Evidence map›Paper›PMID 41065216›Full record

Observational studyTherapeutic advances in respiratory disease

Identification of early changes in multiple biomarkers following CFTR modulator initiation in patients with cystic fibrosis.

Pascal Heer, Clara Fernandez Elviro, Angela Koutsokera, Anne Mornand, Isabelle Rochat, Nicolas Regamey, Sylvain Blanchon

Abstract readObservational StudyMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Therapeutic advances in respiratory disease. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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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

7 authors.

Pascal HeerPediatric Pulmonology and Cystic Fibrosis Unit, Children's Hospital of Central Switzerland, Lucerne, Switzerland.ORCID 0009-0008-3491-6363
Clara Fernandez ElviroPediatric Pulmonology and Cystic Fibrosis Unit, Division of Pediatrics, Department Woman-Mother-Child, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.
Angela KoutsokeraAdult Cystic Fibrosis and CFTR-related disorders Center, Lung Transplant Center, Division of Pulmonology, Lausanne University Hospital and University of Lausanne, Switzerland.ORCID 0000-0003-1227-1679
Anne MornandPediatric Pulmonology and Cystic Fibrosis Unit, University Hospital of Geneva, Geneva, Switzerland.ORCID 0000-0001-9398-5723
Isabelle RochatPediatric Pulmonology and Cystic Fibrosis Unit, Division of Pediatrics, Department Woman-Mother-Child, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.
Nicolas RegameyPediatric Pulmonology and Cystic Fibrosis Unit, Children's Hospital of Central Switzerland, Lucerne, Switzerland.ORCID 0000-0001-6849-6469
Sylvain BlanchonPediatric Pulmonology and Cystic Fibrosis Unit, Division of Pediatrics, Department Woman-Mother-Child, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.ORCID 0000-0003-0284-9705

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThere are currently no early parameters that allow prediction of long-term responses to Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator treatment on an individual level.

objectivesTo identify early parameters measured within 7 to 14 days after initiation of treatment with a CFTR modulator to assess CFTR modulator efficacy. STUDY

designProspective observational study of patients diagnosed with CF who begin elexacaftor/tezacaftor/ivacaftor (ETI) therapy at 3 CF clinics in Switzerland (Geneva, Lausanne, Lucerne).

methodsStandardized measurements were taken within 2 months prior to and 7 to 14 days after starting CFTR modulator treatment.

resultsETI treatment was started on 47 patients [median age: 12 years] of whom 12 (26%) were switching from lumacaftor/ivacaftor (

conclusionThis study identified clinical, biologic, and functional parameters showing treatment effect early after initiation of CFTR modulator therapy. These parameters may serve as potential predictors of long-term responses to CFTR modulator treatment.

Indexed as

AminophenolsAminopyridinesBenzodioxolesChloride Channel AgonistsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorIndolesPyrazolesQuinolonesAdolescentAdultBiomarkersChildDrug CombinationsFemaleHumansAminophenolsAminopyridinesBenzodioxolesBiomarkersCFTR protein, humanChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsIndoleslumacaftor, ivacaftor drug combinationPyrazolesPyrrolidinesQuinolonesCFTR modulator treatmentelexacaftorivacaftornasal potential differenceshort-term responsetezacaftor

Identifiers

PMID41065216
PMCPMC12515279

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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.