PCR176 Time Trade-Off Utility Values for Health States Characterizing Progressive Muscular Degeneration in Duchenne Muscular Dystrophy (DMD)

Audhya, Ivana F.; Szabo, S; Bever, A.; Malone, DC; Feeny, David; Neumann, PI; Bolatova, T.; Iannaccone, ST; Gooch, KL

doi:10.1016/j.jval.2022.04.1519

Cited by 3 publications

(1 citation statement)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The present study is a secondary analysis of research initially conducted to develop vignettes describing the clinical progression of DMD, with the objective of eliciting utility values for DMD health states for use in economic models [ 27 ]. Individuals with cognitive or behavioural disorders that substantially impacted social interaction were excluded from this analysis to gain a clear picture of the social and emotional implications of DMD in the absence of potentially confounding comorbidities.…”

Section: Methodsmentioning

confidence: 99%

“You Take This Day by Day, Come What May”: A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy

Bever,

Audhya,

Szabo

et al. 2024

Adv Ther

View full text Add to dashboard Cite

Introduction Studies have reported health-related quality-of-life impacts of Duchenne muscular dystrophy (DMD); however, further research is needed to understand how those with DMD experience their condition and how psychosocial impacts evolve over time in response to disease progression. This qualitative study explores the social and emotional implications of key transitions, challenges and adaptations throughout the disease course from the perspective of patients and family caregivers. Methods Semi-structured interviews were conducted with men and boys with DMD, and/or their caregivers, in the USA. Thematic analysis was used to examine patterns in data collected across the interviews. Results Nineteen participants were included. Three major themes were identified: (1) barriers to participation are multifaceted; (2) an emotional journey shaped by ‘inevitable progression;' (3) family provides critical tangible and emotional support. This study illustrates that psychosocial impacts of DMD are shaped by knowledge of the condition’s natural history alongside other factors including the extent of social barriers, personal growth and adaptation, and family support. Conclusions Findings provide insight into the strength and resilience with which individuals and their families respond to daily challenges and major clinical milestones and highlight the relative importance of loss of upper limb function as a transition in DMD affecting health-related quality-of-life. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-024-02867-0.

show abstract

Section: Methodsmentioning

confidence: 99%

“You Take This Day by Day, Come What May”: A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy

Bever,

Audhya,

Szabo

et al. 2024

Adv Ther

View full text Add to dashboard Cite

show abstract

Discriminatory Properties of the Quality-Adjusted Life Year Based Cost-Effectiveness Analyses for Patients With Disabilities: A Duchenne Muscular Dystrophy Case Study

Klimchak,

Sedita,

Perfetto

et al. 2024

Value in Health

View full text Add to dashboard Cite

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L

Audhya,

Szabo,

Bever

et al. 2023

J Patient Rep Outcomes

View full text Add to dashboard Cite

Background The progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, few health state utility data exist; published utilities tend to be derived from small samples for a limited number of health states and are often based on caregiver-reported patient health status. This study estimated utility values for varied clinical and functional health states in DMD, based on patient-reported health status. Methods Individuals with DMD in the US aged 12–40 years completed the EQ-5D (5-level) and Health Utilities Index (HUI) preference-based instruments. Based on responses to a clinical questionnaire, participants self-classified into functional health states according to level of lower and upper limb function, use of respiratory support, and presence of cardiomyopathy. Mean [standard deviation (SD)] utility and EQ-5D visual analogue scale (VAS) scores were estimated according to health state; and median (interquartile range) attribute levels calculated to understand which domains of health are most severely affected in DMD. Results Of 63 males with DMD, mean (SD) age was 19.8 (6.1) years and 11 (17.5%) were ambulatory. Mean (SD) utility values were 0.92 (0.08; HUI2), 0.84 (0.20; HUI3), and 0.84 (0.13; EQ-5D) for ambulatory patients without cardiomyopathy (n = 10). For non-ambulatory patients with moderately impaired upper limb function, night and daytime ventilation without cardiomyopathy, mean (SD) utilities were 0.49 (0.07) for the HUI2, 0.16 (0.15) for the HUI3 and 025 (0.14) for the EQ-5D. Mean (SD) VAS scores for the same health states were 91 (9) and 83 (21), respectively. In addition to impairments in mobility/ambulation, and self-care, attributes like usual activities and pain also showed notable effects of DMD. Conclusions In DMD, although a relationship between disease progression and HRQoL is observed, there is large variability in utility within functional health states, and across instruments. Utility values for less severe non-ambulatory health states described by level of upper limb function are novel. These utility values, derived based on direct patient feedback rather than from caregiver report, are relevant to individuals of varying functional statuses and augment scarce DMD-specific utility data.

show abstract

PCR176 Time Trade-Off Utility Values for Health States Characterizing Progressive Muscular Degeneration in Duchenne Muscular Dystrophy (DMD)

Cited by 3 publications

References 0 publications

“You Take This Day by Day, Come What May”: A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy

“You Take This Day by Day, Come What May”: A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy

Discriminatory Properties of the Quality-Adjusted Life Year Based Cost-Effectiveness Analyses for Patients With Disabilities: A Duchenne Muscular Dystrophy Case Study

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L

Contact Info

Product

Resources

About