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Research

Verschlimmbesserung: Craniospinal Radiotherapy Is Essential in WNT Medulloblastoma Patients

Standard-risk WNT medulloblastoma patients have an excellent prognosis using the combination of standard dose craniospinal radiotherapy (CSI) followed by platinum and alkylator based chemotherapy. A recent pilot study that attempted to completely omit radiotherapy was terminated early as all patients relapsed rapidly. The study highlights that therapy is the most important prognostic factor, with CSI still required to cure even the most favorable subgroup of medulloblastoma patients. 

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The effectiveness of community friendship groups on participant social and mental health: a meta-analysis

Social disconnectedness and isolation are risk factors for poor mental health. Community-based friendship group interventions have been designed to increase an individual's social capital and consequently their mental wellbeing. Structured and unstructured friendship groups reflect two distinct approaches to friendship group interventions.

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Presence of onco-fetal neighborhoods in hepatocellular carcinoma is associated with relapse and response to immunotherapy

Onco-fetal reprogramming of the tumor ecosystem induces fetal developmental signatures in the tumor microenvironment, leading to immunosuppressive features. Here, we employed single-cell RNA sequencing, spatial transcriptomics and bulk RNA sequencing to delineate specific cell subsets involved in hepatocellular carcinoma  relapse and response to immunotherapy. 

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Outcomes following intensive day rehabilitation for young people in Western Australia

Intensive rehabilitation aims to improve and maintain functioning in young people who experience disability due to illness or injury. Day rehabilitation may have advantages for families and healthcare systems over inpatient models of rehabilitation. 

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An evaluation of GPT models for phenotype concept recognition

Clinical deep phenotyping and phenotype annotation play a critical role in both the diagnosis of patients with rare disorders as well as in building computationally-tractable knowledge in the rare disorders field.

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Subcutaneous infusion of high-dose benzathine penicillin G is safe, tolerable, and suitable for less-frequent dosing for rheumatic heart disease secondary prophylaxis: a phase 1 open-label population pharmacokinetic study

Since 1955, the recommended strategy for rheumatic heart disease secondary prophylaxis has been benzathine penicillin G injections administered intramuscularly every 4 weeks. Due to dosing frequency, pain, and programmatic challenges, adherence is suboptimal. It has previously been demonstrated that BPG delivered subcutaneously at a standard dose is safe and tolerable and has favorable pharmacokinetics, setting the scene for improved regimens with less frequent administration.

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Morbidity of Scabies in Resource-Limited Countries: Rheumatic Heart Disease (RHD) and Post-Streptococcal Glomerulonephritis (APSGN)

Scabies is one of the world’s most prevalent diseases, with approximately 147 million cases at any one time and an estimated annual incidence of 455 million new episodes. Although Group A streptococcal (GAS) pharyngitis has long been implicated in the pathogenesis of acute rheumatic fever (ARF) and subsequent rheumatic heart disease (RHD), impetigo caused by GAS has recently been postulated as a link between scabies and the pathogenesis of ARF.

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Research opportunities for the primordial prevention of rheumatic fever and rheumatic heart disease - streptococcal vaccine development: a national heart, lung and blood institute workshop report

Streptococcus pyogenes, also known as group A streptococcus (StrepA), is a bacterium that causes a range of human diseases, including pharyngitis, impetigo, invasive infections, and post-infection immune sequelae such as rheumatic fever and rheumatic heart disease. StrepA infections cause some of the highest burden of disease and death in mostly young populations in low-resource settings. Despite decades of effort, there is still no licensed StrepA vaccine, which if developed, could be a cost-effective way to reduce the incidence of disease. 

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Intravascular Tumor Extension and Pulmonary Tumor Embolism in Children With Solid Malignancies: Is There a Role for Inferior Vena Cava Filters?

Intravascular tumor extension is an uncommon complication of solid malignancies that, when present in the inferior vena cava (IVC), can result in fatal pulmonary tumor embolism. Currently, neoadjuvant chemotherapy and surgery are the mainstays of treatment; however, there are no consensus guidelines for management.  

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Predictors and outcomes of engagement in an online depression prevention program for final year secondary school students

Although school-based delivery of online interventions can effectively prevent depression and other common mental disorders, little is known about the characteristics of students who engage with these programs. This study aimed to identify predictors of two indicators of adolescent engagement (program usage and skill enactment) with a school-based online depression prevention program. The study also explored the association between skill enactment and mental health outcomes.