A novel patient-reported outcome for paediatric localized scleroderma: A qualitative assessment of content validity.
Date
2019-09-09
Journal Title
Journal ISSN
Volume Title
Citation Stats
Attention Stats
Abstract
BACKGROUND:According to current standards, no existing patient-reported outcome (PRO) measures have high quality validity evidence for use with individuals diagnosed with paediatric localized scleroderma. This lack severely hinders patient-centred LS-focused research, including much needed clinical trials. OBJECTIVES:To develop a valid health-related quality of life measure for individuals with paediatric-localized scleroderma (LS) and to qualitatively evaluate its content validity using a patient-centred approach. METHODS:Previously collected qualitative data from youth with LS and their caregivers was used to develop items. The resulting item-set was administered in a clinical setting to participants aged 8-18 years old. Cognitive interviews were used to evaluate time to survey completion, readability/understanding of the items, appropriateness of the recall period, and construct representation. RESULTS:Seventeen children and adolescents with LS participated in the study. Interviews supported readability, understanding of the items, and appropriateness of the recall period in individuals >10 years old. Revisions were made to simplify the instructions and to be more inclusive of different subtypes of localized scleroderma. Three items were added to improve content representation. CONCLUSIONS:Content validity was supported by the patient-centred development process of the outcome measure and via direct feedback from individuals with LS and their families. Although an important first step, the resulting PRO, termed the "Localized Scleroderma Quality of Life Instrument", should be further evaluated in a larger sample before being implemented.
Type
Department
Description
Provenance
Subjects
Citation
Permalink
Published Version (Please cite this version)
Publication Info
Zigler, CK, K Ardalan, S Lane, KL Schollaert and KS Torok (2019). A novel patient-reported outcome for paediatric localized scleroderma: A qualitative assessment of content validity. The British journal of dermatology. 10.1111/bjd.18512 Retrieved from https://hdl.handle.net/10161/19373.
This is constructed from limited available data and may be imprecise. To cite this article, please review & use the official citation provided by the journal.
Collections
Scholars@Duke
Christina Kelsey Zigler
Dr. Zigler is an independent investigator dedicated to improving the ways we measure patient-centered, meaningful health states, specializing in pediatrics, rare diseases, and clinical trials. She also is a methodological co-investigator who supports the application of novel statistical approaches and measurement strategies across a diverse array of studies.
Her methodological expertise includes established qualitative and quantitative techniques (i.e. concept elicitation, cognitive testing, classical test theory, item-response theory) alongside innovative methods (i.e. novel ways to identify meaningful change), to ensure the measures are ultimately centered in the patients’ meaningful experiences. Further, she works in a variety of populations and contexts, including pediatrics, where patient-reported outcome measures empower children to report directly on their own health status, and rare diseases, where a creative and pragmatic approach to measure development is necessary and patient-centeredness is essential.
Areas of Expertise: Health Measurement, Health Behavior, and Health Services Research
Kaveh Ardalan
Dr. Ardalan's clinical interests are in juvenile myositis, lupus, and scleroderma. He serves as Co-Director of the Duke Children's Myositis Center (Cure JM Foundation Center of Excellence) and as co-leader of the Duke Pediatric Lupus Clinic. Building on his prior expertise in psychometrics, outcomes research (including patient-reported outcome measures), and the impact of pediatric rheumatic diseases on mental health and cardiovascular disease risk, Dr. Ardalan led the Lupus Erythematosus and Dermatomyositis Stress and Cardiovascular Health (LEADS-CV) biopsychosocial cohort study evaluating associations of emotional distress, inflammation, and cardiovascular health in juvenile-onset lupus and dermatomyositis. Dr. Ardalan's current work is focused on the co-design and iterative refinement of behavioral interventions for youth with lupus and dermatomyositis as well as support for clinical trials in pediatric rheumatic diseases, including cellular therapies.
Unless otherwise indicated, scholarly articles published by Duke faculty members are made available here with a CC-BY-NC (Creative Commons Attribution Non-Commercial) license, as enabled by the Duke Open Access Policy. If you wish to use the materials in ways not already permitted under CC-BY-NC, please consult the copyright owner. Other materials are made available here through the author’s grant of a non-exclusive license to make their work openly accessible.