HDQLIFE: Development and Assessment of Health-Related Quality of Life in Huntington Disease (HD)

Authors

N E Carlozzi, University of Michigan - Ann Arbor
S G Schilling, University of Michigan - Ann Arbor
J-S Lai, Northwestern University
J S Paulsen, University of Iowa
E A Hahn, Northwestern University
J S Perlmutter, Washington University in St. Louis
C A Ross, Johns Hopkins University
N R Downing, University of Iowa
A L Kratz, University of Michigan - Ann Arbor
M K McCormack, Rowan University
M A Nance, Struthers Parkinson's Center, Golden Valley, MN
K A Quaid, Indiana University - Indianapolis
J C Stout, Monash University, Australia
R C Gershon, Northwestern University
R E Ready, University of Massachusetts Amherst
J A Miner, University of Michigan - Ann Arbor
S K Barton, Washington University in St. Louis
S L Perlman, University of California, Los Angeles
S M Rao, Cleveland Clinic, Cleveland, OH
S Frank, Beth Israel Deaconess Medical Center, Boston, MA
I Shoulson, Georgetown University
H Marin, Rutgers University - New Brunswick/Piscataway
M D Geschwind, University of California, San Francisco
P Dayalu, University of Michigan - Ann Arbor
S M Goodnight, University of Michigan - Ann Arbor
D Cella, Northwestern University

Document Type

Article

Version Deposited

Accepted for publication (PostPrint)

Publication Date

10-1-2016

Publication Title

Quality of Life Research

DOI

10.1007/s11136-016-1386-3

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.

Comments

This is a post-peer-review, pre-copyedit version of an article published in Quality of Life Research. The final authenticated version is available online at: http://dx.doi.org/10.1007/s11136-016-1386-3

Recommended Citation

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. HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD). Quality of Life Research. 2016 Oct;25(10):2441-2455. Epub 2016 Aug 13. doi: 10.1007/s11136-016-1386-3. PMID: 27522213. PMCID: PMC6108175.