Evidence map›Paper›PMID 40047648›Full record

Observational studyInternational forum of allergy & rhinology2025

Beyond the Lung. Impact of Elexacaftor/Tezacaftor/Ivacaftor on Sinonasal Disease in Children With Cystic Fibrosis.

Margaux Petitjean, Alexia Letierce, Anne-Sophie Bonnel, Philippe Reix, Eric Deneuville, Nathalie Stremler, Romain Luscan, Vincent Couloigner, Laurent Mely, Katia Bessaci and 7 more

Abstract readMulticenter StudyObservational Study
In one paragraph

Observational study in International forum of allergy & rhinology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. Review
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

17 authors.

Margaux PetitjeanService d'Otorhinolaryngologie, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Université Paris Cité, Paris, France.
Alexia LetierceQualitystat, Morangis, France.
Anne-Sophie BonnelCentre de Référence Maladies Rares Mucoviscidose et Maladies Apparentées, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Groupe Hospitalier Paris Centre, Paris, France.
Philippe ReixCentre de Ressources et de Compétence pour la Mucoviscidose, Hospices Civils de Lyon, Lyon, France.
Eric DeneuvilleCentre de Ressources et de Compétence pour la Mucoviscidose, Centre Hospitalo-Universitaire, Rennes, France.
Nathalie StremlerCentre de Ressources et de Compétence pour la Mucoviscidose, Hôpital La Timone-Enfants, Marseille, France.
Romain LuscanService d'Otorhinolaryngologie, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Université Paris Cité, Paris, France.
Vincent CouloignerService d'Otorhinolaryngologie, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Université Paris Cité, Paris, France.
Laurent MelyCentre de Ressources et de Compétence pour la Mucoviscidose, Hôpital Renée Sabran, Hospices civils de Lyon, Giens, France.
Katia BessaciCentre de Ressources et de Compétence pour la Mucoviscidose, Centre Hospitalo-Universitaire, Reims, France.
Guillaume LabbeCentre de Ressources et de Compétence pour la Mucoviscidose, Centre Hospitalo-Universitaire, Clermont Ferrand, France.
Christophe MarguetCentre de Ressources et de Compétence pour la Mucoviscidose, Centre Hospitalo-Universitaire, Rouen, France.
Mairead Kelly-AubertINSERM U1151, Université Paris Cité, Paris, France.
Fatiha HassaniCentre de Référence Maladies Rares Mucoviscidose et Maladies Apparentées, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Groupe Hospitalier Paris Centre, Paris, France.
Isabelle Sermet-GaudelusCentre de Référence Maladies Rares Mucoviscidose et Maladies Apparentées, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Groupe Hospitalier Paris Centre, Paris, France.ORCID https://orcid.org/0000-0001-5537-9482
François SimonService d'Otorhinolaryngologie, Hôpital Necker Enfants malades, Assistance Publique Hôpitaux de Paris, Université Paris Cité, Paris, France.
MODUL‐CF Study Group

Funding

Association Vaincre la Mucoviscidose RC20240503466CFF 003341121
6 · The paper itself

Abstract

backgroundElexacaftor/tezacaftor/ivacaftor (ETI) is a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) therapy that improves pulmonary function and chronic rhinosinusitis (CRS) in cystic fibrosis (CF) adults with at least one copy of the F508del CFTR mutation. The purpose of this study is to evaluate the impact of ETI on CRS symptoms in children and adolescents with CF.

methodsThe MODUL-CF observational study is a multicenter prospective cohort study enrolling CF children with at least 1 F508del mutation in France. Subjects answered a standardized questionnaire on nasal obstruction and smell and were invited to complete the Sinus and Nasal Quality of Life Survey (SN-5) questionnaire prior to ETI therapy and at 1, 3, 6, and 12 months ETI. Part of the cohort underwent sinus computerized tomography (CT) within an ancillary study, scored by the Lund-Mackay CT score (LMKS).

resultsOf 391 subjects, 94 (24.0%) were aged between 6 and 12 years, and 297 were adolescents. Sixty-four (16.3%) reported nasal obstruction at baseline. One hundred and sixty-three completed the SN-5 questionnaire at M0, 181 at M12, and 123 at M0 and M12. Mean SN-5 global score baseline value was similar to that of a healthy pediatric control cohort. SN-5 global score, nasal obstruction, sinus infection, and emotion domain subscores improved significantly at 1 month and this was sustained over 12 months. LMKS improved significantly in 43 patients who underwent sinus CT at M0 and M12.

conclusionETI improves sinonasal symptoms and related quality of life (QOL), including emotion domain, in children and adolescents with CF.

Indexed as

AminophenolsBenzodioxolesCystic FibrosisIndolesPyrazolesPyridinesPyrrolidinesQuinolonesRhinitisSinusitisAdolescentChildChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleAminophenolsBenzodioxolesChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftor, ivacaftor, tezacaftor drug combinationIndolesivacaftorPyrazolesPyridinesPyrrolidinesQuinolinesQuinolonesCFTRCFTR modulatorschronic rhinosinusitiscystic fibrosisquality of life

Identifiers

PMID40047648
PMCPMC12227108

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.