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  1. Home
  2. Comprehensive Evaluation Of Elexacaftor/tezacaftor/ivacaftor In Paediatric Cystic Fibrosis: Nutritional, Pulmonary, And Quality-of-life Outcomes.
  1. Home
  2. Comprehensive Evaluation Of Elexacaftor/tezacaftor/ivacaftor In Paediatric Cystic Fibrosis: Nutritional, Pulmonary, And Quality-of-life Outcomes.

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Comprehensive Evaluation of Elexacaftor/Tezacaftor/Ivacaftor in Paediatric Cystic Fibrosis: Nutritional, Pulmonary,

Katarzyna Walicka-Serzysko1,2, Magdalena Postek1,2, Monika Mielus1,2

  • 1Cystic Fibrosis Department, Institute of Mother and Child, 01-211 Warsaw, Poland.

Journal of Clinical Medicine
|November 27, 2025

View abstract on PubMed

Summary
This summary is machine-generated.

Elexacaftor/tezacaftor/ivacaftor (ETI) therapy significantly improved nutritional status, lung function, and quality of life in pediatric cystic fibrosis patients. This real-world study confirms ETI

Keywords:
CFTR modulatorbody compositioncystic fibrosisfat free massfat masshealth-related quality of lifelung clearance indexnutritional statusspirometry

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Area of Science:

  • Pediatric Pulmonology
  • Clinical Pharmacology
  • Medical Nutrition Therapy

Background:

  • Cystic fibrosis transmembrane conductance regulator modulator (mCFTR) therapies show efficacy in clinical trials.
  • Real-world data on the comprehensive effects of elexacaftor/tezacaftor/ivacaftor (ETI) in mCFTR treatment-naïve pediatric patients are limited.
  • Understanding ETI's impact beyond clinical trials is crucial for patient management.

Purpose of the Study:

  • To evaluate the comprehensive effects of ETI on nutritional status, respiratory system, and quality of life in pediatric cystic fibrosis patients over 12 months of real-world use.
  • To assess changes in anthropometry, body composition, lung function (spirometry, LCI), and quality of life (CFQ-R) following ETI initiation.
  • To provide post-approval insights into ETI's clinical utility in a treatment-naïve pediatric population.

Main Methods:

  • Retrospective analysis of medical records from 58 cystic fibrosis adolescents treated with ETI.
  • Evaluation of anthropometric measurements, body composition (BIA), spirometry (ppFEV1, ppFVC), and lung clearance index (LCI) before and at 3 and 12 months post-therapy.
  • Assessment of quality of life using the Cystic Fibrosis Questionnaire-Revised (CFQ-R).

Main Results:

  • Significant increases in body weight (mean gain 7.10 kg at 12 months), BMI z-score, fat-free mass, and fat mass were observed (p < 0.001).
  • Pulmonary function improved significantly, with increases in ppFEV1 and ppFVC, and a decrease in LCI (p < 0.001).
  • The CFQ-R Respiratory score improved by 11.75 points, correlating with pulmonary and nutritional parameters.

Conclusions:

  • In real-world settings, ETI therapy demonstrates a comprehensive positive impact on nutritional status, body composition, and pulmonary function in adolescents with cystic fibrosis.
  • ETI treatment leads to significant improvements in quality of life, as reflected by the CFQ-R scores.
  • These findings support the broad clinical benefits of ETI in pediatric CF patients initiating modulator therapy.