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2015 ; 10
(ä): 125
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Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac
outcomes after 10 years treatment
#MMPMID26416388
Kampmann C
; Perrin A
; Beck M
Orphanet J Rare Dis
2015[Sep]; 10
(ä): 125
PMID26416388
show ga
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.