Estimation of Health Utility Scores for Glycogen Storage Disease Type Ia.

ABSTRACT:

Background

Glycogen Storage Disease Type Ia (GSDIa) is a rare genetic metabolic disorder characterized by hypoglycemia, hepatomegaly, growth failure, and possible seizures/death. Patients frequently consume cornstarch to maintain blood glucose. Evidence demonstrating the impact of GSDIa on health-related quality of life (HRQoL) is lacking. This study aims to understand the burden of GSDIa by obtaining utility values for economic appraisals.

Methods

A targeted literature review and interviews with experts (n = 4) and caregivers (n = 4) informed the development of health state vignettes describing different GSDIa severities by age and level of hypothetical treatment (i.e., gene therapy) response. Health states reflecting caregivers' experiences were also developed. A convenien

SUBMITTER: Kruger E 

PROVIDER: S-EPMC10333147 | biostudies-literature | 2023 Jul

REPOSITORIES: biostudies-literature