Observational studyJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2022
Disease burden in people with cystic fibrosis heterozygous for F508del and a minimal function mutation.
Observational study in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
What it found
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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.
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.
Who cites it
8 citing papers in PubMed, 9 citations in OpenAlex.
- Elexacaftor/tezacaftor/ivacaftor is associated with long-term reduction in use of chronic respiratory therapies in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Observational
- Height Velocity in Pediatric Cystic Fibrosis Under Triple CFTR Modulator Therapy: A Real-Life Monocentric Experience.Journal of clinical medicine · 2025Article
- Improved detection of cystic fibrosis by the California Newborn Screening Program for all races and ethnicities.Pediatric pulmonology · 2024Article
- TMEM16 proteins: CaInternational journal of molecular medicine · 2024Review
- Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.Thorax · 2024Article
- Ethnic differences in staphylococcus aureus acquisition in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023Article
- The changing landscape of the cystic fibrosis lung environment: From the perspective of Pseudomonas aeruginosa.Current opinion in pharmacology · 2022Review
- Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis.Paediatric respiratory reviews · 2022Review
Corrections and comments
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Authors and funding
7 authors at 5 institutions in 1 country.
Funding
Abstract
backgroundPeople with cystic fibrosis (CF) heterozygous for F508del-CFTR and a minimal function CFTR mutation (F/MF) that results in no CFTR protein or results in CFTR protein that is not responsive to tezacaftor, ivacaftor, and tezacaftor/ivacaftor in vitro comprise a sizeable percentage of the US CF population. This retrospective, cross-sectional, observational study aimed to characterize CF burden in this subpopulation.
methodsPeople ≥2 years of age in the US CF Foundation Patient Registry with a CF diagnosis, F/MF genotype, and ≥1 encounters in 2017 were included. Descriptive analyses assessed lung function, nutritional parameters, microbiology, hospitalization and pulmonary exacerbation rates, and CF-related complications. Results were stratified by age group; select characteristics were summarized by percent predicted FEV
results5348 people met inclusion criteria. Rates of positive bacterial cultures, pulmonary exacerbations, and hospitalizations were generally higher in older age groups. Prevalence of prescribed symptomatic CF therapies was substantial and also generally higher in older age groups. ppFEV
conclusionsPeople with F/MF genotypes have substantial disease burden that worsened in older age groups consistent with the progressive nature of CF, indicating need for additional treatment options in this subpopulation.
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What OpenQuestion holds
Registered trials
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.