Elexacaftor/tezacaftor/ivacaftor is associated with long-term reduction in use of chronic respiratory therapies in cystic fibrosis.

BACKGROUND: Limited data exist on the real-world impact of elexacaftor/tezacaftor/ivacaftor (ETI) on use of chronic respiratory therapies. This post-hoc analysis evaluated long-term changes in use of chronic respiratory therapies following ETI initiation among people with (pw)CF aged ≥6 years in the United States and examined characteristics of those who were taking ≥2 chronic therapies at baseline and post-ETI.

METHODS: PROMISE is a prospective, multicenter, observational study. Self-reported use of inhaled antibiotics (cycled or continuous), hypertonic saline, dornase alfa, and oral azithromycin were captured before and through 54 months post-ETI initiation in 479 participants ≥12 years and through 36 months post-ETI in 124 participants aged 6-11 years.

RESULTS: ETI was associated with reductions in use of four chronic respiratory therapies through 54 months in participants ≥12 years (mean number of therapies decreasing from 2.6 [baseline] to 1.4 [54 months post-ETI]) and 36 months post-ETI in participants aged 6-11 years (mean number of therapies decreasing from 1.9 [baseline] to 1.3 [36 months post-ETI]). Consistent downward trends in use of all four therapies were observed among subgroups based on baseline age strata, sex at birth, prior CFTR modulator use, and ppFEV1. Mean improvements in ppFEV1 and mean changes in respiratory symptoms post-ETI did not differ between participants who remained on ≥2 therapies and participants taking <2 therapies.

CONCLUSIONS: ETI was associated with long-term reductions in use of chronic respiratory therapies (inhaled antibiotics, hypertonic saline, dornase alfa, azithromycin) among pwCF ≥6 years, indicating a sustained decrease in respiratory treatment burden among those on ETI therapy.