Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.

ABSTRACT:

Objective

To evaluate the cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care without ERT in the Dutch cohort of patients with type 1 Gaucher disease (GD I).

Design

Cost-effectiveness analysis was performed using a life-time state-transition model of the disease's natural course. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort.

Setting

The tertiary referral center for Gaucher disease in the Netherlands.

Participants

The Dutch cohort of patients with GD I.

Intervention

ERT versus standard medical care without ERT in symptomatic patients.

Main outcome measures

Years free of end organ damage (YFEOD) (splenectomy, bone c

SUBMITTER: van Dussen L 

PROVIDER: S-EPMC4022049 | biostudies-literature | 2014 Apr

REPOSITORIES: biostudies-literature