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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This is a platform trial testing the addition of experimental treatments to standard of care (SoC) with three 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 efficacy 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 of each ...

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We are looking for new and better ways to manage melanoma, an aggressive type of skin cancer. Surgery to remove the melanoma will cure the majority of patients with early stage disease. However, a small percentage of these patients will go on to develop further disease, which may spread to other places in their body. At present, following surgery, patients with early stage melanoma can either choose to have close monitoring with regular scans and skin checks or they can ...

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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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To evaluate the effect of AP01 100 mg given twice daily (BID) and AP01 50 mg BID compared to AP01 placebo (hereafter referred to as placebo) on lung function over 52 weeks in subjects with PPF B17: Secondary objective(s) To evaluate the effect of AP01 100 mg given twice daily (BID) and AP01 50 mg BID compared to AP01 placebo (hereafter referred to as placebo) on lung function over 52 weeks in subjects with PPF B18: Full inclusion criteria

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Design: A 2x2 factorial pragmatic open-label randomised controlled trial,with an embedded randomised double-blind parallel group trial Setting: 60 general ICUs in the UK Target population: Adult patients who are critically ill due to sepsis Inclusion criteria - Adult patients admitted to ICU due to suspected sepsis and expected to stay for at least 48 hours - Receiving intravenous antibiotics for suspected sepsis - Initial early fluid resuscitation complete - Acute organ dysfunction due to infection Additional criteria for GM-CSF subset - Mechanically ventilated - Receiving vasopressors/inotropes - Absolute lymphocyte count <1.2 ...

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