ people with psychosis: A scoping review

LGBTQA+ individuals are at increased risk of experiencing psychosis and face barriers in accessing appropriate and timely mental health support. This scoping review maps the existing literature to identify barriers and facilitators to access and engagement to care for LGBTQA+ people across the psychosis spectrum. 

Research

Invasive Fungal Disease in Immunocompromised Children: Current and Emerging Therapies

In an era of expanding indications for iatrogenic immunosuppression, invasive fungal disease (IFD) remains a significant challenge in immunocompromised children, with case fatality rates ranging from 10 to 70%. Understanding of current recommendations and recent evidence is essential to guide optimal IFD management.

Research

Systematic Review and Meta-Analysis: Early Irritability as a Transdiagnostic Neurodevelopmental Vulnerability to Later Mental Health Problems

Irritability is a transdiagnostic indicator of child and adolescent internalizing and externalizing problems that is measurable from early life. The objective of this systematic review was to determine the strength of the association between irritability measured from 0 to 5 years and later internalizing and externalizing problems, to identify mediators and moderators of these relationships, and to explore whether the strength of the association varied according to irritability operationalization.

Research

Gene editing and cardiac disease modelling for the interpretation of genetic variants of uncertain significance in congenital heart disease

Genomic sequencing in congenital heart disease (CHD) patients often discovers novel genetic variants, which are classified as variants of uncertain significance (VUS). Functional analysis of each VUS is required in specialised laboratories, to determine whether the VUS is disease causative or not, leading to lengthy diagnostic delays.

Research

Prevalence, distribution, and inequitable co-occurrence of mental ill-health and substance use among gender and sexuality diverse young people in Australia: epidemiological findings from a population-based cohort study

To estimate the prevalence, distribution, and co-occurrence of mental ill-health and substance use among gender and sexuality diverse young people relative to their cisgender and heterosexual peers in Australia using population-level, nationally representative data.