Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

Cystic Fibrosis: Management

Cystic Fibrosis: Management

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.
Sinus disease and chronic...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Development of a Blocking ELISA for the Serological Diagnosis of Getah Virus.

Transboundary and emerging diseases·2026

Same author

Randomized Phase I/II Clinical Trial of a Melanoma Helper Peptide Vaccine with or without Systemic Agonistic Anti-CD27 Antibody (Varlilumab).

Cancer research communications·2026

Same author

Renal function decline in adults with cystic fibrosis experiencing pulmonary exacerbations.

Annals of the American Thoracic Society·2026

Same author

Social risk factors in patients with cystic fibrosis: impacts on exacerbations, lung function, and BMI.

Therapeutic advances in respiratory disease·2025

Same author

Remotely addressing health-related social needs (ELICIT) in cystic fibrosis: design and implementation of a multicenter screening and intervention quality improvement project.

Therapeutic advances in respiratory disease·2025

Same author

Cutaneous Adverse Effects of Elexacaftor-Tezacaftor-Ivacaftor: A Single Center Cohort Study on Acne Severity in Adults With Cystic Fibrosis.

Journal of cosmetic dermatology·2025

Same journal

Non-Linear Dysanaptic Lung Growth in Patients With Post-Infectious Bronchiolitis Obliterans.

Pediatric pulmonology·2026

Same journal

Safety of Less Invasive Surfactant Administration in Preterm Infants: A Meta-Analysis.

Pediatric pulmonology·2026

Same journal

Early Oxygenation Trajectory and Noninvasive Ventilation Failure in Non-Immunocompromised Children With Pediatric ARDS.

Pediatric pulmonology·2026

Same journal

Pulmonary Exacerbations and Treatment in PCD: A Narrative Review.

Pediatric pulmonology·2026

Same journal

Asthma in Children With Congenital Lung Malformations: A Population-Based Case-Control Study.

Pediatric pulmonology·2026

Same journal

Long-Term Effect Of COVID-19 on Children's Lung Function: A Prospective Longitudinal Study.

Pediatric pulmonology·2026

See all related articles

Search research articles

Related Experiment Video

Updated: Sep 14, 2025

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

Improving Modulator Management and Side Effect Screening: A CF Learning Network Innovation Lab.

Kevin P Lonabaugh¹, Anna Saulitis², Elizabeth Boettinger²

¹University of Virginia, Charlottesville, Virginia, USA.

Pediatric Pulmonology

|July 22, 2025

Summary

This summary is machine-generated.

Quality improvement methods increased cystic fibrosis transmembrane conductance regulator (CFTR) modulator use and side effect screening in people with CF. This approach enhanced modulator management and identified common, often unlisted, side effects.

Keywords:

cystic fibrosis elexacaftor‐tezacaftor‐ivacaftor modulators quality Improvement side effects

More Related Videos

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

A Fluorescence-Based Assay of Membrane Potential for High-Throughput Functional Study of Two Endogenous Ion Channels in Two Epithelial Cell Lines

A Fluorescence-Based Assay of Membrane Potential for High-Throughput Functional Study of Two Endogenous Ion Channels in Two Epithelial Cell Lines

Published on: June 22, 2022

Related Experiment Videos

Last Updated: Sep 14, 2025

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

A Fluorescence-Based Assay of Membrane Potential for High-Throughput Functional Study of Two Endogenous Ion Channels in Two Epithelial Cell Lines

A Fluorescence-Based Assay of Membrane Potential for High-Throughput Functional Study of Two Endogenous Ion Channels in Two Epithelial Cell Lines

Published on: June 22, 2022

Area of Science:

Pulmonology
Pharmacology
Quality Improvement Science

Background:

Over 90% of people with cystic fibrosis (PwCF) are eligible for CFTR modulators, yet 12% are not prescribed these life-changing therapies.
Gaps in modulator uptake and management prompted the CF Foundation CF Learning Network (CFLN) to launch a quality improvement (QI) initiative.

Purpose of the Study:

To investigate barriers to CFTR modulator use in eligible PwCF.
To implement and evaluate best practices for modulator initiation, management, and side effect screening.

Main Methods:

Thirty-one CF centers utilized the Model for Improvement and Plan-Do-Study-Act cycles to adapt interventions.
The study focused on two aims: increasing elexacaftor-tezacaftor-ivacaftor/ivacaftor (ETI/I) use or documented deferral, and improving side effect screening rates for PwCF on ETI.
Data were collected through weekly center submissions and analyzed using control and run charts.

Main Results:

From December 2022 to May 2023, 98.5% of evaluated PwCF had documented ETI/I use (89.3%) or deferral (9.2%), with previous side effects being the most common deferral reason (39.0%).
Between March and August 2024, ETI side effect screening rates improved from a baseline of 67.1% to 80.8%.
Potential side effects, including mood changes, inattention/brain fog, and weight gain, were identified in 24.5% of screenings.

Conclusions:

QI methods successfully enhanced CFTR modulator management and side effect screening across multiple CF centers.
Commonly reported side effects, some not listed in prescribing information, underscore the need for vigilant screening and management.
The developed processes and tools are reproducible and applicable to future CF therapies.