Laura Behan1,2,3, Margaret W Leigh4, Sharon D Dell5,6,7,8, Alexandra L Quittner9, Claire Hogg10,11, Jane S Lucas1,2,3. 1. Primary Ciliary Dyskinesia Centre, University Hospital Southampton NHS Foundation Trust, University of Southampton, Southampton, UK. 2. NIHR Southampton Respiratory Biomedical Research Unit, University Hospital Southampton NHS Foundation Trust, University of Southampton, Southampton, UK. 3. Academic Unit of Clinical and Experimental Sciences Faculty of Medicine, University of Southampton, Southampton, UK. 4. Department of Pediatrics and Marsico Lung Institute, University of North Carolina School of Medicine, Chapel Hill, North Carolina. 5. Division of Respiratory Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada. 6. Child Health Evaluative Sciences, The Hospital for Sick Children, Toronto, Ontario, Canada. 7. Department of Pediatrics, University of Toronto, Toronto, Ontario, Canada. 8. Institute of Health Policy, Management and Education, University of Toronto, Toronto, Ontario, Canada. 9. Nicklaus Children's Research Institute, Nicklaus Children's Hospital, Miami, Florida. 10. Department of Paediatric Respiratory Medicine, Primary Ciliary Dyskinesia Centre, Royal Brompton Hospital, London, UK. 11. National Heart and Lung Institute, Imperial College, London, UK.
Abstract
RATIONALE: Having 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. METHODS: Children (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. RESULTS: The 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. CONCLUSION: Age-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.
RATIONALE: Having 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. METHODS: Children (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. RESULTS: The 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. CONCLUSION: Age-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.
Authors: Sharon D Dell; Margaret W Leigh; Jane S Lucas; Thomas W Ferkol; Michael R Knowles; Adrianne Alpern; Laura Behan; Anjana M Morris; Claire Hogg; Audrey DunnGalvin; Alexandra L Quittner Journal: Ann Am Thorac Soc Date: 2016-10
Authors: I Christopher McManus; Hannah M Mitchison; Eddie M K Chung; Georgina F Stubbings; Naomi Martin Journal: BMC Pulm Med Date: 2003-11-27 Impact factor: 3.317
Authors: Laura Behan; Margaret W Leigh; Sharon D Dell; Audrey Dunn Galvin; Alexandra L Quittner; Jane S Lucas Journal: Thorax Date: 2017-02-28 Impact factor: 9.139
Authors: Helene E Kobbernagel; Frederik F Buchvald; Eric G Haarman; Carmen Casaulta; Samuel A Collins; Claire Hogg; Claudia E Kuehni; Jane S Lucas; Heymut Omran; Alexandra L Quittner; Claudius Werner; Kim G Nielsen Journal: BMC Pulm Med Date: 2016-07-22 Impact factor: 3.317
Authors: Laura E Gardner; Katie L Horton; Amelia Shoemark; Jane S Lucas; Kim G Nielsen; Helene Kobbernagel; Bruna Rubbo; Robert A Hirst; Panayiotis Kouis; Nicola Ullmann; Ana Reula; Nisreen Rumman; Hannah M Mitchison; Andreia Pinto; Charlotte Richardson; Anne Schmidt; James Thompson; René Gaupmann; Maciej Dabrowski; Pleasantine Mill; Siobhan B Carr; Dominic P Norris; Claudia E Kuehni; Myrofora Goutaki; Claire Hogg Journal: BMC Proc Date: 2020-06-19
Authors: Myrofora Goutaki; Leonie Hüsler; Yin Ting Lam; Helena M Koppe; Andreas Jung; Romain Lazor; Loretta Müller; Eva S L Pedersen; Claudia E Kuehni Journal: ERJ Open Res Date: 2022-04-11
Authors: Myrofora Goutaki; Jean-François Papon; Mieke Boon; Carmen Casaulta; Ernst Eber; Estelle Escudier; Florian S Halbeisen; Amanda Harris; Claire Hogg; Isabelle Honore; Andreas Jung; Bulent Karadag; Cordula Koerner-Rettberg; Marie Legendre; Bernard Maitre; Kim G Nielsen; Bruna Rubbo; Nisreen Rumman; Lynne Schofield; Amelia Shoemark; Guillaume Thouvenin; Hannah Willkins; Jane S Lucas; Claudia E Kuehni Journal: ERJ Open Res Date: 2020-02-10