Study design (if review, criteria of inclusion for studies)
RCT
List of included studies
x
Participants
patients aged >6 years with CF and the G551D mutation.
Interventions
Patients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks.
Outcome measures
Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R).
Main results
Studies included 213 patients (aged 20 years, n = 108). In patients 20 years, adjusted mean change from baseline to week 48 in body weight was 2.7 versus -0.2 kg (p = 0.0003). Mean BMI change at week 48 was 0.9 versus -0.1 kg/m(2) (p = 0.0003). There was no linear correlation evident between changes in body weight and improvements in lung function or sweat chloride. Significant CFQ-R improvements were seen in perception of eating, body image, and sense of ability to gain weight.
Authors' conclusions
Nutritional status improved following treatment with ivacaftor for 48 weeks.