Reliability and Validity of the HD-PRO-TriadTM, a Health-Related Quality of Life Measure Designed to Assess the Symptom Triad of Huntington’s Disease

Boileau, Nicholas R.; Stout, Julie C.; Paulsen, Jane S.; Cella, David; McCormack, Michael K.; Nance, Martha; Frank, Samuel; Lai, Jin Shei; Carlozzi, Noelle E.

doi:10.3233/jhd-170238

Cited by 5 publications

(6 citation statements)

References 76 publications

(72 reference statements)

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“…Cognitive symptoms are a key driver of disability and functioning deficits in HD, and often results in loss of work and independent functioning [ 32 , 34 ]. Therefore, it is unclear whether EQ-5D is appropriate for measuring HRQOL impacts associated with cognitive impairment in HD.…”

Section: Discussionmentioning

confidence: 99%

“…No value set (providing utility values for all the possible health states) is currently available for the EQ-5D including the cognition bolt-on, so this cannot be used to calculate QALYs. Cognitive changes often appear in the prodromal stage, prior to motor onset (diagnostic confidence level < 4) [ 41 , 42 ] and can include a decline in executive function (including working memory, flexible thinking, and self-control), memory problems, and difficulty concentrating [ 34 ]. Further research into the appropriateness of the EQ-5D and whether other generic utility measures such as the Health Utilities Index, 15 Dimensions, or EQ Health and Wellbeing (EQ-HWB) instrument, which include specific dimensions for cognition and mental function, would be valuable.…”

Section: Discussionmentioning

confidence: 99%

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Health state utility estimates for value assessments of novel treatments in Huntington’s disease: a systematic literature review

Sawant,

Paret,

Petrillo

et al. 2024

Health Qual Life Outcomes

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Background Huntington’s disease (HD) is a progressive neurodegenerative disease with a devastating impact on patients and their families. Quantifying how treatments affect patient outcomes is critical for informing reimbursement decisions. Many countries mandate a formal value assessment in which the treatment benefit is measured as quality-adjusted life-years, calculated with the use of utility estimates that reflect respondents’ preferences for health states. Objective To summarize published health state utility data in HD and identify gaps and uncertainties in the data available that could be used to inform value assessments. Methods We conducted a systematic literature review of studies that used preference-based instruments (e.g., EQ-5D and SF-6D) to estimate utility values for people with HD. The studies were published between January 2012 and December 2022. Results Of 383 articles screened, 16 articles reported utility values estimated in 11 distinct studies. The utility measure most frequently reported was EQ-5D (9/11 studies). Two studies reported SF-6D data; one used time trade-off methods to value health state descriptions (vignettes). Although utility scores generally worsened to a lower value with increased HD severity, the estimates varied considerably across studies. The EQ-5D index range was 0.89 − 0.72 for mild/prodromal HD and 0.71 − 0.37 for severe/late-stage disease. Conclusions This study uncovered high variability in published utility estimates, indicating substantial uncertainty in existing data. Further research is needed to better understand preferences and valuation across all stages and domains of HD symptoms and the degree to which generic utility measures capture the impact of cognitive changes on quality of life.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Health state utility estimates for value assessments of novel treatments in Huntington’s disease: a systematic literature review

Sawant,

Paret,

Petrillo

et al. 2024

Health Qual Life Outcomes

View full text Add to dashboard Cite

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“…However, the results might be explained by the low sensitivity of the questionnaires. While it is well known that quality of life is inversely associated with increasing stages of the disease55–58 and increased symptoms are associated with lower quality of life,59,60 less is known about the magnitude of longitudinal changes in quality of life 61. Indeed, there is currently no health-related quality of live measure recommended to measure change of severity over time 62…”

Section: Discussionmentioning

confidence: 99%

Exercise in Huntington’s Disease: Current State and Clinical Significance

Mueller

Petersen

Jung

2019

Tremor and Other Hyperkinetic Movements

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BackgroundHuntington’s disease (HD) is a rare, progressive neurodegenerative disease. Currently, there is no cure for the disease, but treatment may alleviate HD symptoms. In recent years, several exercise training interventions have been conducted in HD patients. In the current article, we review previous studies investigating targeted exercise training interventions in HD patients.MethodsWe performed a literature search using the PubMed, Scopus, Web of Science, and Google Scholar databases on exercise training interventions in HD patients. Six publications fulfilled the criteria and were included in the review.ResultsExercise training resulted in beneficial effects on cardiovascular and mitochondrial function. Training effects on cognition, motor function, and body composition were less congruent, but a positive effect seems likely. Health-related quality of life during the training interventions was stable. Most studies reported no related adverse events in response to training.DiscussionExercise training seems to be safe and feasible in HD patients. However, current knowledge is mainly based on short, small-scale studies and it cannot be transferred to all HD patients. Therefore, longer-term interventions with larger HD patient cohorts are necessary to draw firm conclusions about the potentially positive effects of exercise training in HD patients.

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“…While it is well known that quality of life is inversely associated with increasing stages of the disease 55 – 58 and increased symptoms are associated with lower quality of life, 59 , 60 less is known about the magnitude of longitudinal changes in quality of life. 61 Indeed, there is currently no health-related quality of live measure recommended to measure change of severity over time. 62 …”

Section: Discussionmentioning

confidence: 99%