{"doi":"10.1002/ppul.27412","title":"Therapies Used by Children With Primary Ciliary Dyskinesia: A Natural History Study","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.","journal":"Pediatric Pulmonology","year":2024,"id":447913,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":6,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9276,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2024-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":539053,"name":"Thomas Ferkol","orcid":null,"position":1,"is_corresponding":false},{"id":442437,"name":"Stephanie D. Davis","orcid":"0000-0002-9390-0651","position":2,"is_corresponding":false},{"id":393553,"name":"Margaret Rosenfeld","orcid":"0000-0002-8105-614X","position":3,"is_corresponding":false},{"id":317016,"name":"Scott D. Sagel","orcid":"0000-0001-6172-4465","position":4,"is_corresponding":false},{"id":482851,"name":"Sharon Dell","orcid":"0000-0003-2169-9407","position":5,"is_corresponding":false},{"id":264757,"name":"Carlos Milla","orcid":"0000-0001-5515-3053","position":6,"is_corresponding":false},{"id":1099661,"name":"Lang Li","orcid":"0000-0002-6479-5952","position":7,"is_corresponding":false},{"id":381755,"name":"Feng‐Chang Lin","orcid":"0000-0002-2638-1775","position":8,"is_corresponding":false},{"id":538065,"name":"Kelli M. Sullivan","orcid":"0000-0001-8564-4726","position":9,"is_corresponding":false},{"id":302579,"name":"Maimoona A. Zariwala","orcid":"0000-0003-1619-1393","position":10,"is_corresponding":false},{"id":302583,"name":"Michael R. Knowles","orcid":"0000-0002-4672-5969","position":11,"is_corresponding":false},{"id":393545,"name":"Margaret W. Leigh","orcid":"0000-0002-6420-2459","position":12,"is_corresponding":false},{"id":1149263,"name":"Robert A. Gardner","orcid":"0000-0002-9123-0762","position":0,"is_corresponding":true}],"reference_count":29,"raw_metadata":null,"created_at":"2026-07-19T02:02:03.354708Z","pmid":"39575633","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}