Evidence map›Paper›PMID 37958017›Full record

ArticleHealthcare (Basel, Switzerland)2023

Long-Term Follow-Up of Health-Related Quality of Life and Short-Term Intervention with CFTR Modulator Therapy in Adults with Cystic Fibrosis: Evaluation of Changes over Several Years with or without 33 Weeks of CFTR Modulator Therapy.

Wolfgang Gruber, Matthias Welsner, Christopher Blosch, Stefanie Dillenhoefer, Margarete Olivier, Folke Brinkmann, Cordula Koerner-Rettberg, Sivagurunathan Sutharsan, Uwe Mellies, Christian Taube and 1 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Healthcare (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
2.3field-weighted citation impact, top 11% of its field
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

6 citing papers in PubMed, 1 synthesis or guideline pooled it, 9 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Observational
  6. Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.American journal of respiratory and critical care medicine · 2024
    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

11 authors at 5 institutions in 1 country.

Wolfgang GruberPediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.ORCID 0000-0003-2186-9269
Matthias WelsnerDepartment of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, 45239 Essen, Germany.ORCID 0000-0003-1259-8911
Christopher BloschPediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.
Stefanie DillenhoeferDepartment of Pediatric Pulmonology, University Children's Hospital, Ruhr University Bochum, 44801 Bochum, Germany.
Margarete OlivierPediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.
Folke BrinkmannDepartment of Pediatric Pulmonology, University Children's Hospital, Ruhr University Bochum, 44801 Bochum, Germany.
Cordula Koerner-RettbergChildren's Hospital, Marienhospital Wesel, 46483 Wesel, Germany.
Sivagurunathan SutharsanDepartment of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, 45239 Essen, Germany.
Uwe MelliesPediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.
Christian TaubeDepartment of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, 45239 Essen, Germany.
Florian StehlingPediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.
University of Duisburg-Essen · DERuhrlandklinik · DEUniversity Hospitals of the Ruhr-University of Bochum · DEChristian-Albrechts-Universität zu Kiel · DEMarien Hospital Wesel · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundLongitudinal data on changes in health-related quality of life (HRQoL) in adult people with cystic fibrosis (pwCF) and the longitudinal effects of Elexacaftor/Tezacaftor/Ivacaftor therapy (ETI) on HRQoL or HRQoL domains are currently scarce. This study aimed to investigate the effects of ETI on HRQoL and compare them with those of pwCF who did not receive highly effective CFTR modulators over a longer period.

methodsBaseline assessment and follow-up data for 5.6 years in pwCF with (

resultsETI was found to improve all HRQoL domains at more than four points over time, and their increases were significant except for vitality, digestion, treatment burden, and social functioning (

conclusionsThe results of the present study show that ETI therapy has a positive effect on HRQoL and clinical outcomes over time but not in pwCF without ETI treatment. Furthermore, our results suggest that disease progression over time affects the physical domains of HRQoL more than the psychosocial domains. Due to the small sample size and the heterogeneity of the study population (CFTR mutation genotype), the results should be interpreted with some caution.

Indexed as

adultscystic fibrosisElexacaftor/Tezacaftor/Ivacaftorhealth-related quality of lifelongitudinal changes

Identifiers

PMID37958017
PMCPMC10647537
OpenAlexW4388040553

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.