Nutritional Impact of CFTR Modulators in Children with Cystic Fibrosis

Overview

Journal Front Pediatr

Specialty Pediatrics

Date 2023 Apr 17

PMID 37063653

Authors

Margaux Gaschignard

Fabien Beaufils

Florian Lussac-Sorton

Pauline Gallet

Haude Clouzeau

Joris Menard

Aurelie Costanzo

Lucie Nouard

Laurence Delhaes

Candice Tetard

Thierry Lamireau

Michael Fayon

Stephanie Bui

Raphael Enaud

Affiliations

Soon will be listed here.

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.

Citing Articles

Challenges to Optimizing Nutrition in Children With Cystic Fibrosis.

Chen C, Granneman J, Yadav S Curr Gastroenterol Rep. 2025; 27(1):20.

PMID: 40053205 DOI: 10.1007/s11894-025-00969-5.

Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data.

Imrei M, Keri A, Gacs E, Gonczi I, Melath M, Kosaras E Sci Rep. 2025; 15(1):2247.

PMID: 39824960 PMC: 11748628. DOI: 10.1038/s41598-025-86010-1.

Changes in vitamins and trace elements after initiation of highly effective CFTR modulator therapy in children and adults with cystic fibrosis - a real-life insight.

Fabricius D, Knieling T, Zurmuehl N, Makedon L, Freihorst J, Schmidt H Mol Cell Pediatr. 2024; 11(1):4.

PMID: 38717689 PMC: 11078909. DOI: 10.1186/s40348-024-00178-6.

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