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

Louis S MatzaKatie D StewartMarie FournierDonna RowenRobin LachmannMaurizio ScarpaEugen MengelTravis ObermeyerEvren AyikFernando LaredoRuth Pulikottil-Jacob

Published in: The European journal of health economics : HEPAC : health economics in prevention and care (2024)

Greater severity of ASMD was associated with lower mean utility. Results have implications for valuation of pediatric health states. The resulting utilities may be useful in cost-utility modeling estimating the value of treatment for ASMD.

Keyphrases

public health
healthcare
mental health
health information
replacement therapy
combination therapy
smoking cessation