Vitamin D replacement in children with acute wheeze: a dose-escalation study.

https://bit.ly/3J43Ouo.

Identification of a novel cystic fibrosis mutation in three patients of South Asian descent.

Introduction: The cystic fibrosis (CF) clinical profile and associated CFTR mutation spectrum is poorly understood in the South Asian population. This is likely due to the lack of diagnostic resources and the absence of a centralised CF database and screening programme, despite a relatively large proportion of the global population.

Methods: Following identification of a previously unreported CFTR mutation (c.

Quality Improvement Project to Evaluate Discharge Prescriptions in Children With Cystic Fibrosis.

Unlabelled: Cystic Fibrosis (CF) requires multiple pharmaceutical treatments, elevating the risk of medication errors (ME), which may compromise patient safety. This study aimed to improve the quality of discharge prescriptions (DPs) using indicators following admissions for IV antibiotics in pediatric CF patients.

Methods: This project involved a longitudinal observational retrospective descriptive study followed by a longitudinal quasi-experimental prospective phase between January 2013 and December 2016 in CF patients admitted to a London Children's Hospital.

Vitamin D for secondary prevention of acute wheeze attacks in preschool and school-age children.

Introduction: Vitamin D is best known for its role in bone health; however, the discovery of the vitamin D receptor and the expression of the gene encoding the vitamin D 1α-hydroxylase (CYP27B1) enzyme in a wide variety of tissues including immune cells and respiratory epithelium has led to the discovery of potential roles for vitamin D in the prevention of acute wheeze.

Methods: We review here the literature concerning the relationships between circulating 25-hydroxyvitamin D (25(OH)D) concentration and secondary prevention of acute wheeze attacks in preschool and school-age children.

Results: Epidemiological data suggest that vitamin D insufficiency (25(OH)D <75 nmol/L) is highly prevalent in preschool and school-age children with wheeze.

[Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation].

Introduction: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has been shown to improve the nutritional status and lung function of cystic fibrosis patients with the G551D mutation in clinical trials. The objective of this study was to describe the real-world progress of children receiving ivacaftor.

Methods: We describe the real-world progress of four children with cystic fibrosis and the F508del/G551D genotype comparing data during ivacaftor treatment with baseline and with the year before commencing treatment.

Preschool wheeze, genes and treatment.

Preschool wheeze is a common but poorly understood cause of respiratory morbidity that is both distinct from and overlaps with infantile bronchiolitis and school age asthma. Attempts at classification by epidemiology, pathophysiology, therapeutic response and clinical phenotype are imperfect and yet fundamental to both treatment choice and research design. The four main therapeutic classes for preschool wheeze, namely beta agonists, anticholinergics, corticosteroids and leukotriene modifiers are employed with variable and often scanty evidence base, with evidence for a genetic influence on response variations.

Cystic fibrosis newborn screening: outcome of infants with normal sweat tests.

Newborn babies positively screened for cystic fibrosis (CF) (high serum immunoreactive trypsin (IRT) with DNA analysis) are referred for a diagnostic sweat test, which may be normal (sweat chloride <30 mmol/L). Unless two gene mutations are identified during Newborn screening (NBS), the babies are discharged from follow-up. We wished to check that none had subsequently developed symptoms suggestive of CF.

Highlights of the 28(th) North American Cystic Fibrosis Conference 2014.

This is a selection of papers presented at the 28(th) North American Cystic Fibrosis Conference held in Atlanta in October 2014. The papers discussed are thought to be of particular interest to CF caregivers in the UK. Topics discussed include recent progress in the modification of the cystic fibrosis transmembrane regulator (CFTR), the potential of OligoG, a novel inhaled alginate mucolytic, and the changing approach to cystic fibrosis-related diabetes screening.

Intermittent montelukast in children aged 10 months to 5 years with wheeze (WAIT trial): a multicentre, randomised, placebo-controlled trial.

Background: The effectiveness of intermittent montelukast for wheeze in young children is unclear. We aimed to assess whether intermittent montelukast is better than placebo for treatment of wheeze in this age group. Because copy numbers of the Sp1-binding motif in the arachidonate 5-lipoxygenase (ALOX5) gene promoter (either 5/5, 5/x, or x/x, where x does not equal 5) modifies response to montelukast in adults, we stratified by this genotype.

Recruiting ethnic minority participants to a clinical trial: a qualitative study.

Objectives: To compare the motives and experiences of different ethnic groups participating in a randomised double blind placebo-controlled trial of montelukast in preschool wheeze, and to assess parents' or guardians' understanding of trial procedures and their implications, including the collection of genetic material.

Design: Qualitative interviews with parents or guardians.

Setting: Interviews occurred in the homes of London children recruited to a national multicentre clinical trial following primary and secondary care attendance with wheeze.

Cycling to work in London and inhaled dose of black carbon.

Modelling studies suggest that urban cycling is associated with an increased inhaled dose of fossil fuel-derived black carbon (BC). Using the amount of black material in airway macrophages as a marker of long-term inhaled BC, we sought to compare inhaled BC dose in London (UK) cyclists and non-cyclists. Airway macrophage carbon was assessed in 28 (58%) out of 48 healthy adults (14 cyclists and 14 non-cyclists) who attended for induced sputum.