Therapies Used by Children With Primary Ciliary Dyskinesia: A Natural History Study
Bibliographic record
Abstract
INTRODUCTION: Primary ciliary dyskinesia (PCD) management has not been systematically evaluated and is largely empirical. METHODS: Pediatric 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. RESULTS: A 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.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".