Elexacaftor\/tezacaftor\/ivacaftor well tolerated in children aged 6 through 11 years with cystic fibrosis and at least one F508del-CFTR<\/em> allele<\/p>\n<\/h3>\n <\/b><\/p>\n <\/b><\/p>\n WEDNESDAY, April 14, 2021 (HealthDay News) — The safety and efficacy of elexacaftor\/tezacaftor\/ivacaftor (ELX\/TEZ\/IVA) for the treatment of cystic fibrosis with at least one F508del-CFTR <\/em>allele in children aged 6 to 11 years are consistent with the results reported for this combination in adolescents and adults, according to a study published online March 18 in the American Journal of Respiratory and Critical Care Medicine<\/em>.<\/p>\n Edith T. Zemanick, M.D., from the University of Colorado Anschutz Medical Campus and Children’s Hospital Colorado in Aurora, and colleagues examined the safety, pharmacokinetics, and efficacy of ELX\/TEZ\/IVA in children aged 6 to 11 years with F508del<\/em>-minimal function or F508del-F508del<\/em> genotypes in a 24-week, phase 3, open-label study. A total of 66 children received ELX\/TEZ\/IVA (50 percent of adult daily dose for those weighing <30 kg; full adult daily dose for those weighing \u00e2\u0089\u00a530 kg).<\/p>\n The researchers found that the safety and pharmacokinetic profiles were generally consistent with those seen in older patients. Cough, headache, and pyrexia were the most commonly reported adverse events (AEs); AEs were mostly mild or moderate in severity. ELX\/TEZ\/IVA treatment improved the percentage of predicted forced expiratory volume in one second, Cystic Fibrosis Questionnaire-Revised respiratory domain score, lung clearance index2.5<\/sub>, and sweat chloride concentration through week 24; compared with the pretreatment baseline, there was an increase in body mass index-for-age z-<\/em>score during the 24-week treatment period.<\/p>\n “The most exciting aspect of our findings is that this population of children had normal lung function at the start of the study and still had a significant improvement,” a coauthor said in a statement.<\/p>\n Several authors disclosed financial ties to Vertex Pharmaceuticals, which manufactures ELX\/TEZ\/IVA and funded the study.<\/p>\n Abstract\/Full Text (subscription or payment may be required)<\/a><\/p>\n <\/i><\/p>\n