HDQLIFE

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

N. E. Carlozzi*, S. G. Schilling, Jin-Shei Lai, J. S. Paulsen, Elizabeth 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, Richard Gershon, R. E. Ready, J. A. Miner, S. K. Barton, S. L. Perlman, S. M. Rao, S. Frank & 6 others I. Shoulson, H. Marin, M. D. Geschwind, P. Dayalu, S. M. Goodnight, David Cella

*Corresponding author for this work

Research output: Contribution to journalArticle

19 Citations (Scopus)

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 languageEnglish (US)
Pages (from-to)2441-2455
Number of pages15
JournalQuality of Life Research
Volume25
Issue number10
DOIs
StatePublished - Oct 1 2016

Fingerprint

Huntington Disease
Quality of Life
Chorea
Inborn Genetic Diseases
Deglutition
Nervous System Diseases
Information Systems
Self Report
Statistical Factor Analysis

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

Cite this

Carlozzi, N. E. ; Schilling, S. G. ; Lai, Jin-Shei ; Paulsen, J. S. ; Hahn, Elizabeth 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, Richard ; Ready, R. E. ; Miner, J. A. ; Barton, S. K. ; Perlman, S. L. ; Rao, S. M. ; Frank, S. ; Shoulson, I. ; Marin, H. ; Geschwind, M. D. ; Dayalu, P. ; Goodnight, S. M. ; Cella, David. / HDQLIFE : development and assessment of health-related quality of life in Huntington disease (HD). In: Quality of Life Research. 2016 ; Vol. 25, No. 10. pp. 2441-2455.
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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.",
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author = "Carlozzi, {N. E.} and Schilling, {S. G.} and Jin-Shei Lai and Paulsen, {J. S.} and Hahn, {Elizabeth 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 Richard Gershon 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 David Cella",
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Carlozzi, NE, Schilling, SG, Lai, J-S, Paulsen, JS, Hahn, EA, Perlmutter, JS, Ross, CA, Downing, NR, Kratz, AL, McCormack, MK, Nance, MA, Quaid, KA, Stout, JC, Gershon, R, 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

HDQLIFE : development and assessment of health-related quality of life in Huntington disease (HD). / Carlozzi, N. E.; Schilling, S. G.; Lai, Jin-Shei; Paulsen, J. S.; Hahn, Elizabeth 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, Richard; Ready, R. E.; Miner, J. A.; Barton, S. K.; Perlman, S. L.; Rao, S. M.; Frank, S.; Shoulson, I.; Marin, H.; Geschwind, M. D.; Dayalu, P.; Goodnight, S. M.; Cella, David.

In: Quality of Life Research, Vol. 25, No. 10, 01.10.2016, p. 2441-2455.

Research output: Contribution to journalArticle

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, Jin-Shei

AU - Paulsen, J. S.

AU - Hahn, Elizabeth 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, Richard

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, David

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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