Observational studyJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2017
The effects of ivacaftor on CF fatty acid metabolism: An analysis from the GOAL study.
Observational study in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 19 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.
Nutritional Impact of Ivacaftor Treatment in 4 Month to 2 Year Old Children With CF Gating Mutations
Nutritional Impact of Orkambi Treatment in 2 to 5 Year Old Children Homozygous for F508del Mutations
Who cites it
19 citing papers in PubMed, 23 citations in OpenAlex.
- Understanding Nasal Polyposis: The Roles of Ion Channels, Inflammation, Ionocytes, and Prostaglandin E2-The IJournal of clinical medicine · 2026Article
- Cystic fibrosis and inflammatory bowel disease: parallels in gut physiology and microbiota.Journal of bacteriology · 2025Review
- Review
- Dysregulation of the Arachidonic Acid Pathway in Cystic Fibrosis: Implications for Chronic Inflammation and Disease Progression.Pharmaceuticals (Basel, Switzerland) · 2024Review
- The Omega-6 Lipid pathway shift is associated with neutrophil influx and structural lung damage in early cystic fibrosis lung disease.Clinical & translational immunology · 2024Article
- Impact of lumacaftor/ivacaftor on nutrition and growth in modulator-naïve children over 24 weeks.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024Article
- Elevated Prostaglandin EJournal of clinical medicine · 2024Article
- Changes in nutrition and growth status in young children in the first 12 weeks of ivacaftor therapy.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023Article
- The fatty acid imbalance of cystic fibrosis exists at birth independent of feeding in pig and ferret models.Clinical science (London, England : 1979) · 2022Article
- Essential Fatty Acid Deficiency in Cystic Fibrosis Disease Progression: Role of Genotype and Sex.Nutrients · 2022Review
- Impact ofInternational journal of molecular sciences · 2022Review
- Review
- Role for animal models in understanding essential fatty acid deficiency in cystic fibrosis.Cellular and molecular life sciences : CMLS · 2021Review
- Is the ENaC Dysregulation in CF an Effect of Protein-Lipid Interaction in the Membranes?International journal of molecular sciences · 2021Review
- Airway Inflammation and Host Responses in the Era of CFTR Modulators.International journal of molecular sciences · 2020Review
- Metabolomics profiling of tobacco exposure in children with cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2020Article
- The Extrapulmonary Effects of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis.Annals of the American Thoracic Society · 2020Review
- Secondhand smoke alters arachidonic acid metabolism and inflammation in infants and children with cystic fibrosis.Thorax · 2019Article
- CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis.Frontiers in pharmacology · 2016Review
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
backgroundIvacaftor has produced significant improvement in certain individuals with cystic fibrosis (CF), though the full metabolic effects of treatment remain unknown. Abnormalities in fatty acid metabolism have previously been shown to be a characteristic of CFTR dysfunction. We hypothesized that as a reflection of this clinical improvement, ivacaftor would improve plasma fatty acid levels and decrease urine prostaglandin E metabolite levels.
methodsThis study analyzed plasma fatty acid levels and urine prostaglandin E metabolites (PGE-M) in 40 subjects with CF participating in the G551D observational (GOAL) study who demonstrated response to the medication by a significant decrease in sweat Cl levels. Paired samples were analyzed before and after 6months of ivacaftor treatment.
resultsLinoleic acid and docosahexaenoic acid levels, which are typically low in individuals with CF, did not significantly increase with ivacaftor treatment. However, arachidonic acid levels did decrease with ivacaftor treatment and there was a significant decrease in the arachidonic acid metabolite PGE-M as measured in the urine [median: before treatment 17.03ng/mg Cr; after treatment 9.06ng/mg Cr; p<0.001]. Furthermore, there were fatty acid age differences observed, including pediatric participants having significantly greater linoleic acid levels at baseline.
conclusionIvacaftor reduces inflammatory PGE without fully correcting the plasma fatty acid abnormalities of CF. Age-related differences in fatty acid levels were observed, that may be a result of other clinical factors, such as diet, clinical care, or drug response.
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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.