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Evaluation of the effect of CFTR modulator therapy on lipid profiles in children

Ayyüce Ünlü1, Şule Selin Akyan2, Satı Özkan Tabakçı2

  • 1Department of Pediatric Pulmonology, Ankara Bilkent City Hospital, Ankara, Türkiye. ayyuce_aktemur@outlook.com.

Pediatric Research
|June 22, 2026
PubMed

Insights

Cystic fibrosis (CF) modulator therapy improved lung function and BMI in children. Importantly, these treatments did not cause significant lipid changes over six months, offering reassurance for pediatric patients.

Area of Science:

  • Pediatric Pulmonology
  • Cardiometabolic Health
  • Pharmacogenomics

Background:

  • Cystic fibrosis (CF) is a genetic disorder caused by CFTR gene mutations, leading to significant morbidity and reduced quality of life.
  • CFTR modulators have revolutionized CF care, improving pulmonary and nutritional status, but potential metabolic side effects like dyslipidemia require investigation, especially in children.
  • Limited pediatric data exist on the metabolic effects of CFTR modulators, necessitating focused research in this age group.

Purpose of the Study:

  • To evaluate the impact of CFTR modulator therapy on lipid and lipoprotein profiles in pediatric patients with CF.
  • To assess changes in Body Mass Index (BMI) Z-scores and lung function parameters following CFTR modulator initiation.
  • To determine if improvements in nutritional and pulmonary status correlate with alterations in lipid profiles.

Main Methods:

  • A retrospective study design was employed at a tertiary pediatric pulmonology center.
  • Data collected included baseline and six-month post-initiation BMI Z-scores, pulmonary function tests (FEV1, FVC), and serum lipid profiles (HDL, LDL, VLDL, triglycerides, total cholesterol).
  • Statistical comparisons were made between baseline and follow-up measurements to identify significant changes.

Main Results:

  • Twenty-six pediatric patients with CF were analyzed, with 80.8% receiving elexacaftor/tezacaftor/ivacaftor.
  • Significant improvements were observed in BMI Z-scores (p=0.047), FEV1 (p=0.005), and FVC Z-scores (p=0.001) after six months of therapy.
  • While numerical increases in HDL, LDL, VLDL, triglycerides, and total cholesterol were noted, these changes did not reach statistical significance. No correlation was found between BMI changes and lipid alterations.

Conclusions:

  • Six months of continuous CFTR modulator therapy in children with CF led to significant improvements in pulmonary function and nutritional status.
  • No evidence of clinically meaningful lipid deterioration was observed during the six-month treatment period.
  • These findings provide crucial real-world pediatric data, supporting the safety of CFTR modulators regarding lipid profiles while highlighting the need for continued cardiometabolic monitoring as patient survival improves.
Abstract

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