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Research

Minimising Adverse Drug Reactions and Verifying Economic Legitimacy-Pharmacogenomics Implementation in Children (MARVEL- PIC): protocol for a national randomised controlled trial

DNA-informed prescribing (termed pharmacogenomics, PGx) is the epitome of personalised medicine. Despite international guidelines existing, its implementation in paediatric oncology remains sparse.

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Biologic and Clinical Analysis of Childhood Gamma Delta T-ALL Identifies LMO2/STAG2 Rearrangements as Extremely High Risk

Acute lymphoblastic leukemia expressing the gamma delta T-cell receptor (γδ T-ALL) is a poorly understood disease. We studied 200 children with γδ T-ALL from 13 clinical study groups to understand the clinical and genetic features of this disease. We found age and genetic drivers were significantly associated with outcome.

Research

Bacille Calmette-Guérin vaccination to prevent febrile and respiratory illness in adults (BRACE): secondary outcomes of a randomised controlled phase 3 trial

Bacille Calmette-Guérin (BCG) vaccination has off-target (non-specific) effects that are associated with protection against unrelated infections and decreased all-cause mortality in infants. We aimed to determine whether BCG vaccination prevents febrile and respiratory infections in adults.

Research

FeBRILe3: Risk-Stratification and Diagnosis of Serious Bacterial Infections in Febrile Infants Less Than 3 Months Old

Evidence-based recommendations exist for early discharge (before 48 h) of young infants with fever without source (FWS) at low risk of serious bacterial infections (SBIs). However, concerns regarding the applicability of international data to local contexts may hinder implementation. We aimed to describe the local epidemiology of FWS and evaluate a newly implemented risk-stratification guideline to support practice change.

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Immunogenicity, Reactogenicity, and Safety of a Pentavalent Meningococcal ABCWY Vaccine in Adolescents and Young Adults who had Previously Received a Meningococcal ACWY Vaccine

A MenABCWY vaccine containing 4CMenB and MenACWY-CRM vaccine components has been developed to protect against the 5 meningococcal serogroups that cause most invasive disease cases.

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Real-world glycaemic outcomes in children and young people on advanced hybrid closed-loop therapy: A population-based study in Western Australia

To evaluate real-world glycaemic outcomes in children with type 1 diabetes commencing advanced hybrid closed loop therapy and to explore these outcomes based on the cohort's clinical and socioeconomic characteristics.

Research

Short-Term Diabetic Retinopathy Status in People with Type 1 Diabetes Commencing Automated Insulin Delivery

Rapid improvements in glucose control may lead to early worsening of diabetic retinopathy (EWDR). There is a need to demonstrate safety in people commencing automated insulin delivery (AID) due to the known efficacy in rapid glycemic improvement. We aimed to investigate short-term DR outcomes in people (aged ≥13 years) with type 1 diabetes after initiation of AID (use ≥6 months).

Research

Community perspectives on the appropriateness and importance of support goals for young autistic children

Researchers do not know much about what autistic adults, parents and professionals think about support goals for young autistic children. People's views of support goals might also be influenced by their beliefs about early support more generally. This survey involved 87 autistic adults, 159 parents of autistic children and 80 clinical professionals living in New Zealand and Australia.

Research

Australian Clinicians’ Considerations When Choosing an Assessment of Functioning Tool for Children with Neurodevelopmental Conditions

In the Australian disability context, the assessment of children with neurodevelopmental conditions’ functioning (across all domains) is of increasing importance, particularly since the introduction of the National Disability Insurance Scheme. Currently, there is wide variability across assessment of functioning practices, including the choice and use of published tools for assessment.

Research

Early markers of adult symptoms of depression and anxiety in the Raine Study

Early intervention offers the potential to mitigate adult mental illness; however, trials spanning decades present significant challenges, necessitating predictive early markers useable in trial settings. We hypothesised that parent evaluation using the child behaviour checklist (CBCL) total problem score at age two years predicted adult depressive and anxious symptoms and explored other potential parent ratings.