Cystic Fibrosis News

The FDA has expanded use of elexacaftor/tezacaftor/ivacaftor for children aged 2 to 5 years with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.

Lumacaftor/ivacaftor showed various improvements in kids aged 2 to 5 years homozygous for F508del-CFTR over 48 weeks, according to study results published in Annals of the American Thoracic Society.

Pediatric patients with cystic fibrosis hospitalized with an exacerbation showed no lung function improvement with systemic corticosteroids, according to study results published in Annals of the American Thoracic Society.

Combination therapy with tezacaftor/ivacaftor was safe and well tolerated for up to 216 weeks in children aged 12 years or older, according to results of an extension study published in Journal of Cystic Fibrosis.

Elexacaftor/tezacaftor/ivacaftor appeared safe and well tolerated for up to 24 weeks in kids aged 2 to 5 years with one or more F508del allele, according to results published in American Journal of Respiratory and Critical Care Medicine.

Discontinuing hypertonic saline or dornase alfa for 6 weeks did not appear to have a negative effect on pulmonary function among patients with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor, according to study results.

Elexacaftor/tezacaftor/ivacaftor safely improved lung function and respiratory symptoms among children aged 6 to 11 years with the F508del/minimal function genotypes of cystic fibrosis, according to study results.

Healio Pulmonology has compiled the top news on cystic fibrosis posted in 2022.

Challenged urine bicarbonate excretion provided a simple and safe quantification of cystic fibrosis transmembrane conductance regulator function among adults with cystic fibrosis, according to data published in Annals of Internal Medicine.

Patients with cystic fibrosis who experience hemoptysis were found to be at higher risk for a lung transplant and death without a transplant, according to a study published in Annals of the American Thoracic Society.