Open AccessREAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report
Author(s) -
Fuchs Teresa,
Appelt Dorothea,
Niedermayr Katharina,
Ellemunter Helmut
Publication year - 2022
Publication title -
clinical case reports
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.21
H-Index - 9
ISSN - 2050-0904
DOI - 10.1002/ccr3.5364
Subject(s) - ivacaftor , cystic fibrosis , medicine , newborn screening , sweat , cystic fibrosis transmembrane conductance regulator , pediatrics , gastroenterology
This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible.