Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.

ABSTRACT:

Background and objectives

Cystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes.

Methods

In this post hoc analysis, we assessed linear growth and weight in 83 children (aged 6-11 years) enrolled in 2 clinical trials, the longitudinal-observation GOAL study and the placebo-controlled ENVISION study, to evaluate the effects of ivacaftor, a CFTR potentiator. We calculated height and weight z scores and height and weight growth velocities (GVs).

Results

In ivacaftor-treated children in GOAL, height and weight z scores increased significantly from

SUBMITTER: Stalvey MS 

PROVIDER: S-EPMC5260152 | biostudies-literature | 2017 Feb

REPOSITORIES: biostudies-literature