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

Orphanet Journal of Rare Diseases

Michael Beck³  Amandine Perrin²  Christoph Kampmann¹ 

[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;Statistical Programmer, Rare Diseases Business Unit, Global Outcomes Research, Shire, Zug, Switzerland;Professor Emeritus, Department of Pediatrics, University Medical Center, University of Mainz, Mainz, Germany

关键词: Lysosomal storage disorder;    Left ventricular hypertrophy;    Fabry disease;    Enzyme replacement therapy;    Cardiomyopathy;    Agalsidase alfa;   

Others  :  1228435
DOI  :  10.1186/s13023-015-0338-2

 received in 2015-05-04, accepted in 2015-09-06,  发布年份 2015

【 摘 要 】

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.

【 授权许可】

   
2015 Kampmann et al.

【 图 表 】

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