Body composition and weight changes after ivacaftor treatment in adults with cystic fibrosis carrying the G551 D cystic fibrosis transmembrane conductance regulator mutation: A double-blind, placebo-controlled, randomized, crossover study with open-label extension

King, Serina; Tierney, Audrey C.; Edgeworth, D.; Keating, D.; Williams, E.; Kotsimbos, Tom; Button, Brenda; Wilson, John W.

doi:10.1016/j.nut.2020.111124

Cited by 34 publications

(29 citation statements)

References 36 publications

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“…CFTR is expressed within skeletal muscle tissue 25 and CFTR activity might be involve in regulating mitochondrial function. 28 As reported in other studies, 12,14,16,29 BMI increased in all three of our adolescents. Understanding the pattern of weight gain after modulator treatment is an important area of research.…”

Section: Discussionsupporting

confidence: 86%

Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis

Causer

Shute

Cummings

et al. 2022

Pediatric Pulmonology

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Methods: Three adolescents (14.0 ± 1.4 years) with CF (FEV 1 % predicted: 62.5 ± 17.1; F508del/F508del genotype) completed an exhaustive maximal cardiopulmonary exercise test on a cycle ergometer to determine peak oxygen uptake ( VO 2peak ) and measure changes in gas exchange and ventilation during exercise at 6 weeks. We also analyzed wrist-worn device-based physical activity (PA) data in two of the three cases. Validated acceleration thresholds were used to quantify time spent in each PA intensity category.Results: Clinically meaningful improvements in VO 2peak were observed in all three cases (+17.6%, +52.4%, and +32.9%, respectively), with improvements greatest in those with more severe lung disease and lower fitness at baseline. Although lung function increased in all cases, inconsistent changes in markers of ventilatory and peripheral muscle efficiency likely suggest different mechanisms of improvement in this case group of adolescents with CF. Device-based analysis of PA was variable, with one case increasing and one case decreasing. Conclusion:In this case series, we have observed, for the first time, improvements in exercise capacity following 6 weeks of treatment with Elexacaftor/Tezacaftor/ Ivacaftor. Improvements were greatest in the presence of more severe CF lung disease and lower aerobic fitness at baseline. The mechanism(s) responsible for these changes warrant further investigation in larger trials.

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Section: Discussionsupporting

confidence: 86%

Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis

Causer

Shute

Cummings

et al. 2022

Pediatric Pulmonology

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“…Several groups have described an increased prevalence of normal weight obesity (NWO) and decreased fat-free mass distribution (FFMD) in individuals with CF, as well as a link between this type of body habitus and poorer lung function [20,24,25]. There may exist specific DXA body composition variables that better predict long-term CF-specific clinical outcomes than BMI [26]. If identified, these variables could predict the risk of pulmonary decline and metabolic abnormalities in patients with CF, and help guide individualized advice regarding dietary composition and physical activity.…”

Section: Introductionmentioning

confidence: 99%

The Relationship between Body Composition, Dietary Intake, Physical Activity, and Pulmonary Status in Adolescents and Adults with Cystic Fibrosis

et al. 2022

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Measures of body fat and lean mass may better predict important clinical outcomes in patients with cystic fibrosis (CF) than body mass index (BMI). Little is known about how diet quality and exercise may impact body composition in these patients. Dual X-ray absorptiometry (DXA) body composition, 24-h dietary recall, and physical activity were assessed in a cross-sectional analysis of 38 adolescents and adults with CF and 19 age-, race-, and gender-matched healthy volunteers. Compared with the healthy volunteers, participants with CF had a lower appendicular lean mass index (ALMI), despite no observed difference in BMI, and their diets consisted of higher glycemic index foods with a greater proportion of calories from fat and a lower proportion of calories from protein. In participants with CF, pulmonary function positively correlated with measures of lean mass, particularly ALMI, and negatively correlated with multiple measures of body fat after controlling for age, gender, and BMI. Higher physical activity levels were associated with greater ALMI and lower body fat. In conclusion, body composition measures, particularly ALMI, may better predict key clinical outcomes in individuals with CF than BMI. Future longitudinal studies analyzing the effect of dietary intake and exercise on body composition and CF-specific clinical outcomes are needed.

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“…In a long-term open label extension study of ivacaftor, King et al found that both weight and fat mass significantly increased after 6 months of treatment and, at two years on the drug, 64% of weight gained was fat mass. Additionally, 25% of participants were classified as overweight and 10% were obese by the end of the two-year study [ 58 ]. More studies of longer duration are needed to examine body composition and fat distribution changes on CFTR modulators in the setting of unintended weight gain and the development of overweight and obesity on these drugs.…”

Section: Overweight and Obesity In Cystic Fibrosismentioning

confidence: 99%

The Changing Landscape of Nutrition in Cystic Fibrosis: The Emergence of Overweight and Obesity

2022

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Cystic fibrosis has historically been characterized by malnutrition, and nutrition strategies have placed emphasis on weight gain due to its association with better pulmonary outcomes. As treatment for this disease has significantly improved, longevity has increased and overweight and obesity have emerged issues in this population. The effect of excess weight and adiposity on CF clinical outcomes is unknown but may produce similar health consequences and obesity-related diseases as those observed in the general population. This review examines the prevalence of overweight and obesity in CF, the medical and psychological impact, as well as the existing evidence for treatment in the general population and how this may be applied to people with CF. Clinicians should partner with individuals with CF and their families to provide a personalized, interdisciplinary approach that includes dietary modification, physical activity, and behavioral intervention. Additional research is needed to identify the optimal strategies for preventing and addressing overweight and obesity in CF.

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Body composition and weight changes after ivacaftor treatment in adults with cystic fibrosis carrying the G551 D cystic fibrosis transmembrane conductance regulator mutation: A double-blind, placebo-controlled, randomized, crossover study with open-label extension

Cited by 34 publications

References 36 publications

Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis

Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis

The Relationship between Body Composition, Dietary Intake, Physical Activity, and Pulmonary Status in Adolescents and Adults with Cystic Fibrosis

The Changing Landscape of Nutrition in Cystic Fibrosis: The Emergence of Overweight and Obesity

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