ArticlePediatrics2017
Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
Article in Pediatrics, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 2 of them syntheses 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
26 citing papers in PubMed, 2 syntheses or guidelines pooled it, 59 citations in OpenAlex.
- 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
- Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial.Annals of the American Thoracic Society · 2021Trial
- An EHR-based framework for modeling growth curves and constructing growth centile charts for genetic disorders.NPJ genomic medicine · 2026Article
- The endocrine complications of cystic fibrosis.Nature reviews. Endocrinology · 2026Review
- Evaluating Long-Term Effectiveness of Cystic Fibrosis Modulator Therapies after Rapid Adoption: A Dual-Approach Study.Annals of the American Thoracic Society · 2026Article
- Article
- Elexacaftor-tezacaftor-ivacaftor pharmacokinetics with concurrent tacrolimus administration after lung transplant.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025Observational
- Endocrine Complications of Cystic Fibrosis.Clinics in chest medicine · 2022Review
- Use of elexacaftor/tezacaftor/ivacaftor among cystic fibrosis lung transplant recipients.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022Article
- Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children.Children (Basel, Switzerland) · 2022Review
- Auxological and Endocrinological Features in Children and Adolescents with Cystic Fibrosis.Journal of clinical medicine · 2022Review
- The Changing Landscape of Nutrition in Cystic Fibrosis: The Emergence of Overweight and Obesity.Nutrients · 2022Review
- Trends in Growth and Maturation in Children with Cystic Fibrosis Throughout Nine Decades.Frontiers in endocrinology · 2022Review
- Article
- Review
- Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021Observational
- Human Growth and Growth Hormone: From Antiquity to the Recominant Age to the Future.Frontiers in endocrinology · 2021Review
- Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications.American journal of respiratory and critical care medicine · 2020Review
- Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease.European respiratory review : an official journal of the European Respiratory Society · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 6 institutions in 2 countries.
Funding
Abstract
BACKGROUND AND
objectivesCystic fibrosis (CF) is known for its impact on the lung and pancreas of individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may affect growth outcomes.
methodsIn this post hoc analysis, we assessed linear growth and weight in 83 children (aged 6-11 years) enrolled in 2 clinical trials, the longitudinal-observation GOAL study and the placebo-controlled ENVISION study, to evaluate the effects of ivacaftor, a CFTR potentiator. We calculated height and weight z scores and height and weight growth velocities (GVs).
resultsIn ivacaftor-treated children in GOAL, height and weight z scores increased significantly from baseline to 6 months (increases of 0.1 [P < .05] and 0.26 [P < .0001], respectively); height GV increased significantly from 3 to 6 months (2.10-cm/year increase; P < .01). In ivacaftor-treated children in ENVISION, height and weight z scores increased significantly from baseline to 48 weeks (increases of 0.17 [P < .001] and 0.35 [P < .001], respectively). Height and weight GVs from baseline to 48 weeks were also significantly higher with ivacaftor than with placebo (differences of 1.08 cm/year [P < .05] and 3.11 kg/year [P < .001], respectively).
conclusionsIvacaftor treatment in prepubescent children may help to address short stature and altered GV in children with CF; results from these analyses support the existence of an intrinsic defect in the growth of children with CF that may be ameliorated by CFTR modulation.
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.