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. 2023 Mar 29:11:1130790.
doi: 10.3389/fped.2023.1130790. eCollection 2023.

Nutritional impact of CFTR modulators in children with cystic fibrosis

Margaux Gaschignard  1 , Fabien Beaufils  1   2 , Florian Lussac-Sorton  2 , Pauline Gallet  1 , Haude Clouzeau  1 , Joris Menard  1 , Aurélie Costanzo  1 , Lucie Nouard  1 , Laurence Delhaes  1   2 , Candice Tetard  1 , Thierry Lamireau  1 , Michael Fayon  1   2 , Stéphanie Bui  1   2 , Raphaël Enaud  1   2
Affiliations

Nutritional impact of CFTR modulators in children with cystic fibrosis

Margaux Gaschignard et al. Front Pediatr. .

Abstract

Background: Nutritional status is a major prognostic factor for breathing and the survival of patients with cystic fibrosis (CF). Since 2012, the development of CFTR modulators has considerably transformed the outcome of this disease. Indeed, both lung function and body mass index are improved by CFTR modulators, such as Lumacaftor/Ivacaftor. However, few data exist regarding the outcome of nutritional intakes under Lumacaftor/Ivacaftor.

Methods: We conducted a prospective single-center study in children with CF treated with Lumacaftor/Ivacaftor to evaluate their nutritional intake before and after treatment.

Results: Thirty-four children were included in this study, with a median age of 12.4 years [11.9; 14.7]. There was no significant improvement in weight, height or BMI. Patients' total energy intake was not significantly changed with Lumacaftor/Ivacaftor, while carbohydrate intakes decreased significantly. We found that blood levels of vitamin E and Selenium were significantly increased under Lumacaftor/Ivacaftor, without a significant increase in supplementation. In patients with a BMI Z-score < 0 at treatment initiation, there was a significant improvement in weight and BMI Z-score, while TEI and carbohydrate intakes were significantly lower.

Conclusion: We showed that treatment with Lumacaftor/Ivacaftor improved the nutritional status of patients without necessarily being associated with an increase in nutritional intake. Although these data need to be confirmed in larger cohorts, they support the hypothesis that weight gain under modulators is multifactorial, and may be related to a decrease in energy expenditure or an improvement in absorption.

Keywords: cystic fibrosis; ivacaftor; lumacaftor; macronutrient; nutrition; nutritional intake; selenium; vitamin E.

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Conflict of interest statement

MG, FLS, PG, HC, JM, AC, LN, CT and TL report no conflict of interest. FB has received a research grant from AstraZeneca and reports personal fees and non-financial support from Novartis, Chiesi and AstraZeneca. LD and RE are the co-coordinators of the French national cohort “Lum Iva Biota”, which received grants from Vertex. RE also reports a grant from Biocodex, personal fees from Biocodex and Menarini, and non-financial supports from Pfizer, Vertex, MSD, Nutricia, Nestle, Abbvie, Mayoly Spindler, Gilead sciences, Hospira and Aptalis Pharma. MF has received a research and educational grant from Novartis, and reports personal fees as a speaker from GlaxoSmithKine. In addition, MF has 1 co-patent (N° 176; FR 19/14751: i.e., New in situ pulmonary treatment device in premature babies). SB has received fees for expertise and boards for Zambon and Vertex.

Figures

Figure 1
Figure 1
Z-score changes in body mass index (A) and total energy intake (B) under lumacaftor/ivacaftor.
See this image and copyright information in PMC

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