ArticlePediatric pulmonology2022
A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators.
Article in Pediatric pulmonology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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Who cites it
26 citing papers in PubMed, 34 citations in OpenAlex.
- Recalibration: Exploring the Impact of Elexacaftor/Tezacaftor/Ivacaftor on Self-Concept for Adults with Cystic Fibrosis.The patient · 2026Article
- Antiviral defenses are diminished at birth in cystic fibrosis pig airways.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Article
- Nucleic acid-based therapies for people with cystic fibrosis.The Cochrane database of systematic reviews · 2026Article
- Myeloperoxidase impairs mucociliary transport on human airway epithelium.Disease models & mechanisms · 2026Article
- Mental Health in Cystic Fibrosis in the Modulator Era: Epidemiology, Prognostic Significance, and Therapeutic Implications.Journal of clinical medicine · 2026Review
- A qualitative exploration of the experiences of adults with cystic fibrosis unable to benefit from triple combination therapy.Journal of health psychology · 2026Article
- Psychological impact of genetic eligibility for CFTR modulator therapy in children with cystic fibrosis and their parents.European journal of pediatrics · 2026Article
- Maximising opportunity for therapeutic success: sequential participation in cystic fibrosis nucleic acid-based therapy trials.The Lancet. Respiratory medicine · 2025Review
- Myeloperoxidase impairs mucociliary transport on human airway epithelium.bioRxiv : the preprint server for biology · 2025Article
- Hypercubes to identify geomarkers of rapid cystic fibrosis lung disease progression.BMC medical informatics and decision making · 2025Article
- Species turnover within cystic fibrosis lung microbiota is indicative of acute pulmonary exacerbation onset.Microbiome · 2025Article
- Elexacaftor/Tezacaftor/Ivacaftor Treatment Accessibility and Mental Health: Reducing Anxiety in People With Cystic Fibrosis.Pediatric pulmonology · 2025Article
- Psychosocial and mental health in cystic fibrosis in the modern era of care: time to evolve.BMJ open respiratory research · 2025Review
- Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation.Children (Basel, Switzerland) · 2025Article
- Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.American journal of respiratory and critical care medicine · 2024Review
- Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells.Nucleic acids research · 2024Article
- Cystic fibrosis.Nature reviews. Disease primers · 2024Review
- Increasing Diversity, Equity, Inclusion, and Accessibility in Rare Disease Clinical Trials.Pharmaceutical medicine · 2024Review
- Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy.The Lancet. Respiratory medicine · 2023Review
- Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective.The Lancet. Respiratory medicine · 2023Review
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundWhile the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use has improved daily life and long-term prognosis of CF for many with approved CFTR mutations, approximately 10% of people with CF (pwCF) have only symptomatic treatments available.
methodsBetween June 10 and July 1, 2021, Emily's Entourage distributed a 38-question anonymous survey targeted at pwCF not benefitting from approved modulators via social media and email to pwCF and CF advocacy groups in and outside the United States regarding health status, impact of CF, unmet needs, and clinical research interest.
resultsThere were 431 survey respondents representing pwCF on five continents. The majority of pwCF had moderate lung disease (50.3%). Ineligibility based on CFTR mutation (64.1%) was the most frequently reported reason pwCF were not on modulators. PwCF reported the most impacted aspects of life were mental (66.7%) and physical (40.7%) health. Financial concerns and feelings of isolation were commonly reported. Witnessing improvements for peers with access to modulators was both uplifting and disheartening. The majority of pwCF would be interested in participating in future clinical research (77.6%), although some living outside of the United States cited lack of opportunity to participate in clinical trials as a barrier.
conclusionsPwCF who are ineligible, intolerant, or lack access to modulators have a high burden of disease impacting their physical and mental health. Although most are happy for those who are benefiting from modulators, they are eager for the opportunity to experience similar improvements for themselves, and willing to participate in clinical trials of new therapies.
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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.