Observational studyPediatric pulmonology2025
Therapies Used by Children With Primary Ciliary Dyskinesia: A Natural History Study.
Observational study in Pediatric pulmonology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 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
6 citing papers in PubMed.
- Pulmonary exacerbations in primary ciliary dyskinesia.European journal of pediatrics · 2026Review
- Airway Clearance and Pediatric Pulmonary Rehabilitation in Primary Ciliary Dyskinesia: A Clinical Framework for Children and Adolescents.Journal of clinical medicine · 2026Review
- Advances in Health-Related Quality of Life in Primary Ciliary Dyskinesia.Pediatric pulmonology · 2026Review
- Early Life Disease Burden and Outcomes in Children Diagnosed With Primary Ciliary Dyskinesia in Infancy.Pediatric pulmonology · 2026Article
- Mucociliary and cough clearance in primary ciliary dyskinesia as affected by mutations inERJ open research · 2026Article
- Beyond the present: current and future perspectives on the role of infections in pediatric PCD.Frontiers in pediatrics · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
Abstract
introductionPrimary ciliary dyskinesia (PCD) management has not been systematically evaluated and is largely empirical.
methodsPediatric participants with PCD were enrolled in a prospective, longitudinal, multicenter, observational study. Therapies were recorded at annual visits and categorized by type. Age-related trends in prevalence of therapies were described by serial cross-sectional analyses. Generalized estimating equations analyzed covariates affecting prevalence of certain therapies and whether these covariates impacted oral antibiotic courses.
resultsA total of 137 participants completed 897 visits over 13 years. All but one received ≥ 1 antibiotic courses during study participation, most often cephalosporins (74%) or amoxicillin-clavulanate (73%). Thirty-one percent reported chronic azithromycin use. Per participant, there was an average of 2.3 (SD = 2.2) oral antibiotic courses annually. The rate of reported antibiotic courses at the 6 United States sites was 2.6 times higher compared to the Canadian site (p < 0.001). As patients got older, they were more likely to report use of amoxicillin-clavulanate (p < 0.001), chronic azithromycin (p < 0.001), fluroquinolones (p < 0.001), inhaled steroids with long-acting beta-agonists (p = 0.010), and hypertonic saline (p < 0.001). Compared to outer dynein arm defects, those with inner dynein arm/microtubular disorganization defects reported increased use of chronic azithromycin (p = 0.011) and inhaled steroids (p = 0.015). DISCUSSION: Older participants and those with inner dynein arm/microtubular disorganization defects reported more therapies likely due to disease progression and more severe phenotypes, respectively. We report that a wide range of therapies are used in PCD without disease-specific studies defining benefits and risks.
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.