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Research

Population-level 5-year event-free survival for children with cancer in Australia

Event-free survival considers other adverse events in addition to mortality. It therefore provides a more complete understanding of the effectiveness and consequences of treatment than standard survival measures, but is rarely reported at the population level for childhood cancer.

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Challenges and considerations for antifungal prophylaxis in children with acute myeloid leukemia

Children receiving treatment for acute myeloid leukemia (AML) are at high risk of invasive fungal disease (IFD). Evidence from pediatric studies support the efficacy of antifungal prophylaxis in reducing the burden of IFD in children receiving therapy for AML, yet existing antifungal agents have specific limitations and comparative data to inform the optimal prophylactic approach are lacking.

Research

Developmental Relationship-Based Interventions for Autistic Children

Andrew Videos Whitehouse Watch and listen to Andrew PhD Deputy Director (Research); Angela Wright Bennett Professor of Autism Research at The Kids

Research

Autism likelihood in infants born to mothers with asthma is associated with blood inflammatory gene biomarkers in pregnancy

Mothers with asthma or atopy have a higher likelihood of having autistic children, with maternal immune activation in pregnancy implicated as a mechanism. This study aimed to determine, in a prospective cohort of mothers with asthma and their infants, whether inflammatory gene expression in pregnancy is associated with likelihood of future autism. 

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Short-Term Active Safety Surveillance of the Spikevax and Nuvaxovid Priming Doses in Australia

Australia commenced administration of the Spikevax (Moderna mRNA-1273) COVID-19 vaccine in August 2021 and Nuvaxovid (Novavax NVX-CoV2373) in January 2022. This study describes the short-term safety profile of priming doses of the Spikevax and Nuvaxovid vaccines given between September 2021 and September 2023. 

Research

Per Os to Protection – Targeting the Oral Route to Enhance Immune-mediated Protection from Disease of the Human Newborn

Valerie Verhasselt MD, PhD Head, Immunology and Breastfeeding 0402997617 Valerie.verhasselt@thekids.org.au Head, Immunology and Breastfeeding @

Research

Widespread dyspigmentation in a child

Bernadette Ricciardo MBBS (hon) DCH FACD PhD Candidate Bernadette.Ricciardo@thekids.org.au PhD Candidate Dr Bernadette Ricciardo is a PhD student on

Research

Feasibility and acceptability of implementing an evidence-based ESCALATION system for paediatric clinical deterioration

The ESCALATION system is a novel paediatric Early Warning System that incorporates family involvement and sepsis recognition. This study aimed to assess the feasibility and iteratively refine the ESCALATION system in a variety of hospital settings in preparation for full-service implementation.

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ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

Diffuse midline gliomas (DMG), including diffuse intrinsic pontine gliomas (DIPG), are the most lethal of childhood cancers. Palliative radiotherapy is the only established treatment, with median patient survival of 9 to 11 months. ONC201 is a DRD2 antagonist and ClpP agonist that has shown preclinical and emerging clinical efficacy in DMG. 

Research

A novel transcriptional signature identifies T-cell infiltration in high-risk paediatric cancer

Molecular profiling of the tumour immune microenvironment (TIME) has enabled the rational choice of immunotherapies in some adult cancers. In contrast, the TIME of paediatric cancers is relatively unexplored. We speculated that a more refined appreciation of the TIME in childhood cancers, rather than a reliance on commonly used biomarkers such as tumour mutation burden (TMB), neoantigen load and PD-L1 expression, is an essential prerequisite for improved immunotherapies in childhood solid cancers.