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Orphanet J Rare Dis. 2015 Sep 29;10:125. doi: 10.1186/s13023-015-0338-2.

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

Author information

1
Section Head for Congenital Heart Defects, Center for Pediatric and Adolescent Medicine, University Medical Center, University of Mainz, Langenbeckstr. 1, Mainz, DE-55101, Germany. kampmann@mail.uni-mainz.de.
2
Statistical Programmer, Rare Diseases Business Unit, Global Outcomes Research, Shire, Zug, Switzerland. kampmann@uni-mainz.de.
3
Professor Emeritus, Department of Pediatrics, University Medical Center, University of Mainz, Mainz, Germany. kampmann@uni-mainz.de.

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) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.

CONCLUSIONS:

Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.

PMID:
26416388
PMCID:
PMC4587871
DOI:
10.1186/s13023-015-0338-2
[Indexed for MEDLINE]
Free PMC Article
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