Abstract | BACKGROUND: METHODS: We evaluated the safety and effectiveness of recombinant alpha-galactosidase A in a multicenter, randomized, placebo-controlled, double-blind study of 58 patients who were treated every 2 weeks for 20 weeks. Thereafter, all patients received recombinant alpha-galactosidase A in an open-label extension study. The primary efficacy end point was the percentage of patients in whom renal microvascular endothelial deposits of globotriaosylceramide were cleared (reduced to normal or near-normal levels). We also evaluated the histologic clearance of microvascular endothelial deposits of globotriaosylceramide in the endomyocardium and skin, as well as changes in the level of pain and the quality of life. RESULTS: CONCLUSIONS:
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Authors | C M Eng, N Guffon, W R Wilcox, D P Germain, P Lee, S Waldek, L Caplan, G E Linthorst, R J Desnick, International Collaborative Fabry Disease Study Group |
Journal | The New England journal of medicine
(N Engl J Med)
Vol. 345
Issue 1
Pg. 9-16
(07 05 2001)
ISSN: 0028-4793 [Print] United States |
PMID | 11439963
(Publication Type: Clinical Trial, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)
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Chemical References |
- Trihexosylceramides
- globotriaosylceramide
- alpha-Galactosidase
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Topics |
- Adolescent
- Adult
- Capillaries
(drug effects, metabolism)
- Double-Blind Method
- Endothelium, Vascular
(drug effects, metabolism)
- Fabry Disease
(drug therapy, metabolism, pathology)
- Female
- Humans
- Kidney
(blood supply, pathology)
- Male
- Microcirculation
- Middle Aged
- Myocardium
(pathology)
- Pain Measurement
- Quality of Life
- Skin
(pathology)
- Trihexosylceramides
(analysis, blood, metabolism)
- alpha-Galactosidase
(adverse effects, therapeutic use)
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