Related Experiment Video

Updated: Jul 17, 2025

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
08:00

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

Published on: April 11, 2018

10.7K

Update on Cystic Fibrosis in Pediatric Patients

Sarah Shrager Lusman1

  • 1Division of Pediatric Gastroenterology, Hepatology and Nutrition, Columbia University Irving Medical Center, 622 West 168th Street, PH 17 East - 105L, New York, NY, 10032, USA. ses2005@cumc.columbia.edu.

Current Gastroenterology Reports
|August 31, 2023
PubMed

Insights

Cystic fibrosis (CF) management has improved, with CFTR modulator therapies enhancing lung function and weight. Further research is needed to fully understand their impact on gastrointestinal and liver health in CF patients.

Area of Science:

  • Gastroenterology
  • Pulmonology
  • Genetics

Background:

  • Cystic fibrosis (CF) is a genetic, multisystem disorder impacting numerous organs.
  • Gastrointestinal and hepatobiliary complications are significant challenges in CF management.

Purpose of the Study:

  • To review nutritional, gastrointestinal, and hepatobiliary manifestations in CF.
  • To emphasize the impact of CFTR modulator therapy on these manifestations.

Main Methods:

  • Literature review of current research on CF gastrointestinal and hepatobiliary aspects.
  • Analysis of studies investigating CFTR modulator therapy's effects.

Main Results:

  • Increased life expectancy in CF patients due to improved therapies.
  • CFTR modulator therapy shows benefits in pulmonary function and weight gain.
  • Individualized nutritional support and early pancreatic intervention are recommended.
  • Non-invasive screening for hepatobiliary issues is advised.
  • The full impact of CFTR modulators on GI and liver health requires further investigation.

Conclusions:

  • CFTR modulators significantly improve survival and quality of life.
  • Ongoing research is crucial to elucidate the long-term effects of CFTR modulators on GI and hepatobiliary health.
  • Patient-reported outcomes and biomarkers are key for evaluating modulator efficacy.
Abstract

Keywords:
CFTRCystic fibrosisLiverModulatorNutritionPancreatic insufficiency

More Related Videos

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients07:04

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

Published on: February 11, 2017

19.3K
Cystic Fibrosis Aggregate Biofilm Model to Study Infection-relevant Gene Expression08:58

Cystic Fibrosis Aggregate Biofilm Model to Study Infection-relevant Gene Expression

Published on: April 18, 2025

565

Related Concept Videos

Cystic Fibrosis: Management01:24

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...
194
Cystic Fibrosis: Pathogenesis01:23

Cystic Fibrosis: Pathogenesis

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.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
278
Chronic Obstructive Pulmonary Disease-V: Management01:29

Chronic Obstructive Pulmonary Disease-V: Management

Managing Chronic Obstructive Pulmonary Disease (COPD) involves a multifaceted approach to reduce symptoms, prevent exacerbations, improve overall health status, and slow disease progression. Key strategies include lifestyle modifications, pharmacotherapy, supportive therapies, and, in some cases, surgery. Here is an overview of the primary COPD management strategies:
Smoking Cessation
2.6K