ReviewJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2021
Challenges in the use of highly effective modulator treatment for cystic fibrosis.
Review in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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
20 citing papers in PubMed.
- An Observational Cohort Study of Targeted Antibiotic Treatments of Cystic Fibrosis Exacerbations.Open forum infectious diseases · 2026Trial
- Maternal and fetal outcomes associated with CFTR modulator use during pregnancy: a scoping review.European respiratory review : an official journal of the European Respiratory Society · 2026Article
- Evolving Cystic Fibrosis Therapy: The Good, the Sad, and the Hopeful.Children (Basel, Switzerland) · 2026Review
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- Role of elexacaftor-tezacaftor-ivacaftor therapy on fecal elastase-1 and fat-soluble vitamins in children with cystic fibrosis.European journal of pediatrics · 2026Article
- The bacterial serine protease inhibitor ecotin inhibits neutrophil elastase enzymatic activity in cystic fibrosis sputa.Heliyon · 2024Article
- The ageing of people living with cystic fibrosis: what to expect now?European respiratory review : an official journal of the European Respiratory Society · 2024Review
- New drugs, new challenges in cystic fibrosis care.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- Neutrophil serine proteases in cystic fibrosis: role in disease pathogenesis and rationale as a therapeutic target.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- Persistence and evolution ofmBio · 2024Article
- Delivering a New Future for People With Cystic Fibrosis.Pediatrics · 2023Article
- Lung Transplantation in a New Era in the Field of Cystic Fibrosis.Life (Basel, Switzerland) · 2023Review
- Article
- Nutritional Status and Circulating Levels of Fat-Soluble Vitamins in Cystic Fibrosis Patients: A Cohort Study and Evaluation of the Effect of CFTR Modulators.Children (Basel, Switzerland) · 2023Article
- Impact of Elexacaftor/Tezacaftor/Ivacaftor Therapy on the Cystic Fibrosis Airway Microbial Metagenome.Microbiology spectrum · 2022Article
- Article
- Elexacaftor/Tezacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for theJournal of clinical medicine · 2022Article
- Translational Research in Cystic Fibrosis: From Bench to Beside.Frontiers in pediatrics · 2022Review
- CFTR Modulator Therapy and Its Impact on Lung Transplantation in Cystic Fibrosis.Pulmonary therapy · 2021Review
- Novel CFTR Modulators for Treating Cystic Fibrosis.ACS medicinal chemistry letters · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
The last decade has seen development of oral, small molecule therapies that address the basic cystic fibrosis transmembrane conductance regulator (CFTR) protein defect. Highly effective modulator treatment (HEMT) that is efficacious for a large majority of people living with cystic fibrosis (CF) promises to change the landscape of this chronic life-limiting disease. Some people living with CF have a CFTR genotype that renders them eligible for HEMT, but also have comorbidities that excluded them from the original Phase III clinical trials that led to US Food and Drug Administration approval. The purpose of this review is to address the use of HEMT in challenging situations, including initiation for those with advanced CF lung disease, and use after solid organ transplant, during pregnancy, and for individuals with CFTR-related disorders without a definitive diagnosis of CF.
Indexed as
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.