Cost-effectiveness of enzyme replacement therapy for Fabry disease.

ABSTRACT:

Background

The cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care was evaluated in the Dutch cohort of patients with Fabry disease.

Methods

Cost-effectiveness analysis was performed using a life-time state-transition model. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort consisting of males and females aged 5-78 years. Intervention with ERT (either agalsidase alfa or agalsidase beta) was compared to the standard medical care. The main outcome measures were years without end organ damage (renal, cardiac en cerebrovascular complications), quality adjusted life years (QALYs), and costs.

Results

Over a 70 year lifetime, an untreated Fabry p

SUBMITTER: Rombach SM 

PROVIDER: S-EPMC3598841 | biostudies-literature | 2013 Feb

REPOSITORIES: biostudies-literature