implementation-non-invasive-point-care-transient-elastography-for

Implementation of Non-invasive Point of Care Transient Elastography for Evaluation of Liver Disease in Pediatric Populations with Cystic Fibrosis

Cystic Fibrosis (CF) is an inherited condition that impacts multiple organ systems, one of which is the liver. The presentation and manifestation of liver ...

University of Texas Southwestern Medical Center/Children's Health

Is meconium ileus associated with worse outcomes in cystic fibrosis?

The Food and Drug Administration considers patient-reported outcome measures (PROMs) an essential part of clinical research studies for approval of new drugs and new indications for existing drugs. GALAXY evaluated the feasibility of electronic PROMs (ePROMS) to conduct a comprehensive evaluation of gastrointestinal (GI) symptoms in persons with cystic fibrosis (pwCF).

Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study.

Analysis of the liver using imaging for persons with cystic fibrosis (CF) continues to evolve as new medical therapies are developed improving and extending life. In the 2010s, therapies targeted at modulating protein folding became available to those with CF. Therapeutic options have continued to expand, now providing both correction of protein folding and stabilization for most gene mutations that code for the CF transmembrane receptor protein (CFTR). Today, approximately 80% of persons with CF are eligible for highly effective modulator therapy. With these advancements, the impact of CF on the liver has become more complex, adding metabolism of CFTR modulators to intrinsic CF hepatobiliary involvement (CFHBI) and adding not previously appreciated vascular changes within the liver due to increased longevity in persons with CF. A combination of serum biomarkers and imaging is needed to add clarity to the diagnosis and monitoring of the severity of liver disease. A substantial portion of persons with CF will develop at least CFHBI and a subset will develop advanced cystic fibrosis-associated liver disease (aCFLD); therefore, diagnosis and monitoring need to begin in childhood. In this review, we cover the use of and need for imaging, including elastography, ultrasound, and magnetic resonance imaging (MRI), in diagnosing and monitoring CFHBI and its associated complications.

Cystic fibrosis hepatobiliary involvement: an update on imaging in diagnosis and monitoring.

Minimal clinically important difference (MCID) is important to establish as a meaningful outcome in research when using patient reported outcome measures (PROMs). We determined the MCID using the distribution-based approach for three measurements used as part of the GALAXY study, which is an observational prospective study on gastrointestinal (GI) symptoms in cystic fibrosis (CF).

Article	Year
Is meconium ileus associated with worse outcomes in cystic fibrosis? Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society\| PubMed ID: 31668933	2019
Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society\| PubMed ID: 34305007	2021
Cystic fibrosis hepatobiliary involvement: an update on imaging in diagnosis and monitoring. Pediatric radiology\| PubMed ID: 39039200	2024
Estimating minimal clinically important difference (MCID) for gastrointestinal symptoms in cystic fibrosis. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society\| PubMed ID: 39048465	2024

Meghana Sathe

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