Validation of paediatric health-related quality of life instruments for primary ciliary dyskinesia (QOL-PCD)

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.

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Identifiers

ORCID for Jane Lucas: orcid.org/0000-0001-8701-9975

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