Study design (if review, criteria of inclusion for studies)
Single-center, retrospective study
Participants
The study included 174 CF patients on elexacaftor/tezacaftor/ivacaftor (ETI), six years and older with at least one copy of F508del
Interventions
De-escalating cystic fibrosis
Outcome measures
The primary objective was to assess non-inferiority of supportive therapies de-escalation by comparing the absolute change in percent predicted (ppFEV(1)) from baseline to month 1 versus the absolute change from baseline to month 12 after initiating ETI with patients serving as their own control.
Main results
The study included 174 patients. The mean ppFEV(1) at baseline, month 1, and month 12 was 67%, 78%, and 87% respectively. The mean difference in absolute change in ppFEV(1) from baseline to month 1 compared to baseline to month 12 after the initiation of ETI was 1.53% (95% CI: -0.49 to 3.55)
Authors' conclusions
De-escalating supportive therapies for those on ETI was non-inferior to remaining on all supportive therapies. This suggests that medications may be able to be discontinued under the context of a de-escalation algorithm, which may decrease medication burden and cost and increase quality of life.