Archives of disease in childhood
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The principal cause of morbidity and mortality in cystic fibrosis (CF) is pulmonary disease, so the focus of new treatments in this condition is primarily targeted at the lungs. Since the cloning of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene in 1989, there has been significant interest in the possibility of gene therapy as a treatment for CF. Early studies using viral vectors carrying a healthy CFTR plasmid highlighted the difficulties with overcoming the body's host defences. This article reviews the work on gene therapy in CF to date and describes the ongoing work of the UK CF Gene Therapy Consortium in investigating the potential of gene therapy as a treatment for patients with CF.
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Chronic pain is an important clinical problem affecting significant numbers of children and their families. The severity and impact of chronic pain on everyday function is shaped by the complex interaction of biological, psychological and social factors that determine the experience of pain for each individual, rather than a straightforward reflection of the severity of disease or extent of tissue damage. In this article we present the research findings that strongly support a biopsychosocial concept of chronic pain, describe the current best evidence for management strategies and suggest a common general pathway for all types of chronic pain. The principles of management of some of the most important or frequently encountered chronic pain problems in paediatric practice; neuropathic pain, complex regional pain syndrome (CRPS), musculoskeletal pain, abdominal pain and headache are also described.
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To assess recruitment of children to national clinical trials for acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML) in Great Britain during 1980-2007 and describe variation by some factors that might influence trial entry. ⋯ Although recruitment rates to clinical trials for childhood leukaemia are high, future trials should monitor possible variation by birth weight, ethnicity and presence of congenital malformations.
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Exercise training is an underused intervention in paediatric healthcare. This is surprising, since initial evidence demonstrates its effectiveness and safety; furthermore it confers socioeconomic benefits for healthcare systems. ⋯ However, more research is needed to understand the pathophysiology, quantify treatment effects and monitor outcomes. A concerted effort from researchers, health professionals and police makers will be necessary to make exercise training an evidence-based and cost-effective intervention in paediatric care.
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To describe the characteristics of childhood burns and scalds, mechanisms and agents to inform prevention. ⋯ Scalds to infants and toddlers who pull hot beverages over themselves or sustain burns from touching irons, hair straighteners or oven hobs are a high priority for targeted prevention.