Observational studyThe Journal of clinical endocrinology and metabolism2021
The Effects of Ivacaftor on Bone Density and Microarchitecture in Children and Adults with Cystic Fibrosis.
Observational study in The Journal of clinical endocrinology and metabolism, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 1 synthesis or guideline pooled it, 41 citations in OpenAlex.
- The 2025 European Cystic Fibrosis Society position statement on physical activity assessment in cystic fibrosis.European respiratory review : an official journal of the European Respiratory Society · 2025Guideline
- Bone Health in Young Individuals with Primary Ciliary Dyskinesia: Insights from a Comparison with Cystic Fibrosis and Healthy Controls.Hormone research in paediatrics · 2026Trial
- Impact of Genetics on Endocrine Manifestations of Cystic Fibrosis.Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists · 2026Review
- Evaluating the internal architecture of the postnatal human maxilla across the stages of tooth development and eruption.Surgical and radiologic anatomy : SRA · 2026Article
- Updates in Cystic Fibrosis Bone Disease in Adults.Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists · 2026Review
- Bone Structural Parameters in Adults with Cystic Fibrosis: Contribution of Adherence to the Mediterranean Diet.Journal of clinical medicine · 2026Article
- The endocrine complications of cystic fibrosis.Nature reviews. Endocrinology · 2026Review
- Relationship between impaired glucose metabolism and bone mineral density in patients with cystic fibrosis.European journal of pediatrics · 2026Article
- Adverse effects of vanzacaftor-tezacaftor-deutivacaftor in a cystic fibrosis population: A case series.Respiratory medicine case reports · 2026Article
- Cardiometabolic insights in cystic fibrosis and highly effective modulator therapies: a narrative review.Endocrine connections · 2025Review
- Cystic Fibrosis and CFTR Modulators: The Impact on Bone Density, Muscle Mass and Strength in Children and Young Adolescents.Children (Basel, Switzerland) · 2025Review
- Elexacaftor/Tezacaftor/Ivacaftor Effect on Bone Density and Body Composition: A Retrospective Analysis.Pediatric pulmonology · 2025Article
- Treatment with CFTR Modulators for Cystic Fibrosis: What a Pediatric Gastroenterologist Needs to Know.Children (Basel, Switzerland) · 2025Review
- Cystic fibrosis-related bone disease: an update on screening, diagnosis, and treatment.Therapeutic advances in endocrinology and metabolism · 2025Review
- Longitudinal changes in BMD in adults with cystic fibrosis.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2024Article
- Cystic fibrosis transmembrane conductance regulator in COPD: a role in respiratory epithelium and beyond.The European respiratory journal · 2023Review
- Cystic Fibrosis Bone Disease: The Interplay between CFTR Dysfunction and Chronic Inflammation.Biomolecules · 2023Review
- Effect of Trikafta on bone density, body composition and exercise capacity in CF: A pilot study.Pediatric pulmonology · 2023Article
- Endocrine Complications of Cystic Fibrosis.Clinics in chest medicine · 2022Review
- Prevalence and Risk Factors for Low Bone Mineral Density in Adults With Cystic Fibrosis.JBMR plus · 2022Article
Corrections and comments
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Authors and funding
12 authors at 3 institutions in 1 country.
Funding
Abstract
contextCystic fibrosis (CF) transmembrane conductance (CFTR) dysfunction may play a role in CF-related bone disease (CFBD). Ivacaftor is a CFTR potentiator effective in improving pulmonary and nutritional outcomes in patients with the G551D-CFTR mutation. The effects of ivacaftor on bone health are unknown.
objectiveTo determine the impact of ivacaftor on bone density and microarchitecture in children and adults with CF.
designProspective observational multiple cohort study.
settingOutpatient clinical research center within a tertiary academic medical center. PATIENTS OR OTHER
participantsThree cohorts of age-, race-, and gender-matched subjects were enrolled: 26 subjects (15 adults and 11 children) with CF and the G551D-CFTR mutation who were planning to start or had started treatment with ivacaftor within 3 months (Ivacaftor cohort), 26 subjects with CF were not treated with ivacaftor (CF Control cohort), and 26 healthy volunteers.
interventionsAll treatments, including Ivacaftor, were managed by the subjects' pulmonologists.
main outcome measuresBone microarchitecture by high-resolution peripheral quantitative computed tomography (HR-pQCT), areal bone mineral density (aBMD) by dual-energy X-ray absorptiometry (DXA) and bone turnover markers at baseline, 1, and 2 years.
resultsCortical volume, area, and porosity at the radius and tibia increased significantly in adults in the Ivacaftor cohort. No significant differences were observed in changes in aBMD, trabecular microarchitecture, or estimated bone strength in adults or in any outcome measures in children.
conclusionsTreatment with ivacaftor was associated with increases in cortical microarchitecture in adults with CF. Further studies are needed to understand the implications of these findings.
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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.