• Susannah J King, Audrey C Tierney, Deirdre Edgeworth, Dominic Keating, Elyssa Williams, Tom Kotsimbos, Brenda M Button, and John W Wilson.
• Nutrition Department, Alfred Hospital, Melbourne, Victoria, Australia; Cystic Fibrosis Service, Department of Respiratory Medicine, Alfred Hospital, Melbourne, Victoria, Australia; Department of Dietetics, Nutrition and Sport, LaTrobe University, Victoria, Australia. Electronic address: s.king@alfred.org.au.
• Nutrition. 2021 May 1; 85: 111124.

ObjectivesIn patients with cystic fibrosis (CF) who carry the G551D mutation, treatment with ivacaftor improves lung function and weight; however, short- and long-term impacts on body composition have not been well studied.MethodsTwenty adults with CF carrying the G551D mutation (mean ± standard deviation body mass index [BMI] 23.3 ± 4.3 kg/m2) were recruited for a single-center, double-blind, placebo-controlled, 28-d, crossover study of ivacaftor, followed by an open-label extension (OLE) for 5 mo. Eleven patients underwent measurements 2 y later. The study variables included weight, BMI, and body composition (including fat-free mass [FFM] and fat mass).ResultsAfter 28 d of treatment with ivacaftor, weight increased by 1.1 ± 1.3 kg, BMI by 0.4 ± 0.5 kg/m2, and FFM by 1.1 ± 1.2 kg (all P < .005) with no change in fat mass. Differences between 28-d changes on ivacaftor and placebo were not statistically significant. In the following 5 mo of the OLE, there were significant increases in weight (1.2 ± 1.9 kg; P < .05) and fat mass (1.5 ± 1.9 kg; P < .01), but not in FFM. Between baseline and the end of the OLE, the total weight gain was 2.5 ± 2.4 kg (P < .005), comprised of 0.9 ± 1.5 kg FFM (P < .05) and 1.6 ± 1.8 kg fat mass (P < .005). For the 11 participants who were followed for a further 2 y, no further changes in mean weight, BMI, or body composition parameters between 6 mo and 2 y later were observed.ConclusionsSmall gains were seen in FFM in the first month of ivacaftor treatment. Weight, BMI, and fat-mass gains in the first 6 mo on ivacaftor plateaued by 2.5 y. The metabolic and clinical consequences of weight and fat-mass gains remain to be determined.Copyright © 2020 Elsevier Inc. All rights reserved.

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