Cystic fibrosis is the most frequent and lethal inherited disease, affecting populations of European and Caucasian origin. Pneumothorax is life threatening pulmonary complication of cystic fibrosis. Bilateral pneumothorax is rarely seen and is a predictor of poor prognosis. We report a newborn presenting with bilateral pneumothorax whose diagnosis was cystic fibrosis with N1303K mutation on CFTR gene.
Download full-text PDF |
Source |
---|
Epidemiology
December 2024
Department of Public Health, Policy and Systems, University of Liverpool, Liverpool, UK.
Background: Children with cystic fibrosis (CF) from socioeconomically deprived areas have poorer growth, worse lung function, and shorter life expectancy than their less-deprived peers. While early growth is associated with lung function around age 6, it is unclear whether improving early growth in the most deprived children reduces inequalities in lung function.
Methods: We used data from the UK CF Registry, tracking children born 2000-2010 up to 2016.
Unlabelled: Neonatal screening has been implemented internationally with different protocols and has become the routine method in the preclinical stage. Late diagnosis is associated with more severe symptoms with decreased survival and higher treatment costs.
Objectives: To estimate the incidence of cystic fibrosis; to evaluate the performance of the screening algorithms Immunoreactive Trypsinogen and Pancreatitis-Associated Protein (IRT/PAP) and the IRTxPAP product; to analyze the cut-off value for IRT, PAP, and IRTxPAP, and to establish a methodology for its ongoing evaluation; finally, to evaluate the quality of IRT and PAP measurements.
Pediatr Pulmonol
January 2025
Pulmonary and Sleep Medicine, DeVos Children's Hospital, Grand Rapids, Michigan, USA.
Objective: We prospectively monitored rates of change for growth, body mass and composition, muscle strength, and FEV1 in 6-11-year-olds initiating ETI therapy, comparing them to those of US reference children. We assessed factors potentially contributing to rate of change and report ranges of individual variation.
Methods: Body composition was assessed using bioelectrical impedance analysis (BIA), and rates of change were analyzed using linear mixed effects regression models.
J Pharm Anal
December 2024
Institute of Infectious Disease and Infection Control, Jena University Hospital, Jena, 07747, Germany.
In our prior research, polymer nanoparticles (NPs) containing tobramycin displayed robust antibacterial efficacy against biofilm-embedded () and (. ) cells, critical pathogens in cystic fibrosis. In the current study, we investigated the deposition of a nanoparticulate carrier composed of poly(d,l-lactic--glycolic acid) (PLGA) and poly(ethylene glycol)--PLGA (PEG-PLGA) that was either covalently bonded with cyanine-5-amine (Cy5) or noncovalently bound with freely embedded cationic rhodamine B (RhB), which served as a drug surrogate.
View Article and Find Full Text PDFLancet Respir Med
December 2024
Population Policy and Practice Department, UCL Great Ormond Street Institute of Child Health, University College London, London, UK; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. Electronic address:
Background: In phase 2 trials in people with cystic fibrosis aged 18 years and older, vanzacaftor-tezacaftor-deutivacaftor has been shown to be a safe and effective, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator. Restoring normal CFTR function early in life has the potential to prevent manifestations of cystic fibrosis. We aimed to evaluate the safety, tolerability, efficacy, and pharmacokinetics of vanzacaftor-tezacaftor-deutivacaftor in children with cystic fibrosis aged 6-11 years.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!