Identification of early changes in multiple biomarkers following CFTR modulator initiation in patients with cystic fibrosis.

ABSTRACT:

Background

There are currently no early parameters that allow prediction of long-term responses to Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator treatment on an individual level.

Objectives

To identify early parameters measured within 7 to 14 days after initiation of treatment with a CFTR modulator to assess CFTR modulator efficacy.

Study design

Prospective observational study of patients diagnosed with CF who begin elexacaftor/tezacaftor/ivacaftor (ETI) therapy at 3 CF clinics in Switzerland (Geneva, Lausanne, Lucerne).

Methods

Standardized measurements were taken within 2 months prior to and 7 to 14 days after starting CFTR modulator treatment.

Results

ETI treatment was started on 47 patients [median age: 12 years] of whom 12 (26

SUBMITTER: Heer P 

PROVIDER: S-EPMC12515279 | biostudies-literature | 2025 Jan-Dec

REPOSITORIES: biostudies-literature