Publications by authors named "R D Szczesniak"
Article Synopsis
- PCD (Primary Ciliary Dyskinesia) and CF (Cystic Fibrosis) are both genetic diseases that affect how the lungs work and cause issues from a young age, but PCD is thought to be milder, even though evidence is limited.
- The study wanted to see how children with PCD compare to those with CF in terms of health over time, especially focusing on children with certain severe forms of PCD.
- They found that kids with PCD and the severe type had lower lung function than kids with CF, but their nutrition was similar, with more CF kids getting sick from bacteria in their lungs than PCD kids.
Background: Elexacaftor/tezacaftor/ivacaftor (ETI), which is approved for people with cystic fibrosis (pwCF) with a F508del variant, was further approved based on data in the USA for those carrying at least one of 177 rare (cystic fibrosis transmembrane conductance regulator) variants.
Methods: PwCF, aged ≥6 years, carrying no F508del variant but with at least one of these 177 rare variants, were identified within the US Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2020 and 2022. The evolution of forced expiratory volume in 1 s (FEV) percentage predicted and rates of pulmonary exacerbations were analysed over the first year following ETI initiation, using a linear regression with generalised estimating equations and a negative binomial model, respectively.
Introduction: Patients with cystic fibrosis (CF) experience frequent episodes of acute decline in lung function called pulmonary exacerbations (PEx). An existing clinical and place-based precision medicine algorithm that accurately predicts PEx could include racial and ethnic biases in clinical and geospatial training data, leading to unintentional exacerbation of health inequities.
Methods: We estimated receiver operating characteristic curves based on predictions from a nonstationary Gaussian stochastic process model for PEx within 3, 6, and 12 months among 26,392 individuals aged 6 years and above (2003-2017) from the US CF Foundation Patient Registry.
Article Synopsis
- Environmental factors and community characteristics impact lung function decline in cystic fibrosis (CF) patients, but comprehensive research on geomarkers as predictors is limited.
- A study examined the predictive potential of various geomarkers related to lung function decline and pulmonary exacerbations using data from 151 CF patients aged 6-20 over a decade.
- Four Bayesian regression models identified key geomarkers (like air temperature and socioeconomic indicators) that could predict lung function decline, with the elastic-net model showing the highest accuracy and sensitivity for predicting pulmonary exacerbations.
Background: We characterized people with cystic fibrosis (CF) ineligible by genotype (not age) for currently approved CFTR modulator therapy using data from the US CF Foundation Patient Registry (CFFPR).
Methods: We summarized clinical characteristics using CFFPR data from 2017 to 2022. Annual rate of change in percent predicted of forced expiratory volume in one second (ppFEV) was estimated using generalized estimating equations.