• Drucy Borowitz, Barry Lubarsky, Michael Wilschanski, Anne Munck, Daniel Gelfond, Frank Bodewes, and Sarah Jane Schwarzenberg.
• Department of Pediatrics, University at Buffalo, State University of New York, Women and Children's Hospital of Buffalo, 219 Bryant Street, Buffalo, NY, 14222, USA. dborowitz@upa.chob.edu.
• Dig. Dis. Sci. 2016 Jan 1; 61 (1): 198-207.

BackgroundThe cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability.AimTo further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged ≥6 years with CF and the G551D mutation.MethodsPatients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks. Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R).ResultsStudies included 213 patients (aged ≤ 20 years, n = 105; aged > 20 years, n = 108). In patients ≤20 years, adjusted mean change from baseline to week 48 in body weight was 4.9 versus 2.2 kg (ivacaftor vs. placebo, p = 0.0008). At week 48, change from baseline in mean weight-for-age z-score was 0.29 versus -0.06 (p < 0.0001); change in mean BMI-for-age z-score was 0.26 versus -0.13 (p < 0.0001). 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.ConclusionsNutritional status improved following treatment with ivacaftor for 48 weeks.

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