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Research

The psychosocial impact of rare diseases among children and adolescents attending mainstream schools in Western Australia

Living with a long-term medical condition is associated with heightened risk for mental health and psychosocial difficulties, but further research is required on this risk for children and adolescents with a rare disease in the educational setting. The aim of this study is to describe parents’ perceptions of the psychosocial impact of rare diseases on their school-aged children in Western Australia.

Research

Investigation of structural brain correlates of neurological soft signs in individuals at ultra-high risk for psychosis

Increased severity of neurological soft signs (NSS) in schizophrenia have been associated with abnormal brain morphology in cerebello-thalamo-cortical structures, but it is unclear whether similar structures underlie NSS prior to the onset of psychosis. The present study investigated the relationship between severity of NSS and grey matter volume in individuals at ultra-high risk for psychosis stratified for later conversion to psychosis.

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A screening tool to identify risk for bronchiectasis progression in children with cystic fibrosis

The marked heterogeneity in cystic fibrosis (CF) disease complicates the selection of those most likely to benefit from existing or emergent treatments. We aimed to predict the progression of bronchiectasis in preschool children with CF.

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Temporally restricted activation of IFNβ signaling determines response to immune checkpoint therapy

The biological determinants of the response to immune checkpoint blockade (ICB) in cancer remain incompletely understood. Little is known about dynamic biological events that underpin therapeutic efficacy due to the inability to frequently sample tumours in patients.

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Antifungal prescribing in neonates: Using national point prevalence survey data from Australia

We describe contemporary antifungal use in neonates, with point-prevalence survey data from the National Antimicrobial Prescribing Survey across Australian hospitals from 2014 to 2018.

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Novel method to select meaningful outcomes for evaluation in clinical trials

A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes.

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Carriage prevalence of extended-spectrum β-lactamase producing enterobacterales in outpatients attending community health centers in Blantyre, Malawi

Antimicrobial resistance due to extended-spectrum β-lactamase (ESBL) production by Enterobacterales is a global health problem contributing to increased morbidity and mortality, particu-larly in resource-constrained countries. We aimed to determine the prevalence of extended-spectrum β-lactamase-producing Enterobacterales (ESBL-E) in community patients in Blantyre, Malawi.

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Parent-observed thematic data on quality of life in children with autism spectrum disorder

Parent observations provide an initial framework for understanding quality of life in autism spectrum disorder

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Time to tackle rheumatic heart disease: Data needed to drive global policy dialogues

This report provides an update on the contemporary global and regional policy landscapes relevant to rheumatic heart disease