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

ABSTRACT:

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 sel

SUBMITTER: Audhya IF 

PROVIDER: S-EPMC10724100 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature