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Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
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  1. Home
  3. Identification Of Early Changes In Multiple Biomarkers Following Cftr Modulator Initiation In Patients With Cystic Fibrosis.
  1. Home
  3. Identification Of Early Changes In Multiple Biomarkers Following Cftr Modulator Initiation In Patients With Cystic Fibrosis.

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Identification of early changes in multiple biomarkers following CFTR modulator initiation in patients with cystic

Pascal Heer1, Clara Fernandez Elviro2, Angela Koutsokera3

  • 1Pediatric Pulmonology and Cystic Fibrosis Unit, Children's Hospital of Central Switzerland, Lucerne, Switzerland.

Therapeutic Advances in Respiratory Disease
|October 9, 2025

View abstract on PubMed

Summary
This summary is machine-generated.

Early assessment of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator efficacy is possible using clinical, biologic, and functional parameters within days of treatment initiation. These early indicators may predict long-term treatment success in CF patients.

Keywords:
CFTR modulator treatmentelexacaftorivacaftornasal potential differenceshort-term responsetezacaftor

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Area of Science:

  • Pulmonology
  • Medical Genetics
  • Pharmacology

Background:

  • Predicting long-term responses to Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy remains challenging without early indicators.
  • Current methods lack individual-level prediction parameters for CFTR modulator treatment efficacy.

Purpose of the Study:

  • To identify early parameters for assessing CFTR modulator efficacy within 7 to 14 days of treatment initiation.
  • To establish potential predictors of long-term responses to CFTR modulator therapy.

Main Methods:

  • Prospective observational study involving 47 Cystic Fibrosis (CF) patients across three Swiss clinics.
  • Standardized clinical, biological, and functional measurements were taken within 2 months pre-treatment and 7-14 days post-initiation of elexacaftor/tezacaftor/ivacaftor (ETI) therapy.

Main Results:

  • Significant improvements were observed in BMI z-score, inflammatory markers (white blood cells, neutrophils, immunoglobulin G, fecal calprotectin), and CFTR functional assays (sweat chloride, nasal potential difference).
  • Lung function parameters including FVC, FEV1, MMEF25-75, and LCI2.5% showed significant early improvement (p < 0.001).
  • No significant changes were noted in blood pressure, SpO2, respiratory rate, ESR, CRP, or fecal elastase.

Conclusions:

  • Clinical, biological, and functional parameters demonstrate early treatment effects following CFTR modulator initiation.
  • These early-identified parameters show potential as predictors for long-term CFTR modulator treatment responses in CF patients.