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Research

A matter of timing: Biting by malaria-infected Anopheles mosquitoes and the use of interventions during the night in rural southeastern Tanzania

Knowing when and where infected mosquitoes bite is required for estimating accurate measures of malaria risk, assessing outdoor exposure, and designing intervention strategies. This study combines secondary analyses of a human behaviour survey and an entomological survey carried out in the same area to estimate human exposure to malaria-infected Anopheles mosquitoes throughout the night in rural villages in south-eastern Tanzania.

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

Exploring Hope in Australian Justice Involved Youth with Fetal Alcohol Spectrum Disorder

Hope is well recognised as a positive protective factor for mental health, improved coping responses to adverse childhood events and better educational outcomes. Hope is composed of synergistic constituents – agency and pathway. A retrospective chart review was conducted of 53 justice-involved youths (10−17 years old) who underwent Fetal Alcohol Spectrum Disorder (FASD) diagnostic assessments with Patches in Western Australia between 2019 and 2020.

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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.

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Skin health of Aboriginal children living in urban communities

Skin concerns are frequent among urban-living Aboriginal children, yet specialist dermatology consultations are limited with studies highlighting the need for improved cultural security. Through newly established paediatric dermatology clinics at two urban Aboriginal Community Controlled Health Organisations (ACCHOs), we aimed to describe clinic and patient data, including disease frequencies and associations, to inform dermatology service provision and advocacy. 

Research

Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes. 

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Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trials

Existing clinical tools that measure non-seizure outcomes lack the range and granularity needed to capture skills in developmental and epileptic encephalopathy (DEE)-affected individuals who also fall in the severe to profound range of intellectual disability. This effectively excludes those with severe impairments from clinical trials, impeding the ability of sponsors to evaluate disease-modifying therapies.

Research

Efficacy thresholds and target populations for antiviral COVID-19 treatments to save lives and costs: a modelling study

In 2023 severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was declared endemic, yet hospital admissions have persisted and risen within populations at high and moderate risk of developing severe disease, which include those of older age, and those with co-morbidities. Antiviral treatments, currently only available for high-risk individuals, play an important role in preventing severe disease and hospitalisation within this subpopulation.

Research

Seasonal malaria chemoprevention and the spread of Plasmodium falciparum quintuple-mutant parasites resistant to sulfadoxine–pyrimethamine: a modelling study

Seasonal malaria chemoprevention (SMC) with sulfadoxine-pyrimethamine plus amodiaquine prevents millions of clinical malaria cases in children younger than 5 years in Africa's Sahel region. However, Plasmodium falciparum parasites partially resistant to sulfadoxine-pyrimethamine (with quintuple mutations) potentially threaten the protective effectiveness of SMC. We evaluated the spread of quintuple-mutant parasites and the clinical consequences. 

Research

The ETO2 transcriptional cofactor maintains acute leukemia by driving a MYB/EP300-dependent stemness program

Transcriptional cofactors of the ETO family are recurrent fusion partners in acute leukemia. We characterized the ETO2 regulome by integrating transcriptomic and chromatin binding analyses in human erythroleukemia xenografts and controlled ETO2 depletion models. We demonstrate that beyond its well-established repressive activity, ETO2 directly activates transcription of MYB, among other genes.