{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T05:19:45Z","timestamp":1788239985358,"version":"build-2803163510"},"reference-count":59,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2026,8,12]],"date-time":"2026-08-12T00:00:00Z","timestamp":1786492800000},"content-version":"vor","delay-in-days":11,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T00:00:00Z","timestamp":1785542400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2026,8]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Primary ciliary dyskinesia (PCD) is a rare, genetic disorder characterized by impaired mucociliary clearance and progressive sino\u2010pulmonary disease. Individuals have chronic, progressive chest symptoms throughout life with wet cough, frequent exacerbations and bronchiectasis. Traditional measures such as lung function and imaging do not capture the impact of PCD from the patient's perspective. This article examined health\u2010related quality of life (HRQoL) measures and their applications in PCD.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      Published literature on HRQoL in children, adolescents and adults with PCD was reviewed. Several databases were searched, including PubMed, PsychInfo, Embase, Elsevier's Scopus and\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/ClinicalTrials.gov\">ClinicalTrials.gov<\/jats:ext-link>\n                      to identify measures of HRQoL and their outcomes. The QOL\u2010PCD is the only condition\u2010specific HRQoL measure developed using FDA guidelines. Associations between QOL\u2010PCD scores and physical, mental health, and clinical trial outcomes were evaluated.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Substantial progress has been made in developing reliable and valid HRQoL measures for PCD, revealing how PCD affects the progression of pulmonary, sinus, oto\u2010rhinologic symptoms, and treatment burden across the lifespan. Although upper and lower respiratory symptoms worsen with time, ear and hearing symptoms\n                      <jats:italic>improve<\/jats:italic>\n                      moderately in adolescence. Disease severity increases in adulthood as evidenced by lower scores across most HRQoL domains. Strong associations were found between QOL\u2010PCD scores and physical, psychological, and social functioning, however, its sensitivity to change in clinical trials has not been demonstrated. Additional research is needed to identify the minimal important difference score on the QOL\u2010PCD to better interpret meaningful changes in interventional trials.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The QOL\u2010PCD was developed using the FDA framework that seeks patient input across all phases of development, including qualitative interviews, cognitive testing, and psychometric validation. Including these tools in clinical care, PCD registries and as outcomes in clinical trials is recommended. Longitudinal trajectories of HRQoL, linking genotypes to HRQoL outcomes, and evaluation of interventions targeting chronic cough, ENT symptoms, treatment burden and psychological functioning are needed. Embedding QOL\u2010PCD measures in trials ensures that new therapies reflect what matters most to patients and families.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ppul.71785","type":"journal-article","created":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T06:56:39Z","timestamp":1786604199000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Advances in Health\u2010Related Quality of Life in Primary Ciliary Dyskinesia"],"prefix":"10.1002","volume":"61","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4139-6983","authenticated-orcid":false,"given":"Alexandra L.","family":"Quittner","sequence":"first","affiliation":[{"name":"Joe DiMaggio Cystic Fibrosis, Pulmonary and Sleep Center Hollywood Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-8897-1009","authenticated-orcid":false,"given":"Ivan 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