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A blueprint for a multi-disease, multi-domain Bayesian adaptive platform trial incorporating adult and paediatric subgroups: the Staphylococcus aureus Network Adaptive Platform trialThe Staphylococcus aureus Network Adaptive Platform (SNAP) trial is a multifactorial Bayesian adaptive platform trial that aims to improve the way that S. aureus bloodstream infection, a globally common and severe infectious disease, is treated. In a world first, the SNAP trial will simultaneously investigate the effects of multiple intervention modalities within multiple groups of participants with different forms of S. aureus bloodstream infection.
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Geldanamycin treatment does not result in anti-cancer activity in a preclinical model of orthotopic mesotheliomaMesothelioma is characterised by its aggressive invasive behaviour, affecting the surrounding tissues of the pleura or peritoneum. We compared an invasive pleural model with a non-invasive subcutaneous model of mesothelioma and performed transcriptomic analyses on the tumour samples.
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Blinatumomab Added to Chemotherapy in Infant Lymphoblastic LeukemiaKMT2A-rearranged acute lymphoblastic leukemia (ALL) in infants is an aggressive disease with 3-year event-free survival below 40%. Most relapses occur during treatment, with two thirds occurring within 1 year and 90% within 2 years after diagnosis. Outcomes have not improved in recent decades despite intensification of chemotherapy.
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Childhood-onset type 1 diabetes in Western Australia: An update on incidence and temporal trends from 2001 to 2022To determine the incidence and incidence trends over 2001-2022 of childhood-onset type 1 diabetes (T1D) in Western Australia and assess the impact of the COVID-19 pandemic.
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Short-course, high-dose primaquine regimens for the treatment of liver-stage vivax malaria in childrenTo assess the pharmacokinetics, safety, and tolerability of two high-dose, short-course primaquine (PQ) regimens compared with standard care in children with Plasmodium vivax infections.
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Protocol for a systematic review of long-term physical sequelae and financial burden of multidrug-resistant and extensively drug-resistant tuberculosisMultidrug resistant (MDR) and extensively drug-resistant (XDR) tuberculosis (TB) are major public health threats that are significant causes of physical sequelae and financial consequences for infected people. Treatment for MDR- and XDR-TB are more toxic and take longer duration than for drug-susceptible-TB. As a result, the long-term sequelae are thought to be more common among patients with MDR- and XDR-TB than drug-susceptible-TB, but this is yet to be quantified.
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Bivalent Prefusion F Vaccine in Pregnancy to Prevent RSV Illness in InfantsWhether vaccination during pregnancy could reduce the burden of respiratory syncytial virus (RSV)-associated lower respiratory tract illness in newborns and infants is uncertain.
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Arcuate fasciculus and pre-reading language development in children with prenatal alcohol exposurePrenatal alcohol exposure (PAE) contributes to widespread neurodevelopmental challenges, including reading, and has been associated with altered white matter. Here, we aimed to investigate whether arcuate fasciculus development is associated with pre-reading language skills in young children with PAE.
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Psychometric Properties of the Polish Version of the Perth Emotional Reactivity ScaleThe Perth Emotional Reactivity Scale (PERS) is a 30-item self-report questionnaire that assesses the ease of activation, intensity, and duration of negative and positive emotions. Our study aimed to introduce and validate the Polish version of the PERS.
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Incidence and survival for childhood cancer by endorsed non-stage prognostic indicators in AustraliaNick Gottardo MBChB FRACP PhD Head of Paediatric and Adolescent Oncology and Haematology, Perth Children’s Hospital; Co-head, Brain Tumour Research