Abstract | INTRODUCTION: PATIENTS AND METHODS: Ten patients (seven male, three female) with genetically confirmed AFD and at least 48 months continuous data collected during treatment with beta-galactosidase 1 mg/kg every other week, were switched to alpha-galactosidase 0.2 mg/kg every other week for at least 20 months, with prospective clinical evaluations performed every 6 months. Pre-switch data was collected retrospectively from patient charts. Cardiac functional parameters were assessed using magnetic resonance imaging. RESULTS: Results showed that renal function generally remained stable after the switch. Cardiac mass had already decreased significantly (p < 0.05 vs pre-ERT) after introduction of beta-galactosidase and remained unchanged after switching to alpha-galactosidase. Symptoms of pain and health status scores did not deteriorate during alpha-galactosidase therapy. Adverse events were mostly mild and infusion -related. DISCUSSION: In conclusion, switching to alpha-galactosidse was generally well tolerated and associated with stable clinical status and preservation of both renal and cardiac function.
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Authors | Antonio Pisani, Bianca Visciano, Ivana Capuano, Antonello Mancini, Eleonora Riccio |
Journal | Giornale italiano di nefrologia : organo ufficiale della Societa italiana di nefrologia
(G Ital Nefrol)
2013 Jan-Feb
Vol. 30
Issue 1
ISSN: 1724-5990 [Electronic] Italy |
Vernacular Title | Malattia di Fabry: ERT a confronto. |
PMID | 23832445
(Publication Type: Comparative Study, English Abstract, Journal Article, Observational Study)
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Chemical References |
- alpha-Galactosidase
- beta-Galactosidase
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Topics |
- Adult
- Dose-Response Relationship, Drug
- Drug Substitution
- Enzyme Replacement Therapy
(methods)
- Fabry Disease
(drug therapy, enzymology)
- Female
- Glomerular Filtration Rate
- Humans
- Magnetic Resonance Imaging, Cine
- Male
- Middle Aged
- Quality of Life
- Retrospective Studies
- Treatment Outcome
- alpha-Galactosidase
(therapeutic use)
- beta-Galactosidase
(therapeutic use)
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