Therapies Used by Children With Primary Ciliary Dyskinesia: A Natural History Study
Robert A. Gardner, Thomas W. Ferkol, Stephanie D. Davis, Margaret Rosenfeld, Scott D. Sagel, Sharon D. Dell, Carlos E. Milla, Lang Li, Feng‐Chang Lin, Kelli M. Sullivan, Maimoona A. Zariwala, Michael R. Knowles, Margaret W. Leigh,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.