Oxygen saturation targets in infants with bronchiolitis (BIDS): a double-blind, randomised, equivalence trial.

Background: The American Academy of Pediatrics recommends a permissive hypoxaemic target for an oxygen saturation of 90% for children with bronchiolitis, which is consistent with the WHO recommendations for targets in children with lower respiratory tract infections. No evidence exists to support this threshold. We aimed to assess whether the 90% or higher target for management of oxygen supplementation was equivalent to a normoxic 94% or higher target for infants admitted to hospital with viral bronchiolitis.

An observational study of matrix metalloproteinase (MMP)-9 in cystic fibrosis.

Background: In cystic fibrosis (CF), cross-sectional studies have reported sputum matrix metalloproteinase (MMP)-9 to be elevated and negatively correlated with FEV1. This longitudinal study examined the association between MMP-9 and tissue inhibitors of metalloproteinases (TIMPs) to prognostic parameters in CF.

Method: A cross-sectional survey of CF and control subjects; CF patients were followed up for a median of 49 months.

A methodology to establish a database to study gene environment interactions for childhood asthma.

Background: Gene-environment interactions are likely to explain some of the heterogeneity in childhood asthma. Here, we describe the methodology and experiences in establishing a database for childhood asthma designed to study gene-environment interactions (PAGES--Paediatric Asthma Gene Environment Study).

Methods: Children with asthma and under the care of a respiratory paediatrician are being recruited from 15 hospitals between 2008 and 2011.

Comparative demographics of the European cystic fibrosis population: a cross-sectional database analysis.

Background: Country-specific patients' registries are rarely used to make international comparisons because of protocol discrepancies in data collation. We present data from a European cystic fibrosis registry that is dedicated to collection of demographic data, and assess whether the resources available in countries with and without European Union (EU) membership affects care and survival of patients.

Methods: Data for demographic indicators-age, age at diagnosis, sex, and genotype-for patients with cystic fibrosis from 35 European countries were combined, and used to establish the differences in demographic indicators between EU and non-EU countries.

Economic implications of newborn screening for cystic fibrosis: a cost of illness retrospective cohort study.

Background: Newborn screening for cystic fibrosis might not be introduced if implementation and running costs are perceived as prohibitive. Compared with clinical diagnosis, newborn screening is associated with clinical benefit and reduced treatment needs. We estimate the potential savings in treatment costs attributable to newborn screening.

Cystic fibrosis diagnosed after 2 months of age leads to worse outcomes and requires more therapy.

Objective: Newborn screening for cystic fibrosis remains controversial because improved pulmonary function has not been established. Studies to date have not accounted for differences in treatments delivered to clinically diagnosed children and newborn-screened controls. Here, we compare outcomes and treatment of patients clinically diagnosed within the newborn-screening reporting window (early-clinically diagnosed), those presenting after this period (late-clinically diagnosed), and patients diagnosed by newborn screening.

Delayed diagnosis of females with respiratory presentation of cystic fibrosis did not segregate with poorer clinical outcome.

Objective: Does a delay in diagnosis exist in females with cystic fibrosis (CF) presenting with respiratory symptoms alone. Does it segregate with poorer clinical outcomes?

Study Design And Setting: A set of 3,851 patients registered with the UK CF Database (diagnosed 1986-2003) were divided into four mutually exclusive categories by mode of presentation: meconium ileus or distal intestinal obstruction syndrome (MI/DIOS); positive family history; newborn screening; and symptoms excluding MI/DIOS. The last symptom category was subdivided to create a group for respiratory symptoms alone.

Neonatal screening for cystic fibrosis is beneficial even in the context of modern treatment.

Objectives: To determine whether early identification of babies with cystic fibrosis (CF) improves outcome in the current environment of new improved treatments, considering the criticism that there may be only marginal benefit gained by CF newborn screening (NBS).

Study Design: We tested whether CF NBS in the setting of modern CF center care still afforded benefit using the UK CF Database (UKCFD; ) to compare clinical outcomes in infants who underwent NBS and control subjects who were clinically diagnosed (CD). With Mann-Whitney rank tests, 184 patients who underwent NBS aged 1 to 9 years in 2002 (excluding meconium ileus) were compared with matched patients who were CD in 3-year age groups (950 control subjects).

Newborn screening for cystic fibrosis is associated with reduced treatment intensity.

Objectives: To determine whether the improved clinical status after newborn screening (NBS) for cystic fibrosis (CF) segregates with increased therapeutic intervention compared with presentation by clinical diagnosis (CD).

Study Design: In 2002, two populations (1 to 9 years of age) who presented (excluding meconium ileus) by NBS < or = 3 months of age or by CD were compared in an observational, cross-sectional design. NBS and CD populations (184 and 950 patients, respectively) were divided into 3-year age groups (1 to 3, 4 to 6, and 7 to 9 years).

Comparative analysis of Cystic Fibrosis Registry data from the UK with USA, France and Australasia.

Background: Using the UK Cystic Fibrosis Database, we analysed the health of the UK CF paediatric population (UKPP) in terms of their biographical, clinical and infection status and compared outcomes with the US, French and Australasian CF Registries.

Methods: UKPP data were collected for 2,673 patients aged less than 18 years in 2001 and used as a reference base for comparison with the most recent equivalent CF Registry reports.

Results: Although differences exist between National CF Registries, all record similar demographic factors and key outcomes.

Asians with cystic fibrosis in the UK have worse disease outcomes than clinic matched white homozygous delta F508 controls.

Background: We tested the hypothesis that the Asian cystic fibrosis (CF) phenotype is comparable to the commonest genetic form of CF found in 50% of the white UK CF population using the UK CF Database, a national disease-specific patient registry.

Methods: 50 Asian CF patients were matched by Centre with 143 white homozygous delta F508 patients for gender, age and chronic Pseudomonas aeruginosa status (a marker of morbidity). The authors compared FEV1 and FVC% predicted, mean height, weight and BMI Z scores.

Demographics of the UK cystic fibrosis population: implications for neonatal screening.

The objective was to determine the composition of the Cystic Fibrosis (CF) Population attending specialist UK CF centres in terms of age, gender, age at diagnosis, genotype and ethnicity. With the planned introduction of the national CF screening programme in the UK, cystic fibrosis transmembrane regulator (CFTR) mutations were compared between different ethnic groups enabling a UK-specific frequency of mutations to be defined. Data were analysed from the patient biographies held in the UK CF Database (see www.

Readmission with respiratory syncytial virus (RSV) infection among graduates from a neonatal intensive care unit.

We evaluated the incidence of readmission with respiratory syncytial virus (RSV) infection among the graduates of a regional Neonatal Intensive Care Unit (NICU), and characterized those who were rehospitalized. These data were used as a predictive tool to estimate the number of babies likely to suffer readmission with RSV for the year 2000 cohort. Using the published efficacies of palivizumab, the costs and benefits of protecting this cohort were assessed.