Real-world efficacy, safety and heterogenicity of efficacy of elexacaftor-tezacaftor-ivacaftor in younger children (6-11 years old) with cystic fibrosis.

doi:10.1016/j.rmed.2026.109063

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Article

Real-world efficacy, safety and heterogenicity of efficacy of elexacaftor-tezacaftor-ivacaftor in younger children (6-11 years old) with cystic fibrosis.

Respiratory medicine - 1 Oct 2026

Sheikh Shahid, Holtzlander Melissa, Eisner Mariah, Gelpi Courtney, Palacios Sabrina, Mull Eric, Imran Sehyr, Kotha Kavitha, McCoy Karen S

Abstract excerpt

BACKGROUND: Heterogenicity of efficacy of elexacaftor-tezacaftor-ivacaftor (ETI) therapy in the real world in younger children (6-11 years old) is not known. METHODS: This is a longitudinal observational study. Clinical data at baseline and at one-year of therapy were compared for the total cohort and for following subgroups: genotype (homozygous vs. heterozygous for F508del), severity of lung disease at ETI...

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Topics

Humans
Cystic Fibrosis
Child
Quinolones
Benzodioxoles
Aminophenols

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Male

Female

Cystic Fibrosis Transmembrane Conductance Regulator

Drug Combinations

Indoles

Treatment Outcome

Longitudinal Studies

Genotype

Pyrrolidines

Treatment Effect Heterogeneity

Forced Expiratory Volume

Pyrazoles

Source

Open in PubMed

Identifiers

PMID: 42537817
DOI: 10.1016/j.rmed.2026.109063

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 23 Oct 2026.

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