Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment.

ABSTRACT:

Background

To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults.

Methods

Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment.

Results

After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH)

SUBMITTER: Kampmann C 

PROVIDER: S-EPMC4587871 | biostudies-literature | 2015 Sep

REPOSITORIES: biostudies-literature