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Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study

Aim: To describe the real-world effects of trofinetide in individuals with Rett syndrome (RTT) using the 18-month follow-up analysis of the LOTUS study.

A qualitative exploration of the phenomenology of pain in children to inform pain assessment methods

Pain is a common experience associated with healthcare for children, who often recall it as the worst part of hospitalisation. Several factors make assessment of pain more challenging in children. Families have previously identified the development of improved tools to assess pain in children as a key priority. We therefore sough to investigate the nature of this experience from the perspective of children and their parents to inform the development of such tools.

Finding the optimal regimen for Mycobacteroides abscessus treatment (FORMaT) in people with Mycobacteroides abscessus pulmonary disease

Mycobacteroides abscessus (MABS) is within the non-tuberculous mycobacteria family. It inhabits soil and water, exhibits multi-antibiotic resistance and causes opportunistic lung infections, which may progress to symptomatic MABS-pulmonary disease (MABS-PD) associated with substantial morbidity, increased healthcare utilisation, impaired quality of life and increased mortality. 

Scoping review of variation in clinical guidelines for delivery of injectable long-acting penicillin across Australia and Aotearoa New Zealand

This scoping review explores existing clinical guidelines on administration of benzathine benzylpenicillin (Bicillin L-A, Pfizer Australia) in Australia and Aotearoa New Zealand. The objective is to understand existing delivery guidance to address variation in care and cultural safety considerations, to support messaging during periods of stockout and to inform planning for new administration techniques.

Antimicrobial Resistance of Clostridioides (Clostridium) difficile in Cambodia

Antimicrobial resistance (AMR) remains a major topic of interest in infectious disease management. We studied AMR in Clostridioides difficile isolated in Cambodia.

“Wait with me until it's over”: A reflexive thematic analysis of adolescents’ coping with dissociation

Dissociation is often distressing, yet how affected adolescents manage dissociation in their daily lives is not well understood. This study aimed to describe the strategies adolescents use to manage dissociative symptoms from the perspective of adolescents themselves, their parents, and their clinicians.

Perceived stigma and self-stigma in young people at ultra-high risk for psychosis: Associations with identity-related, psychological and functional outcomes

Perceived stigma and self-stigma negatively affect identity-related, psychological and functional outcomes among stigmatised populations. There is limited research exploring the impact of stigma among young people at ultra-high risk (UHR) for psychosis. We investigated the association of perceived stigma and self-stigma with these outcomes in young people at UHR.

Pharmacogenomics in pediatric oncology: Australian adolescent or young adult and caregiver perspectives

Preemptive pharmacogenomic (PGx) testing in pediatric oncology patients could reduce toxicity and improve efficacy of medications yet remains underutilized. Consumer identified implementation barriers have not been extensively explored nor included adolescent or young adult (AYA) patient perspectives. This study describes Australian pediatric oncology consumer perspectives on PGx testing, elucidating barriers to implementation.

Quantifying undetected tuberculosis in Ethiopia using a novel geospatial modelling approach

Tuberculosis (TB) is the leading infectious cause of death globally, with approximately three million cases remaining undetected, thereby contributing to community transmission. Understanding the spatial distribution of undetected TB in high-burden settings is critical for designing and implementing geographically targeted interventions for early detection and control.

The Luminos Project: Co-Designing a Short-Stay Suicide Support Model for Young People

Suicide was the leading cause of death among young Australians aged 15-24 years old in 2023, with 392 lives lost. The continued high numbers of youth suicide demand urgent exploration of alternative approaches to suicide intervention in this population. The United Kingdom-based suicide service Maytree offers an innovative short-term stay for people experiencing suicidal thoughts. Grounded by the Maytree model-of-care, the aim of the current study was to co-design a short-stay service responsive to the specific needs of suicidal young people.