Females with Cystic Fibrosis Demonstrate a Differential Response Profile to Ivacaftor Compared with Males
نویسندگان
چکیده
منابع مشابه
Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
BACKGROUND AND OBJECTIVES Cystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes. METHODS In this post hoc analysis, we assessed linear growth and weight in 83 childre...
متن کاملComputed tomography correlates with improvement with ivacaftor in cystic fibrosis patients with G551D mutation.
BACKGROUND Ivacaftor corrects the cystic fibrosis transmembrane conductance regulator (CFTR) gating defect associated with G551D mutation and is quickly becoming an important treatment in patients with cystic fibrosis (CF) due to this genetic mutation. METHODS A single-center study was performed in CF patients receiving ivacaftor to evaluate the usefulness of high resolution computed tomograp...
متن کاملPseudomonas aeruginosa in cystic fibrosis patients with G551D-CFTR treated with ivacaftor.
BACKGROUND Ivacaftor improves outcomes in cystic fibrosis (CF) patients with the G551D mutation; however, effects on respiratory microbiology are largely unknown. This study examines changes in CF respiratory pathogens with ivacaftor and correlates them with baseline characteristics and clinical response. METHODS The G551D Observational Study enrolled a longitudinal observational cohort of US...
متن کاملIvacaftor and sinonasal pathology in a cystic fibrosis patient with genotype delta F508/S1215N
In patients with Cystic Fibrosis and a type III mutation, ivacaftor (Kalydeco(®), Vertex) can increase the opening time of the CFTR channel and improve chloride transport. Research showed significant improvement of lung function and increase in weight following ivacaftor use. However, ivacaftor showed to have adverse events on the sinonasal system as well, such as upper respiratory tract infect...
متن کاملLumacaftor-ivacaftor (Orkambi) for cystic fibrosis: behind the 'breakthrough'.
Lumacaftor-ivacaftor (Orkambi) was recently approved by the Food and Drug Administration (FDA) in the USA to treat patients at least 12 years old who have cystic fibrosis due to two copies of the F508del (Phe508del) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Lumacaftor-ivacaftor has been designated a ‘breakthrough’ and is slated to cost $259 000 per year pe...
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ژورنال
عنوان ژورنال: American Journal of Respiratory and Critical Care Medicine
سال: 2020
ISSN: 1073-449X,1535-4970
DOI: 10.1164/rccm.201909-1845le