Evidence map›Paper›PMID 41692662›Full record

Observational studyJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2026

Impact of elexacaftor/tezacaftor/ivacaftor on gastrointestinal outcomes, inflammation, exocrine pancreatic function and fat malabsorption: Report of PROMISE pediatric substudy.

Nicole Green, Phuong Vu, Jing Xie, Sarah Jane Schwarzenberg, Steven Freedman, Michael Narkewicz, Jessica Pittman, Felix Ratjen, Margaret Rosenfeld, Christopher Pope and 5 more

Abstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Nicole GreenDepartment of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA. Electronic address: nicole.green@seattlechildrens.org.
Phuong VuCenter for Respiratory Biology and Therapeutics, Seattle Children's Research Institute, Seattle, WA, USA.
Jing XieCystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, USA.
Sarah Jane SchwarzenbergDepartment of Pediatrics, University of Minnesota Masonic Children's Hospital, Minneapolis, MN, USA.
Steven FreedmanBeth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA.
Michael NarkewiczDigestive Health Institute, Children's Hospital Colorado and Section of Pediatric Gastroenterology, Hepatology and Nutrition, University of Colorado School of Medicine, Aurora, CO, USA.
Jessica PittmanDepartment of Pediatrics, Washington University School of Medicine, St Louis, MO, USA.
Felix RatjenDivision of Respiratory Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada.
Margaret RosenfeldDepartment of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA; Center for Respiratory Biology and Therapeutics, Seattle Children's Research Institute, Seattle, WA, USA.
Christopher PopeDepartment of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA; Department of Microbiology, University of Washington, School of Medicine, Seattle, WA, USA.
Jennifer DuongDepartment of Pediatrics, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, University of California San Francisco School of Medicine, San Francisco, CA, USA.
Lucas HoffmanDepartment of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA; Center for Respiratory Biology and Therapeutics, Seattle Children's Research Institute, Seattle, WA, USA; Department of Microbiology, University of Washington, School of Medicine, Seattle, WA, USA.
Sonya HeltsheDepartment of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA; Center for Respiratory Biology and Therapeutics, Seattle Children's Research Institute, Seattle, WA, USA; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, USA.
Sarah S LusmanDepartment of Pediatrics, Columbia University Irving Medical Center, New York, NY, USA.
PROMISE Study Group

Funding

Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss, Lucas R Hoffman · 2010 to 2026
$21.4M
NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

backgroundCFTR mutations likely contribute to inflammation, dysmotility and dysbiosis in the CF intestine. While CFTR modulators such as Elexacaftor/Tezacaftor/Ivacaftor (ETI) have shown pulmonary benefit, their efficacy on gastrointestinal (GI) outcomes is less clear. The PROMISE pediatric sub-study examines GI symptoms and fecal biomarkers in children with CF 6 to <12 years of age who recently initiated ETI.

methodsPatient-Reported Outcome Measures (PROMs) from 3 validated GI symptom questionnaires (PAGI-SYM, PAC-SYM, PAC-QOL) and a stool-specific questionnaire (SSQ) were obtained. Stool samples were collected for biomarker analysis. We report on changes from baseline in PROMs, fecal calprotectin, elastase and steatocrit after ETI initiation. Estimated change and 95 % confidence interval (CI) were obtained from linear regression with adjustment for prior modulator use and sex at birth.

resultsThere was no significant change from baseline in GI symptoms based on PROMs at 12 months. For fecal calprotectin, the proportion of participants with values in the normal range (≤80μg/g) increased from 22 % at baseline to 30 % at 1 month and 40 % at 6 months. There were no statistically significant changes in the proportions of those with fecal elastase in the normal range (>200μg/g), nor in the severe insufficiency range (<100μg/g).

conclusionsAs fecal calprotectin is a marker of intestinal inflammation, the increase in the proportion of patients with normal calprotectin at 6 months suggests that use of ETI potentially led to amelioration of intestinal inflammation. There was little change in exocrine pancreatic function after 6 months or GI symptoms after 12 months of treatment.

Indexed as

Chloride Channel AgonistsCystic FibrosisGastrointestinal DiseasesLeukocyte L1 Antigen ComplexAminophenolsBenzodioxolesBiomarkersChildDrug CombinationsFecesFemaleHumansIndolesInflammationIsoquinolinesMaleAminophenolsBenzodioxolesBiomarkersChloride Channel AgonistsDrug Combinationselexacaftorelexacaftor, ivacaftor, tezacaftor drug combinationIndolesIsoquinolinesLeukocyte L1 Antigen ComplexPyrazolesPyridinesPyrrolidinesQuinolinesQuinolonestezacaftorCFTR modulatorsGastrointestinalGastrointestinal symptomsInflammationPancreatic insufficiencyPatient-reported outcome measures

Identifiers

PMID41692662
PMCPMC13062532

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.