Background
Elexacaftor-Tezacaftor-Ivacaftor (ETI), has led to significant improvements in lung function for CF patients (Middleton PG et al. NEJM 2019). A study estimated that a delay starting ETI could have an impact on long-term survival (Stanojevic S. JCF 2020). ETI use has been approved for children aged >6 years fulfilling the genetic criteria. There is still controversy regarding ETI use for adult patients with preserved lung function [defined as a percentage of predicted forced expiratory volume in the first second (ppFEV1)>90%].
Aims and objectives
To assess whether adult CF patients with ppFEV1>90% and bronchiectasis in the chest CT could benefit from ETI.
Methods
We retrospectively evaluated adult CF patients followed at the Lausanne University Hospital with prescribed ETI with bronchiectasis and a ppFEV1>90%.
Results
5 patients matched our criteria; all Caucasian males (age: 21-37 years), 4 Phe508del homozygous, 1 had a Phe508del/N1303K genotype. After ETI start, all reported a rapid improvement of respiratory symptoms; ppFEV1 increased at 3 and 6 months after ETI start compared to baseline (Figure). No adverse effects were noted.
Conclusions
Our observations support the use of ETI in adult CF patients with an eligible genotype and structural lung disease irrespective of their lung function. A ppFEV1 > 90% should not be a barrier to ETI prescription.
Figure:Evolution of ppFEV1 over time