Abstract | BACKGROUND: METHODS AND RESULTS: We studied 32 Fabry patients over a period of 3 years regarding disease progression and clinical outcome under enzyme replacement therapy. Regional myocardial fibrosis was assessed by magnetic resonance imaging late-enhancement technique. Echocardiographic myocardial mass was calculated with the Devereux formula, and myocardial function was quantified by ultrasonic strain-rate imaging. In addition, exercise capacity was measured by bicycle stress test. All measurements were repeated at yearly intervals. At baseline, 9 patients demonstrated at least 2 fibrotic left ventricular segments (severe myocardial fibrosis), 11 had 1 left ventricular segment affected (mild fibrosis), and 12 were without fibrosis. In patients without fibrosis, enzyme replacement therapy resulted in a significant reduction in left ventricular mass (238+/-42 g at baseline, 202+/-46 g at 3 years; P for trend <0.001), an improvement in myocardial function (systolic radial strain rate, 2.3+/-0.4 and 2.9+/-0.6 seconds(-1), respectively; P for trend=0.045), and a higher exercise capacity obtained by bicycle stress exercise (106+/-14 and 122+/-26 W, respectively; P for trend=0.014). In contrast, patients with mild or severe fibrosis showed a minor reduction in left ventricular hypertrophy and no improvement in myocardial function or exercise capacity. CONCLUSIONS:
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Authors | Frank Weidemann, Markus Niemann, Frank Breunig, Sebastian Herrmann, Meinrad Beer, Stefan Störk, Wolfram Voelker, Georg Ertl, Christoph Wanner, Jörg Strotmann |
Journal | Circulation
(Circulation)
Vol. 119
Issue 4
Pg. 524-9
(Feb 03 2009)
ISSN: 1524-4539 [Electronic] United States |
PMID | 19153271
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
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Chemical References |
- Recombinant Proteins
- alpha-Galactosidase
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Topics |
- Adult
- Cardiomyopathy, Hypertrophic
(diagnosis, drug therapy, etiology)
- Echocardiography
- Evidence-Based Medicine
- Exercise Test
- Fabry Disease
(complications, drug therapy)
- Female
- Follow-Up Studies
- Humans
- Magnetic Resonance Imaging
- Male
- Middle Aged
- Recombinant Proteins
(administration & dosage)
- Treatment Outcome
- alpha-Galactosidase
(administration & dosage)
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