Trial reportDigestive diseases and sciences2016
Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor.
Trial report in Digestive diseases and sciences, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03859531 (Gastrointestinal Outcome Measures Before and After Orkambi Therapy in Cystic Fibrosis), which is not on this map. Cited by 32 papers, 5 of them syntheses that pooled it.
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.
Gastrointestinal Outcome Measures Before and After Orkambi Therapy in Cystic Fibrosis (CF) Patients Carrying the F508del Mutation on Both Alleles
Who cites it
32 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- Evolution of body image across treatment eras: a systematic review in young people and adults living with cystic fibrosis.European respiratory review : an official journal of the European Respiratory Society · 2026Pooled it
- Brazilian guidelines for the pharmacological treatment of the pulmonary symptoms of cystic fibrosis. Official document of the Sociedade Brasileira de Pneumologia e Tisiologia (SBPT, Brazilian Thoracic Association).Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2023Guideline
- Altered intravenous drug disposition in people living with cystic fibrosis: A meta-analysis integrating top-down and bottom-up data.CPT: pharmacometrics & systems pharmacology · 2022Pooled it
- Academy of Nutrition and Dietetics: 2020 Cystic Fibrosis Evidence Analysis Center Evidence-Based Nutrition Practice Guideline.Journal of the Academy of Nutrition and Dietetics · 2021Guideline
- Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis.The Cochrane database of systematic reviews · 2019Pooled it
- Trial
- Assessing Dietary Patterns and Composition Among Adults With Cystic Fibrosis Taking Highly Effective Modulator Therapy.Pediatric pulmonology · 2026Article
- Beyond BMI: Nutritional Recovery and Functional Implications of CFTR Modulators in Cystic Fibrosis.Biology · 2026Review
- Increased fat mass and obesity risk after elexacaftor-tezacaftor-ivacaftor therapy in young adults with cystic fibrosis.Frontiers in nutrition · 2024Article
- 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
- Nutritional Care in Children with Cystic Fibrosis.Nutrients · 2023Review
- Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass.Frontiers in pharmacology · 2023Article
- A randomised crossover trial of tezacaftor-ivacaftor for gut dysfunction in cystic fibrosis with magnetic resonance imaging (MRI) outcomes: a pilot study.NIHR open research · 2023Article
- Dietary intake assessment in children with cystic fibrosis using 3-day food diaries: a single-centre study.Frontiers in pediatrics · 2023Article
- The Changing Landscape of Nutrition in Cystic Fibrosis: The Emergence of Overweight and Obesity.Nutrients · 2022Review
- Effect of elexacaftor-tezacaftor-ivacaftor on body weight and metabolic parameters in adults with cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022Observational
- Utilization of the Healthy Eating Index in Cystic Fibrosis.Nutrients · 2022Article
- Obesity in cystic fibrosis.Journal of clinical & translational endocrinology · 2021Article
- Clinical Pharmacokinetic and Pharmacodynamic Considerations in the Drug Treatment of Non-Tuberculous Mycobacteria in Cystic Fibrosis.Clinical pharmacokinetics · 2021Review
- The Intestinal Microbiome and Cystic Fibrosis Transmembrane Conductance Regulator Modulators: Emerging Themes in the Management of Gastrointestinal Manifestations of Cystic Fibrosis.Current gastroenterology reports · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability.
aimTo further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged ≥6 years with CF and the G551D mutation.
methodsPatients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks. Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R).
resultsStudies included 213 patients (aged ≤ 20 years, n = 105; aged > 20 years, n = 108). In patients ≤20 years, adjusted mean change from baseline to week 48 in body weight was 4.9 versus 2.2 kg (ivacaftor vs. placebo, p = 0.0008). At week 48, change from baseline in mean weight-for-age z-score was 0.29 versus -0.06 (p < 0.0001); change in mean BMI-for-age z-score was 0.26 versus -0.13 (p < 0.0001). In patients >20 years, adjusted mean change from baseline to week 48 in body weight was 2.7 versus -0.2 kg (p = 0.0003). Mean BMI change at week 48 was 0.9 versus -0.1 kg/m(2) (p = 0.0003). There was no linear correlation evident between changes in body weight and improvements in lung function or sweat chloride. Significant CFQ-R improvements were seen in perception of eating, body image, and sense of ability to gain weight.
conclusionsNutritional status improved following treatment with ivacaftor for 48 weeks.
Indexed as
Identifiers
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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.