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Effects on growth, weight and body composition after CFTR modulators in children with cystic fibrosis

C M López Cárdenes1,2,3, A Merino Sánchez-Cañete1,2, S Vicente Santamaría1,2,3

  • 1Unidad de Fibrosis Quística, Servicio Pediatría, Hospital Universitario Ramón y Cajal, Madrid, Spain.

Pediatric Pulmonology
|September 24, 2024
PubMed

Insights

Cystic Fibrosis patients showed improved weight and body composition, including increased fat mass, after CFTR modulator therapy. These findings highlight the positive impact of CFTR modulators on nutritional status in children with CF.

Area of Science:

  • Pediatric Pulmonology
  • Medical Nutrition Therapy
  • Pharmacogenomics

Background:

  • Cystic Fibrosis (CF) is linked to poor nutrition, impacting morbidity, mortality, and lung function.
  • CFTR modulator (CFTRm) therapies aim to address the underlying cause of CF.
  • Assessing nutritional and body composition changes is crucial for evaluating CFTRm efficacy.

Purpose of the Study:

  • To investigate anthropometric and body composition changes in children with CF following CFTRm treatment.
  • To analyze these changes at 6 and 12 months post-treatment initiation.
  • To stratify results based on CFTRm regimen (dual vs. triple therapy).

Main Methods:

  • A cohort of 234 children with CF (median age 13.6 years) was studied.
  • Anthropometric parameters and body composition were assessed using bioelectrical impedance analysis.
  • Comparisons were made between pre-treatment and 6- and 12-month post-treatment data, with a focus on triple therapy (elexacaftor + tezacaftor + ivacaftor).

Main Results:

  • A statistically significant increase in weight Z-score and BMI Z-score was observed post-CFTRm therapy.
  • Fat mass (FM) significantly increased in kilograms and as a percentage at 6 months (p < .05).
  • Fat-free mass (FFM) significantly increased in kilograms at both 6 and 12 months (p < .05).

Conclusions:

  • CFTR modulator therapy leads to improvements in weight status and significant changes in body composition in children with CF.
  • Increased fat mass and fat-free mass were noted, suggesting a positive impact on nutritional status.
  • Further research is warranted to confirm these body composition changes and their long-term effects on CF disease progression.
Abstract

Keywords:
CFTR modulatorsanthropometric parametersbioelectrical impedancebody compositioncystic fibrosisnutrition status

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