Cystic Fibrosis Spotlight-Cystic Fibrosis for the General Pulmonologist

Video Transcription

Video Summary

Cystic fibrosis (CF) is a multi-system disease that affects multiple organs, including the lungs, digestive system, pancreas, and more. Monitoring and managing CF requires a range of tests and therapies, including CFTR modulators that target specific mutations in the CFTR protein.<br /><br />The CFTR protein is responsible for regulating chloride and bicarbonate transport, as well as inflammation and mucus production. Mutations in the CFTR gene can cause dysfunction in these areas, leading to mucus buildup, infections, inflammation, and other symptoms.<br /><br />CFTR modulators work by targeting specific mutations in the CFTR protein. Potentiators enhance chloride channel activity, while correctors help correct the folding and trafficking of the protein to the cell surface. These modulators have shown significant benefits in improving lung function, reducing exacerbations, and increasing body mass index (BMI) in CF patients.<br /><br />There are several CFTR modulators currently available, including Ivacaftor, Lumacaftor/Ivacaftor (Orkambi), Tezacaftor/Ivacaftor (Symdeko), and the most recent one, Elexacaftor/Tezacaftor/Ivacaftor (Trikafta). These medications have shown significant improvements in lung function and quality of life for CF patients.<br /><br />However, it is important to monitor patients on CFTR modulators, as they can have side effects and may interact with other medications. Liver function tests and eye exams are important to monitor potential side effects, and dose adjustments may be necessary for patients with hepatic impairment or those taking certain medications.<br /><br />In conclusion, CFTR modulators have revolutionized the management of CF, improving lung function, reducing exacerbations, and enhancing quality of life for patients. However, careful monitoring and management are essential to ensure optimal outcomes and minimize potential side effects.

Meta Tag

Category Genetic and Developmental Disorders

Speaker Holly Keyt, MD, FCCP

Speaker Meilinh Thi, DO

Speaker Douglas Hornick, MD, FCCP

Speaker Sarah Chalmers, MD

Keywords

Cystic fibrosis

CF

CFTR modulators

mutations

lung function

exacerbations

body mass index

side effects

liver function tests

quality of life

Cystic Fibrosis

CF