The effects of ivacaftor on bone density and microarchitecture in children and adults with cystic fibrosis

Context: Cystic fibrosis 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 planning to start or had started treatment with ivacaftor within three months (Ivacaftor cohort); 26 subjects with CF were not treated with ivacaftor (CF Control cohort); and 26 healthy volunteers.

Interventions: All treatments...