Word, table, figure count
- Word count
- Manuscript body = 3767 (max. 4000 words)
- Abstract = 236 (max. 250 words)
- Tables = 4
- Figures = 4
Bullet point summary:
- What is already known about this subject :
- The clinical efficacy and safety of tezacaftor-ivacaftor in children
with cystic fibrosis is highly variable in clinical practice.
- Tezacaftor-ivacaftor has two fixed dosing regimens in children:
≥30kg receive the adult dose, and <30kg receive half of the
adult dose.
- A limited number of pharmacokinetic studies has been performed in
children; data are sparsely published. No population pharmacokinetic
data of tezacaftor-ivacaftor in children have been published so far.
- What this study adds :
- The first population pharmacokinetic models with real-world data
were developed for tezacaftor-ivacaftor and its active metabolites
using prior information from adolescents/adults.
- AUC of tezacaftor-ivacaftor varied greatly within and across age
groups; a strong correlation between Cmin and AUC
was observed.
- The developed population pharmacokinetic models for
tezacaftor-ivacaftor can be used in future studies evaluating the
exposure-response relationship and its variability as a basis for
more personalized dosing.