Format

Send to

Choose Destination
J Cyst Fibros. 2018 Mar;17(2):e13-e16. doi: 10.1016/j.jcf.2017.09.006. Epub 2017 Oct 24.

Ivacaftor withdrawal syndrome in cystic fibrosis patients with the G551D mutation.

Author information

1
Division of Pulmonary & Critical Care Medicine, Division of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC, United States. Electronic address: atrimble@med.unc.edu.
2
Division of Pulmonary & Critical Care Medicine, Division of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC, United States.

Abstract

Ivacaftor use can lead to dramatic health improvements in cystic fibrosis (CF) patients with gating mutations. Here, we report five instances of dramatic clinical decline following withdrawal of ivacaftor in three individuals with the G551D-CFTR mutation. In each case, the patient's lung function and symptoms rapidly deteriorated after cessation of treatment. Awareness of this phenomenon should inform both clinical practices as well as the design of future clinical trials of highly active CFTR modulators.

KEYWORDS:

Acute pulmonary exacerbation; CFTR modulator; Cystic fibrosis; G551D; Ivacaftor

PMID:
29079142
DOI:
10.1016/j.jcf.2017.09.006

Supplemental Content

Full text links

Icon for Elsevier Science
Loading ...
Support Center