The Effects of Ivacaftor on Bone Density and Microarchitecture in Children and Adults with Cystic Fibrosis.

ABSTRACT:

Context

Cystic fibrosis (CF) transmembrane conductance (CFTR) dysfunction may play a role in CF-related bone disease (CFBD). Ivacaftor is a CFTR potentiator effective in improving pulmonary and nutritional outcomes in patients with the G551D-CFTR mutation. The effects of ivacaftor on bone health are unknown.

Objective

To determine the impact of ivacaftor on bone density and microarchitecture in children and adults with CF.

Design

Prospective observational multiple cohort study.

Setting

Outpatient clinical research center within a tertiary academic medical center.

Patients or other participants

Three cohorts of age-, race-, and gender-matched subjects were enrolled: 26 subjects (15 adults and 11 children) with CF and the G551D-CFTR mutation who were plann

SUBMITTER: Putman MS 

PROVIDER: S-EPMC7947772 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature