Background Paediatric Rheumatology is a term that covers over 80 conditions, affecting different parts of the body, such as Juvenile Arthritis. Children and young people (CYP) with rheumatological conditions can have high levels of mental health problems and therefore are at risk of poor health outcomes. We need to look at novel ways of providing early, essential support, to improve their current wellbeing. Our aims This study will create a digital-service that can guide parents through ways of supporting their CYP at ...

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This study is a multi-centre observational, prospective registry of patients with high-risk neuroblastoma treated with Dinutuximab beta. Pain is an almost universal toxicity associated with anti-GD2 immunotherapy, and can be severe despite opioid analgesia. The aim of the study is to better understand the mechanism of pain is patients receving this immunotherapy, with the ultimate aim of designing better, less toxic anti-GD2 antibodies. The study is non-interventional. All treatment decisions are made according to normal clinical practice and are not ...

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Inflammatory arthritis usually prevents people from doing things through causing joints to become swollen and painful, or their spine to become stiff. People with arthritis usually require long-term treatment with medications and are typically reviewed in outpatient clinics every 6-12 months. Many of these appointments are unnecessary as patients are well at the time of follow-up, wasting patients’ time and NHS resources. NHS England has proposed that many people with inflammatory arthritis should no longer have routine follow-up appointments, but be ...

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Mitochondria are crucial components found in most cells that generate energy from food to enable cells to function normally. “Spelling mistakes” (mutations) in the POLG gene, cause mitochondrial dysfunction and subsequently disease, in organs with high energy demands. Cerebellum, the part of the brain that regulates our balance and movement coordination, and peripheral nerves, are frequently affected in people with POLG-related mitochondrial disease. They develop ataxia, which is a medical term describing symptoms related to balance impairment and muscle incoordination, ...

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In the UK, heart operations have steadily increased since 2010, and 36,166 heart operations were performed in 2016. Following cardiac surgery, patients currently attend their first outpatient review six weeks after hospital discharge, where recovery is assessed and fitness to commence cardiac rehabilitation (CR) is determined. CR is then started from eight weeks. In a survey we conducted in May/June 2017, 35 of the 42 UK cardiac centres responded, and confirmed this as current practice. The long interval before postoperative ...

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The spleen is often injured when the body sustains trauma. This leads to bleeding. The bleeding can be stopped by a big operation cutting open the belly or a small hole in your groin where a blood vessel can be accessed and through which the bleeding can be stopped. We do not know what types of injuries it is best to use this procedure. We do not know why we do not use the smaller technique ...

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Despite advances with the introduction of anti-GD2 immunotherapy,there is a major unmet need to develop new drugs for treatment of high-risk neuroblastoma. Around half of patients relapse despite intensive therapies,and outcome following relapse is dismal (<10% long term survival). The BEACON2 trial will confirm which combination of drugs tested in the phase II BEACON study should be taken forward and identify novel combinations that further improve survival in this patient group. Based on our experience in a previous trial (BEACON),the ...

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This is a platform trial testing the addition of experimental treatments to standard of care (SoC) with two randomised controlled trials called research comparisons. The platform design allows the addition of other research treatments into the structure following protocol amendment. The primary objectives of each comparison are to test whether survival and/or cancer outcomes can be improved by the addition of the research treatments to SoC in men with metastatic prostate cancer and starting long-term androgen deprivation therapy. Key secondary objectives ...

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This is a first-in-human Phase I, open-label, multicenter, dose-escalation and expansion study designed to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary anti-tumour activity of RO7566802 as a single agent and in combination with atezolizumab in patients with locally advanced, recurrent, or metastatic incurable solid tumour malignancies. The study consists of a screening period of up to 28 days, a treatment period, a minimum follow-up period of 90 days after treatment, and survival follow-up. Patients will be enrolled in two stages: a ...

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We will use state-of-the-art imaging to look at heart disease in people with haemophilia. Haemophilia is an inherited disorder in which blood does not clot properly because of lack of a key ‘glue’ blood component (chemicals known as factor VIII or IX). People with haemophilia are 40% less likely to die of heart disease, but we don’t know why. Understanding heart disease in people with haemophilia is important because better treatments for haemophilia mean that these patients are now ...

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