ReviewJournal of clinical medicine2020
Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review.
Review in Journal of clinical medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 60 papers, 1 of them a synthesis that pooled 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.
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
60 citing papers in PubMed, 1 synthesis or guideline pooled it, 123 citations in OpenAlex.
- Cystic Fibrosis Foundation Evidence-Based Guideline for the Management of CRMS/CFSPID.Pediatrics · 2024Guideline
- Evolving Cystic Fibrosis Therapy: The Good, the Sad, and the Hopeful.Children (Basel, Switzerland) · 2026Review
- Inhibition of the QPCT-PDIA4 axis rescues ΔF508 and N1303K CFTR in cystic fibrosis.Nature communications · 2026Article
- A qualitative exploration of the experiences of adults with cystic fibrosis unable to benefit from triple combination therapy.Journal of health psychology · 2026Article
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- Exploring associations and indirect effects between mindfulness, psychological flexibility and wellbeing in adults with cystic fibrosis: Informing future interventions.Chronic illness · 2026Article
- The clinical, psychosocial, and economic burden of cystic fibrosis lung disease in the era of CFTR modulator therapy.Annals of the American Thoracic Society · 2026Review
- Transforming Care Models in Cystic Fibrosis: A Review.Healthcare (Basel, Switzerland) · 2025Review
- Comprehensive Evaluation of Elexacaftor/Tezacaftor/Ivacaftor in Paediatric Cystic Fibrosis: Nutritional, Pulmonary, and Quality-of-Life Outcomes.Journal of clinical medicine · 2025Article
- Adverse Events and Drug Interactions Associated with Elexacaftor/Tezacaftor/Ivacaftor Treatment: A Descriptive Study Across Australian, Canadian, and American Adverse Event Databases.Life (Basel, Switzerland) · 2025Article
- The urban physical environment and leisure-time physical activity in early midlife: a FinnTwin12 study.Health & place · 2025Article
- VX-445 (elexacaftor) inhibits chloride secretion across human bronchial epithelial cells by directly blocking KCa3.1 channels.PNAS nexus · 2025Article
- The Aging Patient with Cystic Fibrosis.Drugs & aging · 2025Review
- Uncomplicated SARS-CoV-2 Infections with Preserved Lung Function in Pediatric Patients with Cystic Fibrosis: A Three-Year Single-Centre Experience.Journal of clinical medicine · 2025Article
- Development and validation of a short form psychometric tool assessing the caregiving Challenge of Living with Cystic Fibrosis (CLCF-SF) in a child.Psychology & health · 2025Article
- A Review of Exocrine Pancreatic Insufficiency in Children beyond Cystic Fibrosis and the Role of Endoscopic Direct Pancreatic Function Testing.Current gastroenterology reports · 2025Review
- Durable reconstitution of sinonasal epithelium by transplant of CFTR gene corrected airway stem cells.bioRxiv : the preprint server for biology · 2025Article
- Article
- Effect of Cystic Fibrosis Transmembrane Conductance Regulator Modulators on Liver Enzymes Among Patients With Cystic Fibrosis: A Systematic Review and Meta-Analysis.Gastro hep advances · 2025Review
- Real-world safety of Symdeko: insights from the food and drug administration adverse event reporting system.Frontiers in medicine · 2025Article
Corrections and comments
- Erratum issued
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies target the underlying cause of cystic fibrosis (CF), and are generally well-tolerated; however, real-world studies indicate the frequency of discontinuation and adverse events (AEs) may be higher than what was observed in clinical trials. The objectives of this systematic review were to summarize real-world AEs reported for market-available CFTR modulators (i.e., ivacaftor (IVA), lumacaftor/ivacaftor (LUM/IVA), tezacaftor/ivacaftor (TEZ/IVA), and elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)), and to identify ways in which the pharmacist on CF healthcare teams may contribute to mitigating and managing these AEs. The MEDLINE, EMBASE, CINAHL, and Web of Science Core Collection online databases were searched from 2012 to Aug 1, 2020. Full manuscripts or conference abstracts of observational studies, case series, and case reports were eligible for inclusion. The included full manuscripts and conference abstracts comprised of 54 observational studies, 5 case series, and 9 case reports. The types of AEs reported generally aligned with what have been observed in clinical trials. LUM/IVA was associated with a higher frequency of respiratory-related AE and discontinuation in real-world studies. A signal for mental health and neurocognitive AEs was identified with all 4 CFTR modulators. A systematic approach to monitoring for AEs in people with CF on CFTR modulators in the real-world setting is necessary to help better understand potential AEs, as well as patient characteristics that may be associated with higher risk of certain AEs. Pharmacists play a key role in the safe initiation and monitoring of people with CF on CFTR modulator therapies.
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Identifiers
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.