Childhood and adolescent cancers comprise a heterogeneous group of rare diseases, mostly distinct to those diagnosed in adults. Survival of children and adolescents with cancer has considerably improved in recent decades, but cancer remains the most common cause of disease-related mortality in developed countries. In the case of disease relapse, the prognosis for paediatric patients is poor with survival rates of < 20%. Better understanding of tumor biology has led to exponentially increased development of novel targeted therapies and immunotherapies in ...

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Stomach cancer is the fifth most common cancer in the UK and most (70%) of cases are diagnosed when the cancer is already at an advanced stage. Stomach cancer arises following chronic inflammation of the stomach. Helicobacter pylori (H. pylori) is a bacteria that can cause an infection in the stomach. It is very common, some people estimate that 50% of people worldwide are infected with H. pylori. For a small number of people, this infection can lead to ...

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Nemaline myopathies(NM) are genetically determined conditions that usually present with muscle weakness, breathing problems and difficultly feeding. The severity of NM varies from significant muscle weakness requiring a wheelchair, breathing and feeding support with signifcantly reduced life expectancy to milder forms in which the person remains able to walk and has a normal lifespan. Current treatments for people living with NM are supportive only. Several potential therapies are in development which may be available in the next 5-10 years. ...

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People with familial hypercholesterolaemia (FH) have raised cholesterol levels in their blood from birth. Cholesterol is a type of fat our bodies need but having raised levels over a long time causes heart disease, such as heart attacks. To prevent future heart disease, guidelines recommend starting cholesterol-reducing medication, usually statins, by age 10. However, there remain unanswered questions about what is the right age and cholesterol level at which to start statins to prevent later life heart disease. Considerations are: avoiding ...

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Multisite,multi-arm,open-label,pragmatic randomised controlled trial with integral internal pilot and concurrent economic evaluation. The trial is predicated on two primary comparisons of (1) a monotherapy (i.e. Iron) and (2) a combination therapy (i.e. Iron + EPO),compared with a single common control group (usual care) respectively,in a superiority hypothesis testing framework.

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This is a Phase 3, randomized, controlled, multicenter, open-label study to evaluate the efficacy of IMC-F106C + nivolumab compared with nivolumab regimens in HLA-A*02:01-positive adult participants with previously untreated advanced (unresectable or metastatic) melanoma. Participants will be randomized 1:1:1 to IMC-F106C 40 mcg + nivolumab (Arm A), IMC-F106C 160 mcg + nivolumab (Arm B), or a nivolumab regimen (Arm C). The control Arm C will be country-specific and include: • Nivolumab + relatlimab, an emerging SoC, in countries where this combination ...

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cases in 2020 globally (11.7% of all new cancers). Breast cancer is also the fifth most common cause of death from cancer, with an estimated 685000 deaths in 2020 (Sung et al 2021). Despite advances in the diagnosis and treatment of breast cancer, around 5% to 10% of women diagnosed with breast cancer have metastatic disease at the time of diagnosis, with a much higher percentage in low- and middle-income countries, and up to 30% of women with early-stage non-metastatic ...

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Each year in England, about 3,000 babies are born with one of about 200 genetic conditions that are treatable in early childhood. Nine of these conditions are currently looked for in the NHS newborn bloodspot test. But there are many more conditions where earlier diagnosis and treatment could help. This study investigates the genomes of newborn babies to see if we can find and treat rare genetic conditions early. A genome is a person’s entire genetic sequence ...

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Patient-reported outcome measures (PROMs) are questionnaires that measure aspects of somebody’s health, like hand function. They can monitor a person’s health over time. Our team has made a smartphone app that make these questionnaires shorter. Previous research suggests that unlike the full-length questionnaire, people do not mind using the app every day. This could show us how quickly people recover after different types of hand surgery. We want to see whether our app could be used to compare patients’ experiences ...

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We may be able to rescue a generation of patients who are being abandoned to progressive injury to their heart from previous-generation pacemakers. This is because there is no randomised evidence to show that they would fare better with upgrade to modern pacing strategies. Today, if patients who require pacemakers for slow heart rates have even mildly impaired heart function, they receive modern pacing strategies (via His bundle, left bundle or biventricular pacing) rather than traditional right ventricular (RV pacing. ...

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