Evidence map›Paper›PMID 42825108›Full record

ArticleData in brief2026

A longitudinal multi-omic dataset of pediatric cystic fibrosis patients receiving lumacaftor/ivacaftor therapy: clinical, microbiome, inflammatory and metabolomic measurements collected over 24 months.

Rebecca Luise Knoll, Virginia Rossow, Juliane Rössler, Katja Hilbert, Víctor Hugo Jarquín-Díaz, Theda Ulrike Patricia Bartolomaeus, Oliver Nitsche, Morgan Essex, Ulrike Löber, Chen Meng and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Data in brief, 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
–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

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

14 authors.

Rebecca Luise KnollChildren's Hospital, University Medical Center of the Johannes Gutenberg University Mainz, Langenbeckstraße 1, 55131, Mainz, Germany.
Virginia RossowCharité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Juliane RösslerChildren's Hospital, University Medical Center of the Johannes Gutenberg University Mainz, Langenbeckstraße 1, 55131, Mainz, Germany.
Katja HilbertChildren's Hospital, University Medical Center of the Johannes Gutenberg University Mainz, Langenbeckstraße 1, 55131, Mainz, Germany.
Víctor Hugo Jarquín-DíazCharité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Theda Ulrike Patricia BartolomaeusCharité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Oliver NitscheChildren's Hospital, University Medical Center of the Johannes Gutenberg University Mainz, Langenbeckstraße 1, 55131, Mainz, Germany.
Morgan EssexCharité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Ulrike LöberCharité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Chen MengBavarian Center for Biomolecular Mass Spectrometry, TUM School of Life Sciences, Technical University of Munich (TUM), Gregor-Mendel-Straße 4, 85354, Freising, Germany.
Karin KleigreweBavarian Center for Biomolecular Mass Spectrometry, TUM School of Life Sciences, Technical University of Munich (TUM), Gregor-Mendel-Straße 4, 85354, Freising, Germany.
Stephan GehringChildren's Hospital, University Medical Center of the Johannes Gutenberg University Mainz, Langenbeckstraße 1, 55131, Mainz, Germany.
Sofia Kirke Forslund-StartcevaCharité - Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, Charitéplatz 1, 10117, Berlin, Germany.
Krystyna PoplawskaChildren's Hospital, University Medical Center of the Johannes Gutenberg University Mainz, Langenbeckstraße 1, 55131, Mainz, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

This article describes a longitudinal multiomic dataset generated within a prospective phase IV pilot study of eight children with cystic fibrosis homozygous for the F508del mutation who initiated lumacaftor/ivacaftor therapy. Participants were followed for up to 24 months with repeated collection of clinical metadata, anthropometric measurements, sweat chloride concentrations, lung function assessments, inflammatory markers, conventional microbiology results, stool samples, respiratory samples, and serum metabolomics. The resulting dataset links host phenotypes, microbiome composition, inflammatory parameters, and metabolomic measurements across multiple body sites and time points. Microbiome data were generated from stool, sputum and throat swab samples using 16S rRNA gene sequencing, while serum metabolomics was assessed using untargeted mass spectrometry. The dataset is publicly available through SRA, MassIVE and GitHub repositories and may support future studies of longitudinal host-microbiome interactions, biomarker discovery, methodological benchmarking and comparative analyses across CFTR modulator eras.

Indexed as

Deep phenotypingLumacaftor/ivacaftorMetabolomeMicrobiomePediatric cystic fibrosis

Identifiers

PMID42825108
PMCPMC13629212

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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.