Understanding patient-reported outcome measures in Huntington disease: at what point is cognitive impairment related to poor measurement reliability?

N E Carlozzi; S Schilling; A L Kratz; J S Paulsen; S Frank; J C Stout

doi:10.1007/s11136-018-1912-6

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Journal article

Understanding patient-reported outcome measures in Huntington disease: at what point is cognitive impairment related to poor measurement reliability?

N E Carlozzi, S Schilling, A L Kratz, J S Paulsen, S Frank and J C Stout

Quality of life research, Vol.27(10), pp.2541-2555

10/2018

DOI: 10.1007/s11136-018-1912-6

PMCID: PMC6295362

PMID: 29909483

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https://www.ncbi.nlm.nih.gov/pmc/articles/6295362View

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Abstract

Symptom progression in Huntington disease (HD) is associated with cognitive decline which may interfere with the self-report of symptoms. Unfortunately, data to support or refute the psychometric reliability of patient-reported outcomes (PROs) as HD progresses are limited. This is problematic given that PROs are increasingly recognized as important measures of efficacy for new treatments. We examined PRO data from the HDQLIFE Measurement System (Speech Difficulties; Swallowing Difficulties; Chorea) in 509 individuals with premanifest, early-stage, or late-stage HD. Clinician-administered assessments of motor functioning (items from the UHDRS) and standardized objective assessments of cognition (Stroop, Symbol Digit Modalities) were also collected. We examined item bias using differential item functioning (DIF) across HD stage (premanifest, early-, late-) and relative to cognitive performance. We also examined the correlations between self-report and clinician ratings. Regression models that considered total cognitive ability were utilized to determine psychometric reliability of the PROs. Most PRO items were free from DIF for both staging and cognition. There were modest correlations between PROs and clinician report (ranged from - 0.40 to - 0.60). Modeling analyses indicated that psychometric reliability breaks down with poorer cognition and more progressed disease stage; split-half reliability was compromised (i.e., split-half reliability < 0.80) when scores were < 136 for Chorea, < 109 for Speech Difficulties, and < 179 for Swallowing Difficulties. Results indicate that the psychometric reliability of PROs can be compromised as HD symptoms progress and cognition declines. Clinicians should consider PROs in conjunction with other types of assessments when total cognition scores exceed critical thresholds.

Measurement

HDQLIFE

Cognition

Patient-reported outcomes

Huntington disease

Details

Title: Subtitle: Understanding patient-reported outcome measures in Huntington disease: at what point is cognitive impairment related to poor measurement reliability?
Creators: N E Carlozzi - University of Michigan
S Schilling - Institute for Social Research, University of Michigan, Ann Arbor, MI, USA
A L Kratz - Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, MI, USA
J S Paulsen - University of Iowa, Psychological and Brain Sciences
S Frank - Beth Israel Deaconess Medical Center, Boston, MA, USA
J C Stout - School of Psychological Sciences, Monash University, Clayton, VIC, Australia
Resource Type: Journal article
Publication Details: Quality of life research, Vol.27(10), pp.2541-2555
DOI: 10.1007/s11136-018-1912-6
PMID: 29909483
PMCID: PMC6295362
NLM abbreviation: Qual Life Res
ISSN: 0962-9343
eISSN: 1573-2649
Grant note: UL1TR000433 / National Center for Advancing Translational Sciences R01 NS040068 / NINDS NIH HHS R01NS040068 / National Institute of Neurological Disorders and Stroke R01NS077946 / National Institute of Neurological Disorders and Stroke R01 NS077946 / NINDS NIH HHS UL1 TR002240 / NCATS NIH HHS UL1 TR000433 / NCATS NIH HHS
Language: English
Date published: 10/2018
Academic Unit: Psychiatry; Psychological and Brain Sciences
Record Identifier: 9984206855602771

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