Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD).

ABSTRACT:

Introduction

Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B.

Methods

Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD. The health states were valued in time trade-off interviews with adult general population respondent

SUBMITTER: Matza LS 

PROVIDER: S-EPMC11442559 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature