Abstract
Purpose: As Huntington disease (HD) progresses, speech and swallowing difficulties become more profound. These difficulties have an adverse effect on health-related quality of life (HRQOL), thus psychometrically robust measures of speech and swallowing are needed to better understand the impact of these domains across the course of the disease. Therefore, the purpose of this study is to establish the clinical utility of two new patient-reported outcome measures (PROs), HDQLIFE Speech Difficulties and HDQLIFE Swallowing Difficulties. Methods: Thirty-one participants with premanifest or manifest HD, and 31 age- and sex-matched healthy control participants were recruited for this study. Participants completed several PROs [HDQLIFE Speech Difficulties, HDQLIFE Swallowing Difficulties, Communication Participation Item Bank (CPIB)], as well as several clinician-rated assessments of speech and functioning. A computational algorithm designed to detect features of spoken discourse was also examined. Analyses were focused on establishing the reliability and validity of these new measures. Results: Internal consistency was good for Swallowing (Cronbach’s alpha = 0.89) and excellent for Speech and the CPIB (both Cronbach’s alpha ≥ 0.94), and convergent/discriminant validity was supported. Known groups validity for the PROs was supported by significant group differences among control participants and persons with different stages of HD (all p < 0.0001). All PROs were able to distinguish those with and without clinician-rated dysarthria (likelihood ratios far exceeded the threshold for clinical decision making [all ≥ 3.28]). Conclusions: Findings support the clinical utility of the HDQLIFE Speech and Swallowing PROs and the CPIB for use across the HD disease spectrum. These PROs also have the potential to be clinically useful in other populations.
Original language | English (US) |
---|---|
Pages (from-to) | 251-265 |
Number of pages | 15 |
Journal | Quality of Life Research |
Volume | 30 |
Issue number | 1 |
DOIs | |
State | Published - Jan 2021 |
Funding
Convergent and discriminant validity correlation coefficients are reported in Table . Correlations among the PRO measures were large supporting convergent validity. Discriminant validity was supported by moderate correlations among the HDQLIFE PROs and other measures of clinician-rated speech and the Algorithm-based features of spoken discourse. Convergent and discriminant validity of the HDQLIFE PROs were also supported by our findings. Specifically, convergent validity was supported by moderate to strong associations (all ≥ 0.44) among the three PROs. Furthermore, discriminant validity was supported by moderate correlations (generally between 0.40 and 0.60) among the PROs and other measures of clinician-rated speech and the algorithm-based features of spoken discourse. This pattern of findings was consistent with our proposed hypotheses and provides support for the validity of the HDQLIFE Speech and Swallowing Difficulties PROs in persons with HD. We thank the Investigators and Coordinators of this study, the study participants, the Huntington Study Group, and the Huntington?s Disease Society of America.
Keywords
- Health-related quality of life
- Huntington disease
- Patient-reported outcome measures
- Speech difficulties
- Swallowing difficulties
ASJC Scopus subject areas
- Public Health, Environmental and Occupational Health