HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD)

N. E. Carlozzi; S. G. Schilling; J. S. Lai; J. S. Paulsen; E. A. Hahn; J. S. Perlmutter; C. A. Ross; N. R. Downing; A. L. Kratz; M. K. McCormack; M. A. Nance; K. A. Quaid; J. C. Stout; R. C. Gershon; R. E. Ready; J. A. Miner; S. K. Barton; S. L. Perlman; S. M. Rao; S. Frank; I. Shoulson; H. Marin; M. D. Geschwind; P. Dayalu; S. M. Goodnight; D. Cella

doi:10.1007/s11136-016-1386-3

HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD)

N. E. Carlozzi^*, S. G. Schilling, J. S. Lai, J. S. Paulsen, E. A. Hahn, J. S. Perlmutter, C. A. Ross, N. R. Downing, A. L. Kratz, M. K. McCormack, M. A. Nance, K. A. Quaid, J. C. Stout, R. C. Gershon, R. E. Ready, J. A. Miner, S. K. Barton, S. L. Perlman, S. M. Rao, S. FrankI. Shoulson, H. Marin, M. D. Geschwind, P. Dayalu, S. M. Goodnight, D. Cella

^*Corresponding author for this work

Medical Social Sciences

Research output: Contribution to journal › Article › peer-review

41 Scopus citations

Abstract

Purpose: Huntington disease (HD) is a chronic, debilitating genetic disease that affects physical, emotional, cognitive, and social health. Existing patient-reported outcomes (PROs) of health-related quality of life (HRQOL) used in HD are neither comprehensive, nor do they adequately account for clinically meaningful changes in function. While new PROs examining HRQOL (i.e., Neuro-QoL—Quality of Life in Neurological Disorders and PROMIS—Patient-Reported Outcomes Measurement Information System) offer solutions to many of these shortcomings, they do not include HD-specific content, nor have they been validated in HD. HDQLIFE addresses this by validating 12 PROMIS/Neuro-QoL domains in individuals with HD and by using established PROMIS methodology to develop new, HD-specific content. Methods: New item pools were developed using cognitive debriefing with individuals with HD, and expert, literacy, and translatability reviews. Existing item banks and new item pools were field tested in 536 individuals with prodromal, early-, or late-stage HD. Results: Moderate to strong relationships between Neuro-QoL/PROMIS measures and generic self-report measures of HRQOL, and moderate relationships between Neuro-QoL/PROMIS and clinician-rated measures of similar constructs supported the validity of Neuro-QoL/PROMIS in individuals with HD. Exploratory and confirmatory factor analysis, item response theory, and differential item functioning analyses were utilized to develop new item banks for Chorea, Speech Difficulties, Swallowing Difficulties, and Concern with Death and Dying, with corresponding six-item short forms. A four-item short form was developed for Meaning and Purpose. Conclusions: HDQLIFE encompasses both validated Neuro-QoL/PROMIS measures, as well as five new scales in order to provide a comprehensive assessment of HRQOL in HD.

Original language	English (US)
Pages (from-to)	2441-2455
Number of pages	15
Journal	Quality of Life Research
Volume	25
Issue number	10
DOIs	https://doi.org/10.1007/s11136-016-1386-3
State	Published - Oct 1 2016

Funding

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, Roland Zschiegner, and Jane S. Paulsen. 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.

Keywords

HDQLIFE
Health-related quality of life
Huntington disease
Neuro-QoL
PROMIS
Patient-reported outcome (PRO)

ASJC Scopus subject areas

Public Health, Environmental and Occupational Health

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1007/s11136-016-1386-3

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Carlozzi, N. E., Schilling, S. G., Lai, J. S., Paulsen, J. S., Hahn, E. A., Perlmutter, J. S., Ross, C. A., Downing, N. R., Kratz, A. L., McCormack, M. K., Nance, M. A., Quaid, K. A., Stout, J. C., Gershon, R. C., Ready, R. E., Miner, J. A., Barton, S. K., Perlman, S. L., Rao, S. M., ... Cella, D. (2016). HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD). Quality of Life Research, 25(10), 2441-2455. https://doi.org/10.1007/s11136-016-1386-3

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abstract = "Purpose: Huntington disease (HD) is a chronic, debilitating genetic disease that affects physical, emotional, cognitive, and social health. Existing patient-reported outcomes (PROs) of health-related quality of life (HRQOL) used in HD are neither comprehensive, nor do they adequately account for clinically meaningful changes in function. While new PROs examining HRQOL (i.e., Neuro-QoL—Quality of Life in Neurological Disorders and PROMIS—Patient-Reported Outcomes Measurement Information System) offer solutions to many of these shortcomings, they do not include HD-specific content, nor have they been validated in HD. HDQLIFE addresses this by validating 12 PROMIS/Neuro-QoL domains in individuals with HD and by using established PROMIS methodology to develop new, HD-specific content. Methods: New item pools were developed using cognitive debriefing with individuals with HD, and expert, literacy, and translatability reviews. Existing item banks and new item pools were field tested in 536 individuals with prodromal, early-, or late-stage HD. Results: Moderate to strong relationships between Neuro-QoL/PROMIS measures and generic self-report measures of HRQOL, and moderate relationships between Neuro-QoL/PROMIS and clinician-rated measures of similar constructs supported the validity of Neuro-QoL/PROMIS in individuals with HD. Exploratory and confirmatory factor analysis, item response theory, and differential item functioning analyses were utilized to develop new item banks for Chorea, Speech Difficulties, Swallowing Difficulties, and Concern with Death and Dying, with corresponding six-item short forms. A four-item short form was developed for Meaning and Purpose. Conclusions: HDQLIFE encompasses both validated Neuro-QoL/PROMIS measures, as well as five new scales in order to provide a comprehensive assessment of HRQOL in HD.",

keywords = "HDQLIFE, Health-related quality of life, Huntington disease, Neuro-QoL, PROMIS, Patient-reported outcome (PRO)",

author = "Carlozzi, {N. E.} and Schilling, {S. G.} and Lai, {J. S.} and Paulsen, {J. S.} and Hahn, {E. A.} and Perlmutter, {J. S.} and Ross, {C. A.} and Downing, {N. R.} and Kratz, {A. L.} and McCormack, {M. K.} and Nance, {M. A.} and Quaid, {K. A.} and Stout, {J. C.} and Gershon, {R. C.} and Ready, {R. E.} and Miner, {J. A.} and Barton, {S. K.} and Perlman, {S. L.} and Rao, {S. M.} and S. Frank and I. Shoulson and H. Marin and Geschwind, {M. D.} and P. Dayalu and Goodnight, {S. M.} and D. Cella",

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doi = "10.1007/s11136-016-1386-3",

language = "English (US)",

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Carlozzi, NE, Schilling, SG, Lai, JS, Paulsen, JS, Hahn, EA, Perlmutter, JS, Ross, CA, Downing, NR, Kratz, AL, McCormack, MK, Nance, MA, Quaid, KA, Stout, JC, Gershon, RC, Ready, RE, Miner, JA, Barton, SK, Perlman, SL, Rao, SM, Frank, S, Shoulson, I, Marin, H, Geschwind, MD, Dayalu, P, Goodnight, SM & Cella, D 2016, 'HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD)', Quality of Life Research, vol. 25, no. 10, pp. 2441-2455. https://doi.org/10.1007/s11136-016-1386-3

TY - JOUR

T1 - HDQLIFE

T2 - development and assessment of health-related quality of life in Huntington disease (HD)

AU - Carlozzi, N. E.

AU - Schilling, S. G.

AU - Lai, J. S.

AU - Paulsen, J. S.

AU - Hahn, E. A.

AU - Perlmutter, J. S.

AU - Ross, C. A.

AU - Downing, N. R.

AU - Kratz, A. L.

AU - McCormack, M. K.

AU - Nance, M. A.

AU - Quaid, K. A.

AU - Stout, J. C.

AU - Gershon, R. C.

AU - Ready, R. E.

AU - Miner, J. A.

AU - Barton, S. K.

AU - Perlman, S. L.

AU - Rao, S. M.

AU - Frank, S.

AU - Shoulson, I.

AU - Marin, H.

AU - Geschwind, M. D.

AU - Dayalu, P.

AU - Goodnight, S. M.

AU - Cella, D.

PY - 2016/10/1

Y1 - 2016/10/1

N2 - Purpose: Huntington disease (HD) is a chronic, debilitating genetic disease that affects physical, emotional, cognitive, and social health. Existing patient-reported outcomes (PROs) of health-related quality of life (HRQOL) used in HD are neither comprehensive, nor do they adequately account for clinically meaningful changes in function. While new PROs examining HRQOL (i.e., Neuro-QoL—Quality of Life in Neurological Disorders and PROMIS—Patient-Reported Outcomes Measurement Information System) offer solutions to many of these shortcomings, they do not include HD-specific content, nor have they been validated in HD. HDQLIFE addresses this by validating 12 PROMIS/Neuro-QoL domains in individuals with HD and by using established PROMIS methodology to develop new, HD-specific content. Methods: New item pools were developed using cognitive debriefing with individuals with HD, and expert, literacy, and translatability reviews. Existing item banks and new item pools were field tested in 536 individuals with prodromal, early-, or late-stage HD. Results: Moderate to strong relationships between Neuro-QoL/PROMIS measures and generic self-report measures of HRQOL, and moderate relationships between Neuro-QoL/PROMIS and clinician-rated measures of similar constructs supported the validity of Neuro-QoL/PROMIS in individuals with HD. Exploratory and confirmatory factor analysis, item response theory, and differential item functioning analyses were utilized to develop new item banks for Chorea, Speech Difficulties, Swallowing Difficulties, and Concern with Death and Dying, with corresponding six-item short forms. A four-item short form was developed for Meaning and Purpose. Conclusions: HDQLIFE encompasses both validated Neuro-QoL/PROMIS measures, as well as five new scales in order to provide a comprehensive assessment of HRQOL in HD.

AB - Purpose: Huntington disease (HD) is a chronic, debilitating genetic disease that affects physical, emotional, cognitive, and social health. Existing patient-reported outcomes (PROs) of health-related quality of life (HRQOL) used in HD are neither comprehensive, nor do they adequately account for clinically meaningful changes in function. While new PROs examining HRQOL (i.e., Neuro-QoL—Quality of Life in Neurological Disorders and PROMIS—Patient-Reported Outcomes Measurement Information System) offer solutions to many of these shortcomings, they do not include HD-specific content, nor have they been validated in HD. HDQLIFE addresses this by validating 12 PROMIS/Neuro-QoL domains in individuals with HD and by using established PROMIS methodology to develop new, HD-specific content. Methods: New item pools were developed using cognitive debriefing with individuals with HD, and expert, literacy, and translatability reviews. Existing item banks and new item pools were field tested in 536 individuals with prodromal, early-, or late-stage HD. Results: Moderate to strong relationships between Neuro-QoL/PROMIS measures and generic self-report measures of HRQOL, and moderate relationships between Neuro-QoL/PROMIS and clinician-rated measures of similar constructs supported the validity of Neuro-QoL/PROMIS in individuals with HD. Exploratory and confirmatory factor analysis, item response theory, and differential item functioning analyses were utilized to develop new item banks for Chorea, Speech Difficulties, Swallowing Difficulties, and Concern with Death and Dying, with corresponding six-item short forms. A four-item short form was developed for Meaning and Purpose. Conclusions: HDQLIFE encompasses both validated Neuro-QoL/PROMIS measures, as well as five new scales in order to provide a comprehensive assessment of HRQOL in HD.

KW - HDQLIFE

KW - Health-related quality of life

KW - Huntington disease

KW - Neuro-QoL

KW - PROMIS

KW - Patient-reported outcome (PRO)

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HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD)

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UN SDGs

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