Evidence map›Paper›PMID 41238903›Full record

ArticlePediatric research2026

Effectiveness of elexacaftor/tezacaftor/ivacaftor therapy in children and adolescents with cystic fibrosis and normal spirometry.

Gianfranco Alicandro, Vito Terlizzi, Laura Zazzeron, Daniela Dolce, Lisa Cariani, Arianna Fiandaca, Silvia Campana, Alberto Terminiello, Diletta Innocenti, Francesco Blasi and 1 more

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Article in Pediatric research, 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
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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

11 authors.

Gianfranco AlicandroDepartment of Physiopathology and Transplantation, Università degli Studi di Milano, Milan, Italy.
Vito TerlizziMeyer Children's Hospital IRCCS, Cystic Fibrosis Regional Reference Centre, Department of Paediatric Medicine, Florence, Italy. vito.terlizzi@meyer.it.
Laura ZazzeronMother and Child Department, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Daniela DolceMeyer Children's Hospital IRCCS, Cystic Fibrosis Regional Reference Centre, Department of Paediatric Medicine, Florence, Italy.
Lisa CarianiMicrobiology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Arianna FiandacaMother and Child Department, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Silvia CampanaMeyer Children's Hospital IRCCS, Cystic Fibrosis Regional Reference Centre, Department of Paediatric Medicine, Florence, Italy.
Alberto TerminielloPediatric Pulmonary Unit, Meyer Children's Hospital IRCCS, Florence, Italy.
Diletta InnocentiMeyer Children's Hospital IRCCS, Rehabilitation Unit, Florence, Italy.
Francesco BlasiDepartment of Physiopathology and Transplantation, Università degli Studi di Milano, Milan, Italy.
Valeria DaccòMother and Child Department, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundCystic fibrosis (CF) is a genetic disorder caused by variants in the CFTR gene. CFTR modulators, especially elexacaftor/tezacaftor/ivacaftor (ETI), have significantly improved CF care, but their effectiveness in children and adolescents with normal spirometry remains unexplored.

methodsThis prospective study included 103 individuals with CF under 18 years old, all with baseline percent predicted forced expiratory volume in one second (ppFEV

resultsDespite normal spirometry at baseline, 73.5% of participants exhibited abnormal LCI

conclusionsETI therapy provided substantial clinical benefits, supporting early intervention in pediatric patients with CF, even with preserved spirometry. IMPACT: This study explored the effects of ETI therapy in children and adolescents with CF and normal spirometry. Most showed early signs of lung disease based on LCI evaluation. ETI led to significant improvements in lung function and growth. Our findings stress the importance of early ETI intervention to optimize both growth and lung health in patients with CF.

Indexed as

AminophenolsBenzodioxolesCystic FibrosisIndolesPyrazolesPyridinesPyrrolidinesQuinolonesAdolescentBody Mass IndexChildChloridesCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleForced Expiratory VolumeAminophenolsBenzodioxolesCFTR protein, humanChloridesCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftor, ivacaftor, tezacaftor drug combinationIndolesPyrazolesPyridinesPyrrolidinesQuinolinesQuinolones

Identifiers

PMID41238903

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.