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Despite recent improvements in treatment modalities for cystic fibrosis (CF), there is currently limited evidence and a lack of consensus regarding optimal treatment strategies for the different aspects of CF, including pulmonary exacerbations (PEx). We aimed to establish a prospective cohort of people with CF (pwCF) to evaluate alternative approaches to managing CF in the era of modulator therapies.
Vaccine policy and guideline recommendations require high quality evidence. A review of the evidence quality used to inform vaccine clinical practice guidelines could help guide researchers on how to improve the design of their clinical studies to produce evidence of greater value to decision-makers.
Acute rheumatic fever is a preventable condition that can lead to chronic illness and early death. Standard prevention with 4-weekly intramuscular (IM) benzathine penicillin G (BPG) injections for ≥10 years may be associated with poor adherence. High-dose 10-weekly subcutaneous penicillin injections (SCIP) may improve adherence by reducing injection frequency.
Pneumonia remains a leading cause of hospitalization and death among young children worldwide, and the diagnostic challenge of differentiating bacterial from non-bacterial pneumonia is the main driver of antibiotic use for treating pneumonia in children. Causal Bayesian networks (BNs) serve as powerful tools for this problem as they provide clear maps of probabilistic relationships between variables and produce results in an explainable way by incorporating both domain expert knowledge and numerical data.
Cystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease. The emergence of innovative trials and use of Bayesian statistical methods has created renewed opportunities for studying heterogeneous populations in rare diseases.
Patient-reported outcome measures (PROMs) are recommended for capturing meaningful outcomes in clinical trials. The use of PROMs for children with acute lower respiratory infections (ALRIs) has not been systematically reported. We aimed to identify and characterise patient-reported outcomes and PROMs used in paediatric ALRI studies and summarise their measurement properties.
The need for coronavirus 2019 (COVID-19) vaccination in different age groups and populations is a subject of great uncertainty and an ongoing global debate. Critical knowledge gaps regarding COVID-19 vaccination include the duration of protection offered by different priming and booster vaccination regimens in different populations, including homologous or heterologous schedules.
Diagnosing urinary tract infections (UTIs) in children in the emergency department (ED) is challenging due to the variable clinical presentations and difficulties in obtaining a urine sample free from contamination.
Population-level studies of severe pertussis extending beyond infancy are sparse, and none in the context of antenatal vaccination. We compared hospitalized pertussis cases from birth to 15 years of age before and after introduction of antenatal immunization.
To determine the proportion of first status epilepticus cases that are vaccine-proximate and compare clinical outcomes to non-vaccine-proximate cases.