Estimating health state utilities for aromatic L-amino acid decarboxylase deficiency (AADCd) in the United States.

ABSTRACT:

Background

AADCd is a rare neurometabolic disorder presenting in infancy. Children with AADCd have motor dysfunction and development delays that result in the need for lifelong care; quality of life is greatly impacted. Current characterizations of health-related quality of life and associated health state utilities (HSUs) may be underestimated in AADCd. Accurate characterization of AADCd burden is important when evaluating the benefits of treatment, especially the improvements observed with the recently approved disease-modifying therapy eladocagene exuparvovec. Time-trade-off (TTO) vignette methods may be used to elicit HSUs in AADCd for assessing the value of new treatments. This study aimed to first update previously published health state vignettes, then estimate AADCd HSUs in

SUBMITTER: Monteleone B 

PROVIDER: S-EPMC11756132 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature