Real-world population pharmacokinetics of tezacaftor-ivacaftor in children with cystic fibrosis: The SYM-CF study.

ABSTRACT:

Aims

The clinical effectiveness of tezacaftor-ivacaftor in children with cystic fibrosis (cwCF) varies; some patients respond while others do not or have adverse effects. The pharmacokinetics (PK) of tezacaftor-ivacaftor are inadequately published, especially in children. Knowledge of the PK in this cohort in relation to clinical outcomes may give further insight into the drug's exposure-response relationship and its associated interindividual variability. The aim of this study was to assess the real-world PK of tezacaftor-ivacaftor in cwCF.

Methods

A prospective, observational PK study was performed in cwCF using tezacaftor-ivacaftor. PK samples were obtained by dried blood spots at home and during routine outpatient hospital visits. Population PK (popPK) models were create

SUBMITTER: Vonk SEM 

PROVIDER: S-EPMC12464662 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature