Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset

BackgroundGastrointestinal (GI) disease in pediatric patients with cystic fibrosis (CF) is a growing concern in the era of improved lung disease; however, the true prevalence of GI diagnoses, medical therapies, and frequency of diagnostic and therapeutic interventions in this population have yet to...

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Main Authors: Carolena Trocchia, Maua Mosha, Bailey Hamner, Neil Goldenberg, Racha Khalaf
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-01-01
Series:Frontiers in Gastroenterology
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Online Access:https://www.frontiersin.org/articles/10.3389/fgstr.2024.1489876/full
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author Carolena Trocchia
Carolena Trocchia
Maua Mosha
Bailey Hamner
Neil Goldenberg
Racha Khalaf
author_facet Carolena Trocchia
Carolena Trocchia
Maua Mosha
Bailey Hamner
Neil Goldenberg
Racha Khalaf
author_sort Carolena Trocchia
collection DOAJ
description BackgroundGastrointestinal (GI) disease in pediatric patients with cystic fibrosis (CF) is a growing concern in the era of improved lung disease; however, the true prevalence of GI diagnoses, medical therapies, and frequency of diagnostic and therapeutic interventions in this population have yet to be explored on a multisystem scale. The aim of the present study was to describe, among pediatric patients with CF (PwCF) compared to a large cohort of healthy controls, the prevalence and types of 1) GI disorders; 2) GI medication use; and 3) GI procedural interventions. MethodsThis was a multicenter case-control analysis using the TriNetX electronic medical record (EMR) global anonymized data platform. Patients were included if they had an ICD9/10 diagnosis code and were between the age of zero to ≤ 21 years between January 1st, 2010, and January 1st, 2020. Those with a history of solid organ transplants were excluded.ResultsThe cohort was comprised of 7,649 patients with a diagnosis of CF (cases) and 22,516,240 patients without CF (controls). The prevalence of any GI disorder was greater in pediatric PwCF compared to those without CF (73% versus <1%). The prevalence of diseases of the biliary tract and pancreas (43% vs. <1%; p<.0001), hepatic disease (8.8% vs. <1%; p<.0001), disease of the esophagus to duodenum (34% vs. <1%; p<.0001), and intestinal disease (43% vs. <1%; p<.0001) were each significantly greater in PwCF (p<0.0001). Furthermore, the frequencies of esophagogastroduodenoscopy (EGD), colonoscopy and endoscopic retrograde cholangiopancreatography (ERCP) were significantly higher among PwCF than controls (9.5% vs. <1%; 2.8%% vs. <1%; and 0.3% vs <.001% respectively; p<0.0001 for all comparisons). ConclusionThe prevalence of GI diagnoses, use of GI medications, and frequency of GI procedures are all higher among pediatric patients PwCF. Prospective multicenter studies are warranted to substantiate these findings, further investigate risk factors for GI disorders, and to describe potential changes in GI disorders with novel CF disease-modifying therapies.
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spelling doaj-art-8dd280d777ac4bac8bef425932a38f272025-01-24T07:13:19ZengFrontiers Media S.A.Frontiers in Gastroenterology2813-11692025-01-01310.3389/fgstr.2024.14898761489876Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world datasetCarolena Trocchia0Carolena Trocchia1Maua Mosha2Bailey Hamner3Neil Goldenberg4Racha Khalaf5School of Medicine, Stanford University, Stanford, CA, United StatesDepartment of Pediatric Gastroenterology, Hepatology and Nutrition, Stanford Healthcare, Stanford, CA, United StatesInstitute for Clinical and Translational Research, Johns Hopkins All Children’s Hospital, Saint Petersburg, FL, United StatesDepartment of Pediatric Gastroenterology, Hepatology and Nutrition, University of South Florida, Tampa, FL, United StatesInstitute for Clinical and Translational Research, Johns Hopkins All Children’s Hospital, Saint Petersburg, FL, United StatesDepartment of Pediatric Gastroenterology, Hepatology and Nutrition, University of South Florida, Tampa, FL, United StatesBackgroundGastrointestinal (GI) disease in pediatric patients with cystic fibrosis (CF) is a growing concern in the era of improved lung disease; however, the true prevalence of GI diagnoses, medical therapies, and frequency of diagnostic and therapeutic interventions in this population have yet to be explored on a multisystem scale. The aim of the present study was to describe, among pediatric patients with CF (PwCF) compared to a large cohort of healthy controls, the prevalence and types of 1) GI disorders; 2) GI medication use; and 3) GI procedural interventions. MethodsThis was a multicenter case-control analysis using the TriNetX electronic medical record (EMR) global anonymized data platform. Patients were included if they had an ICD9/10 diagnosis code and were between the age of zero to ≤ 21 years between January 1st, 2010, and January 1st, 2020. Those with a history of solid organ transplants were excluded.ResultsThe cohort was comprised of 7,649 patients with a diagnosis of CF (cases) and 22,516,240 patients without CF (controls). The prevalence of any GI disorder was greater in pediatric PwCF compared to those without CF (73% versus <1%). The prevalence of diseases of the biliary tract and pancreas (43% vs. <1%; p<.0001), hepatic disease (8.8% vs. <1%; p<.0001), disease of the esophagus to duodenum (34% vs. <1%; p<.0001), and intestinal disease (43% vs. <1%; p<.0001) were each significantly greater in PwCF (p<0.0001). Furthermore, the frequencies of esophagogastroduodenoscopy (EGD), colonoscopy and endoscopic retrograde cholangiopancreatography (ERCP) were significantly higher among PwCF than controls (9.5% vs. <1%; 2.8%% vs. <1%; and 0.3% vs <.001% respectively; p<0.0001 for all comparisons). ConclusionThe prevalence of GI diagnoses, use of GI medications, and frequency of GI procedures are all higher among pediatric patients PwCF. Prospective multicenter studies are warranted to substantiate these findings, further investigate risk factors for GI disorders, and to describe potential changes in GI disorders with novel CF disease-modifying therapies.https://www.frontiersin.org/articles/10.3389/fgstr.2024.1489876/fullcystic fibrosisexocrine pancreatic insufficiencyelexacaftor/tezacaftor/ivacaftor (ETI)modulator therapygastrointestinal disease
spellingShingle Carolena Trocchia
Carolena Trocchia
Maua Mosha
Bailey Hamner
Neil Goldenberg
Racha Khalaf
Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset
Frontiers in Gastroenterology
cystic fibrosis
exocrine pancreatic insufficiency
elexacaftor/tezacaftor/ivacaftor (ETI)
modulator therapy
gastrointestinal disease
title Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset
title_full Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset
title_fullStr Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset
title_full_unstemmed Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset
title_short Prevalence and characteristics of gastrointestinal disorders, medication use, and diagnostic interventions in pediatric patients with cystic fibrosis: a nested case–control analysis from the TriNetX EMR-derived global research network real-world dataset
title_sort prevalence and characteristics of gastrointestinal disorders medication use and diagnostic interventions in pediatric patients with cystic fibrosis a nested case control analysis from the trinetx emr derived global research network real world dataset
topic cystic fibrosis
exocrine pancreatic insufficiency
elexacaftor/tezacaftor/ivacaftor (ETI)
modulator therapy
gastrointestinal disease
url https://www.frontiersin.org/articles/10.3389/fgstr.2024.1489876/full
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