Validation of pediatric 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.

Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD

“…This would permit completing all modules at baseline and during annual reviews and fewer modules during 3-monthly routine follow-up. The working group took into account the validated PCD quality of life questionnaires to avoid overlaps so that the instruments can be used together and complement each other [16][17][18][19]. After each round, MG resolved disagreements among participants, contacting them individually if required, and then made the next draft.…”

Pediatric pulmonology 2019 year in review: rare and diffuse lung disease

“…A pediatric QOL instrument was recently developed through focus groups and interviews with pediatric PCD patients and their parents 71 and joins a similar one developed and validated for use in adults with PCD. 72,73 Behan et al 74 evaluated child, adolescent, and parent-proxy versions of the instrument, and all performed well in terms of internal consistency, test-retest stability, and validity when compared to other generic QOL scales. These scales were used as outcome measures in the first multinational randomized clinical trial of pharmacotherapy in PCD 75 and will certainly have a role in future studies.…”

Proceedings of the 4th BEAT-PCD Conference and 5th PCD Training School

“…Work Group 4 (WG4) Clinical Trialsco-led by Philipp Latzin (Switzerland) and Bruna Rubbo (UK): WG4 focussed on identifying, defining and validating outcome measures for use in longitudinal studies. Laura Behan (Ireland) and Jane Lucas (UK) developed and validated the first disease-specific quality of life questionnaires (QOL-PCD) during BESTCILIA, in collaboration with researchers from the Genetic Disorders of Mucociliary Clearance Consortium [36][37][38][39][40]. Ensuring QOL-PCD is both valid and robust required International collaboration; rigorous literature review [41]; expert and patient interviews with psychometric analysis to ensure the clarity and importance of each question.…”

“…They revisited the development, validation and translation of these tools into clinical practice and reviewed available versions that can be accessed for patients. Laura Behan demonstrated how to deliver and score the QOL-PCD questionnaire during an interactive session [36][37][38]40]. Myrofora Goutaki showed how to navigate FOLLOW-PCD with hands-on experience of entering patient data and extracting their datasets [35].…”