Cataloging health state utility estimates for Duchenne muscular dystrophy and related conditions.

ABSTRACT:

Background

Duchenne muscular dystrophy (DMD) is a genetic disease resulting in progressive muscle weakness, loss of ambulation, and cardiorespiratory complications. Direct estimation of health-related quality of life for patients with DMD is challenging, highlighting the need for proxy measures. This study aims to catalog and compare existing published health state utility estimates for DMD and related conditions.

Methods

Using two search strategies, relevant utilities were extracted from the Tufts Cost-Effectiveness Analysis Registry, including health states, utility estimates, and study and patient characteristics. Analysis One identified health states with comparable utility estimates to a set of published US patient population utility estimates for DMD. A minimal clinica

SUBMITTER: Do LA 

PROVIDER: S-EPMC11367812 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature