TY - JOUR
T1 - Reliability and Validity of the HD-PRO-TriadTM, a Health-Related Quality of Life Measure Designed to Assess the Symptom Triad of Huntington's Disease
AU - Boileau, Nicholas R.
AU - Stout, Julie C.
AU - Paulsen, Janes S.
AU - Cella, David
AU - McCormack, Michael K.
AU - Nance, Martha A.
AU - Frank, Samuel
AU - Lai, Jin Shei
AU - Carlozzi, Noelle E.
N1 - Funding Information:
J.C. Stout has received research funding in the past three years from the Australian National Health and Medical Research Council, University College London, the CHDI Foundation, Prana Biotechnology, and the University of California, Davis. She is a Director of Stout Neuropsych Pty Ltd, which has received funding from Omeros, Teva Pharmaceuticals, Vaccinex, and Isis. She has been a consultant to Prana Biotechnology and Roche. She receives compensation as a member of the Board of the Huntington’s Study Group.
Funding Information:
Despite these limitations, this study provides additional support for the reliability and validity of HD-PRO-TRIADTM. Findings supporting internal consistency and convergent and discriminant validity replicated previous published work. New analyses from this study, demonstrating the lack floor and ceiling effects, as well as analyses supporting known-groups validity, minimal measurement error, and responsiveness of the HD-PRO-TRIADTM, provide additional support for the clinical utility of this measure. Ultimately, findings suggest that the HD- Work on this manuscript was supported by the National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (R01NS077946) and the National Center for Advancing Translational Sciences (UL1TR000433). In addition, a portion of this study sample was collected in conjunction with the Predict-HD study. The Predict-HD study was supported by the NIH, National Institute of Neurological Disorders and Stroke (R01NS040068), the NIH, Center for Inherited Disease Research (provided supported for sample phenotyping), and the CHDI Foundation (award to the University of Iowa). We thank the University of Iowa, the Investigators and Coordinators of this study, the study participants, the National Research Roster for Huntington Disease Patients and Families, the Huntington Study Group, and the Huntington’s Disease Society of America. We acknowledge the assistance of Jeffrey D. Long, Hans J. Johnson, Jeremy H. Bockholt, and Roland Zschieg-ner. We also acknowledge Roger Albin, Kelvin Chou, and Henry Paulsen for the assistance with participant recruitment. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.
Funding Information:
N.E. Carlozzi currently has research grants from the NIH; she is also supported by grant funding from the NIH, NIDILRR, and CHDI; she declares no conflicts of interest.
Funding Information:
S. Frank receives salary support from the Huntington Study Group for a study sponsored by Auspex Pharmaceuticals he declares no conflicts of interest.
Funding Information:
J.-S. Lai currently has research grants from the NIH; she declares no conflicts of interest.
Funding Information:
J.S. Paulsen currently has research grants from the NIH; she is also supported by grant funding from NIH, NINDS, and CHDI; she declares no conflicts of interest.
Publisher Copyright:
© 2017-IOS Press and the authors.
PY - 2017
Y1 - 2017
N2 - Background: Huntington's disease (HD), is a neurodegenerative disorder that is associated with cognitive, behavioral, and motor impairments that diminish health related quality of life (HRQOL). The HD-PRO-TRIADTM is a quality of life measure that assesses health concerns specific to individuals with HD. Preliminary psychometric characterization was limited to a convenience sample of HD participants who completed measures at home so clinician-ratings were unavailable. Objectives: The current study evaluates the reliability and validity of the HD-PRO-TRIADTM in a well-characterized sample of individuals with HD. Methods: Four-hundred and eighty-two individuals with HD (n = 192 prodromal, n = 193 early, and n = 97 late) completed the HD-PRO-TRIADTM questionnaire. Clinician-rated assessments from the Unified Huntington Disease Rating Scales, the short Problem Behaviors Assessment, and three generic measures of HRQOL (WHODAS 2.0, RAND-12, and EQ-5D) were also examined. Results: Internal reliability for all domains and the total HD-PRO-TRIADTM was excellent (all Cronbach's > 0.93). Convergent and discriminant validity were supported by significant associations between the HD-PRO-TRIADTM domains, and other patient reported outcome measures as well as clinician-rated measures. Known groups validity was supported as the HD-PRO-TRIADTM differentiated between stages of the disease. Floor and ceiling effects were generally within acceptable limits. There were small effect sizes for 12-month change over time and moderate effect sizes for 24-month change over time. Conclusions: Findings support excellent internal reliability, convergent and discriminant validity, known groups validity, and responsiveness to change over time. The current study supports the clinical efficacy of the HD-PRO-TRIADTM. Future research is needed to assess the test-retest reliability of this measure.
AB - Background: Huntington's disease (HD), is a neurodegenerative disorder that is associated with cognitive, behavioral, and motor impairments that diminish health related quality of life (HRQOL). The HD-PRO-TRIADTM is a quality of life measure that assesses health concerns specific to individuals with HD. Preliminary psychometric characterization was limited to a convenience sample of HD participants who completed measures at home so clinician-ratings were unavailable. Objectives: The current study evaluates the reliability and validity of the HD-PRO-TRIADTM in a well-characterized sample of individuals with HD. Methods: Four-hundred and eighty-two individuals with HD (n = 192 prodromal, n = 193 early, and n = 97 late) completed the HD-PRO-TRIADTM questionnaire. Clinician-rated assessments from the Unified Huntington Disease Rating Scales, the short Problem Behaviors Assessment, and three generic measures of HRQOL (WHODAS 2.0, RAND-12, and EQ-5D) were also examined. Results: Internal reliability for all domains and the total HD-PRO-TRIADTM was excellent (all Cronbach's > 0.93). Convergent and discriminant validity were supported by significant associations between the HD-PRO-TRIADTM domains, and other patient reported outcome measures as well as clinician-rated measures. Known groups validity was supported as the HD-PRO-TRIADTM differentiated between stages of the disease. Floor and ceiling effects were generally within acceptable limits. There were small effect sizes for 12-month change over time and moderate effect sizes for 24-month change over time. Conclusions: Findings support excellent internal reliability, convergent and discriminant validity, known groups validity, and responsiveness to change over time. The current study supports the clinical efficacy of the HD-PRO-TRIADTM. Future research is needed to assess the test-retest reliability of this measure.
KW - HD-PRO-TRIAD
KW - Health-related quality of life
KW - Huntington's disease
KW - Patient reported outcome (PRO)
KW - Psychometric
KW - Reliability
KW - Validity
UR - http://www.scopus.com/inward/record.url?scp=85030451180&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85030451180&partnerID=8YFLogxK
U2 - 10.3233/JHD-170238
DO - 10.3233/JHD-170238
M3 - Article
C2 - 28968239
AN - SCOPUS:85030451180
SN - 1879-6397
VL - 6
SP - 201
EP - 215
JO - Journal of Huntington's disease
JF - Journal of Huntington's disease
IS - 3
ER -