ArticlePediatric pulmonology2019
Validation of pediatric health-related quality of life instruments for primary ciliary dyskinesia (QOL-PCD).
Article in Pediatric pulmonology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 2 of them syntheses that pooled it.
What it found
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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.
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Who cites it
23 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Self-reported perspective in rare genetic diseases: a systematic review of patient-reported outcome measures classified using the international classification of functioning, disability and health framework.Journal of patient-reported outcomes · 2026Pooled it
- Content Validity of Patient-Reported Outcome Measures Developed for Assessing Disease-Specific Quality of Life in Children With Sinonasal Disease: A Systematic Review.International forum of allergy & rhinology · 2025Pooled it
- Active video gaming in primary ciliary dyskinesia: a randomized controlled trial.European journal of pediatrics · 2022Trial
- 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
- Structure and function imaging in primary ciliary dyskinesia: cross-sectional study withERJ open research · 2026Article
- Health-related quality of life experiences in children with bladder exstrophy-epispadias complex: a Swedish focus group study.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- The Relationship Between Structural and Functional Pulmonary Status and Quality of Life in Children With Primary Ciliary Dyskinesia.Pediatric pulmonology · 2026Article
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- Validation of Polish-Language Questionnaires for Assessing the Quality of Life of Patients with Primary Ciliary Dyskinesia (PCD-QOL).Advances in respiratory medicine · 2024Article
- A BEAT-PCD consensus statement: a core outcome set for pulmonary disease interventions in primary ciliary dyskinesia.ERJ open research · 2024Article
- Patient-reported outcome measures in children, adolescents, and young adults with palliative care needs-a scoping review.BMC palliative care · 2023Article
- Racial disparities in primary ciliary dyskinesia: Defining the problem and potential solutions.Pediatric pulmonology · 2023Article
- Translation of the quality of life questionnaire for primary ciliary dyskinesia (QOL-PCD) into Hebrew: The Israeli experience.Pediatric pulmonology · 2022Article
- Respiratory symptoms of Swiss people with primary ciliary dyskinesia.ERJ open research · 2022Article
- Observational study of health utilities in adult primary ciliary dyskinesia patients: preliminary data on associations with molecular diagnosis, clinical phenotype and HRQOL measures.Multidisciplinary respiratory medicine · 2022Article
- Study protocol: the ear-nose-throat (ENT) prospective international cohort of patients with primary ciliary dyskinesia (EPIC-PCD).BMJ open · 2021Article
- Anxiety and depression in Dutch patients with primary ciliary dyskinesia and their caregivers: associations with health-related quality of life.ERJ open research · 2021Article
- The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment.The patient · 2021Review
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
rationaleHaving developed the first disease-specific, health-related quality of life (HRQoL) instruments for children with primary ciliary dyskinesia (PCD), we aimed to assess the psychometric performance of quality of life (QOL)-PCD child, adolescent, and parent-proxy versions in terms of reliability and validity across cross-cultural settings and caring for patients with this rare disease.
methodsChildren (n = 71), adolescents (n = 85), and parents (n = 68) from multiple centers in the UK and North America completed age-appropriate QOL-PCD and generic QOL measures: pediatric QOL inventory, COPD assessment test (CAT), and Sino-Nasal Outcome Test 20. Total of 13 children, 13 parents, and 17 adolescents repeated QOL-PCD 10 to 14 days later to assess test-retest reliability. Multitrait analysis evaluated how the items loaded to hypothesized scales: physical, emotional & social functioning, treatment burden, role, vitality, upper and lower respiratory symptoms, and ears and hearing symptoms. Examination of item-to-total correlations led to removal of three, five, and six items, respectively in the prototype child, adolescent and parent-proxy versions; the validated measures now comprise between 34 and 38 items.
resultsThe QOL-PCD scales had good internal consistency; Cronbach's α for QOL-PCD parent-proxy ranged 0.62 to 0.86. Test-retest reliability demonstrated stability across all scales; for example QOL-PCD adolescent intraclass correlation coefficients ranged 0.71 to 0.89. Significant relationships were found between QOL-PCD scales and similar constructs on generic questionnaires, for example, QOL-PCD adolescent lower respiratory symptoms and the CAT score (r = .64, P < .01); weaker correlations were found between different constructs.
conclusionAge-specific QOL-PCD demonstrated good internal consistency, test-retest reliability, and validity. QOL-PCD offers promising outcome measures for multicenter clinical trials, as well as monitoring symptoms, functioning, and QOL during routine care.
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