TY - JOUR
T1 - Validation of a health-related quality of life instrument for primary ciliary dyskinesia (QOL-PCD)
AU - Behan, Laura
AU - Leigh, Margaret W.
AU - Dell, Sharon D.
AU - Dunn Galvin, Audrey
AU - Quittner, Alexandra L.
AU - Lucas, Jane S.
N1 - Funding Information:
Funding This research was funded by grant support to JSL, ALQ and MWL by funding from the European Union’s Seventh Framework Programme under EC-GA No. 305404 BESTCILIA; by grant support to SDD and MWL: U54HL096458 from the National Institutes of Health (NIH) through the Genetic Disorders of Mucociliary Clearance Consortium, an initiative of the NIH Office of Rare Diseases Research at the National Center for Advancing Translational Science and the National Heart, Lung and Blood Institute; by grant support to SDD by Maya’s March, The Hospital for Sick Children Foundation, Toronto, Ontario, Canada; ALQ was supported from an investigator-initiated grant, Gilead Sciences; The National PCD Centre in Southampton is commissioned and funded by NHS England. Research in Southampton is supported by NIHR Southampton Respiratory Biomedical Research Unit, NIHR Wellcome Trust Clinical Research Facility and The AAIR Charity (Reg. No. 1129698). JSL, MWL and LB are members of ERS Task Force for PCD Diagnostics (ERS TF-2014-04) and EU-funded COST Action BEAT-PCD (BM1407).
Publisher Copyright:
© Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-And-licensing/.
PY - 2017/9/1
Y1 - 2017/9/1
N2 - Background Quality of life (QOL)-primary ciliary dyskinesia (PCD) is the first disease-specific, health-related QOL instrument for PCD. Psychometric validation of QOL-PCD assesses the performance of this measure in adults, including its reliability, validity and responsiveness to change. Methods Seventy-Two adults (mean (range) age: 33â €..years (18-79â €..years); mean (range) FEV 1 % predicted: 68 (26-115)) with PCD completed the 49-item QOL-PCD and generic QOL measures: Short-Form 36 Health Survey, Sino-Nasal Outcome Test 20 (SNOT-20) and St George Respiratory Questionnaire (SGRQ)-C. Thirty-five participants repeated QOL-PCD 10-14â €..days later to measure stability or reproducibility of the measure. Results Multitrait analysis was used to evaluate how the items loaded on 10 hypothesised scales: physical, emotional, role and social functioning, treatment burden, vitality, health perceptions, upper respiratory symptoms, lower respiratory symptoms and ears and hearing symptoms. This analysis of item-To-Total correlations led to 9 items being dropped; the validated measure now comprises 40 items. Each scale had excellent internal consistency (Cronbach's α: 0.74 to 0.94). Two-week test-retest demonstrated stability for all scales (intraclass coefficients 0.73 to 0.96). Significant correlations were obtained between QOL-PCD scores and age and FEV 1. Strong relationships were also found between QOL-PCD scales and similar constructs on generic questionnaires, for example, lower respiratory symptoms and SGRQ-C (r=0.72, p<0.001), while weak correlations were found between measures of different constructs. Conclusions QOL-PCD has demonstrated good internal consistency, test-retest reliability, convergent and divergent validity. QOL-PCD offers a promising tool for evaluating new therapies and for measuring symptoms, functioning and QOL during routine care.
AB - Background Quality of life (QOL)-primary ciliary dyskinesia (PCD) is the first disease-specific, health-related QOL instrument for PCD. Psychometric validation of QOL-PCD assesses the performance of this measure in adults, including its reliability, validity and responsiveness to change. Methods Seventy-Two adults (mean (range) age: 33â €..years (18-79â €..years); mean (range) FEV 1 % predicted: 68 (26-115)) with PCD completed the 49-item QOL-PCD and generic QOL measures: Short-Form 36 Health Survey, Sino-Nasal Outcome Test 20 (SNOT-20) and St George Respiratory Questionnaire (SGRQ)-C. Thirty-five participants repeated QOL-PCD 10-14â €..days later to measure stability or reproducibility of the measure. Results Multitrait analysis was used to evaluate how the items loaded on 10 hypothesised scales: physical, emotional, role and social functioning, treatment burden, vitality, health perceptions, upper respiratory symptoms, lower respiratory symptoms and ears and hearing symptoms. This analysis of item-To-Total correlations led to 9 items being dropped; the validated measure now comprises 40 items. Each scale had excellent internal consistency (Cronbach's α: 0.74 to 0.94). Two-week test-retest demonstrated stability for all scales (intraclass coefficients 0.73 to 0.96). Significant correlations were obtained between QOL-PCD scores and age and FEV 1. Strong relationships were also found between QOL-PCD scales and similar constructs on generic questionnaires, for example, lower respiratory symptoms and SGRQ-C (r=0.72, p<0.001), while weak correlations were found between measures of different constructs. Conclusions QOL-PCD has demonstrated good internal consistency, test-retest reliability, convergent and divergent validity. QOL-PCD offers a promising tool for evaluating new therapies and for measuring symptoms, functioning and QOL during routine care.
KW - Psychology
KW - Rare lung diseases
KW - Respiratory Measurement
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U2 - 10.1136/thoraxjnl-2016-209356
DO - 10.1136/thoraxjnl-2016-209356
M3 - Article
C2 - 28246220
AN - SCOPUS:85020514939
VL - 72
SP - 832
EP - 839
JO - Thorax
JF - Thorax
SN - 0040-6376
IS - 9
ER -