Evidence map›Paper›PMID 39868969›Full record

ArticlePediatric pulmonology2025

Effects of Elexacaftor-Tezacaftor-Ivacaftor on Nasal and Sinus Symptoms in Children With Cystic Fibrosis.

Guillaume Petit, Aurélie Coudert, Ruben Hermann, Eric Truy, Maxime Bonjour, Philippe Reix, Sonia Ayari

Registry-linked trialAbstract read
In one paragraph

Article in Pediatric pulmonology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05581056 (Evaluation of the Evolution of Quality of Life in Relation to Naso-sinus Symptomatology Under Treatment With CFTR Modulators in Children Aged 6 to 11 Years With Cystic Fibrosis With Compatible Mutation), which is not on this map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
–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.

NCT05581056 completednot on this map

Evaluation of the Evolution of Quality of Life in Relation to Naso-sinus Symptomatology Under Treatment With CFTR Modulators in Children Aged 6 to 11 Years With Cystic Fibrosis With Compatible Mutation

TypeobservationalSponsorHospices Civils de LyonRan2023 to 2024Enrolled27ConditionsCystic FibrosisArmsCFTR modulators
3 · Its place in the literature

Who cites it

2 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Observational
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

7 authors.

Guillaume PetitHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.ORCID http://orcid.org/0009-0007-0210-6983
Aurélie CoudertHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.
Ruben HermannHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.
Eric TruyHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.
Maxime BonjourHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.ORCID http://orcid.org/0000-0003-2205-9655
Philippe ReixHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.ORCID http://orcid.org/0000-0002-9192-8335
Sonia AyariHôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Lyon, France.

Funding

The authors received no specific funding for this work.
6 · The paper itself

Abstract

backgroundNew CFTR Modulator triple therapy Elexacaftor-Ivacaftor-Tezacaftor (ETI) prove efficacy in pulmonary outcomes. However, its impact on nasal sinus symptoms in children has not been specifically studied. The aim of this study is to evaluate the impact of this therapy on nasal sinus symptomatology in children aged 6-12 years.

methodsA prospective, single-center cohort study was conducted over a 12-month follow-up period in children aged 6-12 years at the initiation of ETI therapy. The primary outcome was evolution of the SN-5 score, a validated pediatric questionnaire measuring quality of life related to nasal sinus symptoms. A decrease of 0.5 points is considered clinically significant. Secondary outcomes included changes in clinical examination findings (obstructive turbinate hypertrophy, polyps, presence of pus in the middle meatus, and externalized mucocele), quality of life measured by the Visual Analog Scale (VAS), and number of antibiotic courses during the study period.

resultsTwenty-six patients were included between March and September 2023, with no lost to follow-up. The initial mean SN-5 score was 2.88 (95% CI {1.91; 3.85}). After 1 year, the mean SN-5 score was significantly lower (1.41, 95% CI {1.00; 1.88}, Delta = 1.47, p < 0.001). The VAS related to symptoms also improved (Delta = 1.7, p < 0.001), and the number of antibiotic courses decreased (25 vs. 69, p < 0.001). A trend toward improvement in clinical examination parameters was observed.

conclusionETI therapy appears to significantly improve nasal sinus symptoms in children aged 6-12 years, as evidenced by improved quality-of-life scales and reduced antibiotic use.

Indexed as

AminophenolsBenzodioxolesCystic FibrosisIndolesPyrazolesPyridinesPyrrolidinesQuinolonesChildChloride Channel AgonistsDrug CombinationsFemaleFollow-Up StudiesHumansMaleProspective StudiesAminophenolsBenzodioxolesChloride Channel AgonistsDrug Combinationselexacaftor, ivacaftor, tezacaftor drug combinationIndolesivacaftorPyrazolesPyridinesPyrrolidinesQuinolinesQuinolonestezacaftor, ivacaftor drug combination

Identifiers

PMID39868969
PMCPMC11771559

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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.