Evidence map›Paper›PMID 39811548›Full record

ArticleERJ open research2025

The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol.

Christine M Liu, Jakob L Fischer, Edith T Zemanick, Jason C Woods, Karolin K Markarian, Sean B Fain, Deborah Froh, Sonya L Heltshe, Lucas R Hoffman, Stephen M Humphries and 14 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in ERJ open research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06191640 (Sinus Disease in Young Children With Cystic Fibrosis), which is not on this 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.

NCT06191640 recruitingnot on this map

Sinus Disease in Young Children With Cystic Fibrosis

TypeobservationalSponsorUniversity of California, Los AngelesRan2023 to 2029Enrolled80ConditionsCystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory DisorderArmsIvacaftor or elexacaftor/tezacaftor/ivacaftor
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

24 authors.

Christine M LiuDepartment of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA.
Jakob L FischerDepartment of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA.
Edith T ZemanickDepartment of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA.
Jason C WoodsDepartment of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Karolin K MarkarianUniversity of California, Los Angeles, CTSI, David Geffen School of Medicine, Los Angeles, CA, USA.
Sean B FainDepartment of Radiology, University of Iowa, Iowa City, IA, USA.ORCID https://orcid.org/0000-0001-5461-0646
Deborah FrohDepartment of Pediatrics, University of Virginia, Charlottesville, VA, USA.
Sonya L HeltsheDepartment of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, WA, USA.
Lucas R HoffmanDepartment of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, WA, USA.
Stephen M HumphriesDepartment of Radiology, National Jewish Health, Aurora, CO, USA.ORCID https://orcid.org/0000-0002-5113-4530
Elizabeth L KramerDepartment of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Katie Larson OdeDepartment of Pediatrics, University of Iowa, Iowa City, IA, USA.
Michael LewisDepartment of Pediatrics, University of Kansas Medical Center, Kansas City, KS, USA.
Douglas A LiDepartment of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.
Jaime MataDepartment of Radiology and Medical Imaging, University of Virginia, Charlottesville, VA, USA.
Sarah S MillaDepartment of Radiology, Children's Hospital Colorado, Aurora, CO, USA.
Peter J NiedbalskiDepartment of Pulmonary, Critical Care, and Sleep Medicine, University of Kansas Medical Center, Kansas City, KS, USA.
Benjamin D SawatzkyDepartment of Radiology, University of Vermont, Colchester, VT, USA.
Myung-Shin SimDepartment of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.ORCID https://orcid.org/0000-0001-9706-1499
Jillian S SullivanDepartment of Pediatrics, University of Vermont, Colchester, VT, USA.
Andrew T TroutDepartment of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Christopher H GossDepartment of Medicine and Pediatrics, University of Washington, Seattle, WA, USA.
Jennifer L Taylor-CousarDepartment of Medicine, National Jewish Health, Aurora, CO, USA.ORCID https://orcid.org/0000-0002-5436-9722
Daniel M BeswickDepartment of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA.

Funding

Pulmonary Toxicology Facility CoreP30ES005605 · NIEHS · UNIVERSITY OF IOWA · PI Jong Sung Kim · 1990 to 2026
$40.5M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI PRADEEP k SINGH · 2010 to 2026
$21.4M
Patient-oriented microbiome and advanced culture approaches to identifying the microbial determinants of chronic pediatric diseaseK24HL141669 · NHLBI · UNIVERSITY OF WASHINGTON · PI Lucas R Hoffman · 2018 to 2026
$929k
Enabling careers as surgeon-scientists in otolaryngology-head and neck surgeryR25DC020151 · NIDCD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Jennifer L Long · 2022 to 2026
$887k
NHLBI NIH HHS K24 HL141669NIDCD NIH HHS R25 DC020151NIDDK NIH HHS P30 DK089507NIEHS NIH HHS P30 ES005605
6 · The paper itself

Abstract

Background: Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are prevalent disease complications in people with cystic fibrosis. These understudied comorbidities significantly impact quality of life. The impact of highly effective modulator therapy (HEMT) in young children with cystic fibrosis (YCwCF) on these disease complications is unknown. This proposed study aims to characterise CRS and OD in YCwCF and assess the efficacy of HEMT in improving sinus and olfactory health in this young age group. Methods: This six-centre, prospective, observational study will enrol 80 YCwCF aged 2-8 years. Patients are divided into two groups: those receiving HEMT and those not on HEMT based on clinical indication. Both groups undergo sinus magnetic resonance imaging, psychophysical olfactory tests, and complete patient- or parent-reported quality of life surveys over 2 years. Outcomes will be compared before and after initiation of HEMT and between groups. Ethical approval has been obtained for all sites, and this study has been registered on ClinicalTrials.gov (NCT06191640). Results: Enrolment began in April 2023. 21 participants have been enrolled as of October 2023 with ongoing enrolment at all sites. Conclusion: This investigation is expected to provide critical insights into the potential benefits of early HEMT initiation in managing CRS and OD in YCwCF. It will assist in developing targeted interventions and contribute to the understanding of HEMT's role in altering the disease course in this demographic.

Identifiers

PMID39811548
PMCPMC11726580

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Registered trials

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.