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Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimise new therapeutic interventions.
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Randomised controlled trials in cystic fibrosis: What, when and how?The major morbidity and mortality from cystic fibrosis (CF) comes from progressive lung disease with bronchiectasis leading to respiratory failure
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Dysphagia in laryngomalacia: a prospective cohort studyDysphagia is an under recognised co-morbidity in patients with laryngomalacia. Its rate is variable reported in the literature. We aim to describe the incidence of dysphagia in laryngomalacia, the effect of interventions on this, and the period it persists in these infants.
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Risk factors associated with unsuccessful tuberculosis treatment outcomes in Hunan Province, ChinaGlobally, China has the third highest number of tuberculosis (TB) cases despite high rates (85.6%) of effective treatment coverage. Identifying risk factors associated with unsuccessful treatment outcomes is an important component of maximising the efficacy of TB control programmes.
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Determinants of sleep problems in children with intellectual disabilityChildren with intellectual disabilities are more likely to experience sleep disorders of insomnia, excessive daytime sleepiness and sleep breathing disorders than typically developing children. The present study examined risk factors for these sleep disorders in 447 children (aged 5-18 years), diagnosed with an intellectual disability and comorbid autism spectrum disorder, cerebral palsy, Down syndrome or Rett syndrome. Primary caregivers reported on their child's sleep using the Sleep Disturbance Scale for Children (SDSC), as well as medical comorbidities and functional abilities.
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Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic reviewThere is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations in people with cystic fibrosis (CF). Outcomes used for evaluation should be meaningful; that is, they should capture how people feel, function or survive and be acknowledged as important to people with CF, or should be reliable surrogates of those outcomes. We aimed to summarise the outcomes and corresponding endpoints which have been reported in studies of pulmonary exacerbations, and to identify those which are most likely to be meaningful.
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Structural determinants of long term functional outcomes in young children with cystic fibrosisChest CT identifies children at an early age who have adverse long-term outcomes
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Phage therapy to treat cystic fibrosis Burkholderia cepacia complex lung infections: perspectives and challengesgeBurkholderia cepacia complex is a cause of serious lung infections in people with cystic fibrosis, exhibiting extremely high levels of antimicrobial resistance. These infections are difficult to treat and are associated with high morbidity and mortality.
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Transcriptomic analysis of primary nasal epithelial cells reveals altered interferon signalling in preterm birth survivors at one year of ageMany survivors of preterm birth (<37 weeks gestation) have lifelong respiratory deficits, the drivers of which remain unknown. Influencers of pathophysiological outcomes are often detectable at the gene level and pinpointing these differences can help guide targeted research and interventions. This study provides the first transcriptomic analysis of primary nasal airway epithelial cells in survivors of preterm birth at approximately 1 year of age.
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Multiple-breath washout outcomes are sensitive to inflammation and infection in children with cystic fibrosisThe lung clearance index may be a useful surveillance tool for monitoring the presence and extent of lower airway inflammation and infection