Cardiac manifestations of Anderson-Fabry disease: results from the international Fabry outcome survey
Language English Country England, Great Britain Media print-electronic
Document type Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't
PubMed
17483538
DOI
10.1093/eurheartj/ehm153
PII: ehm153
Knihovny.cz E-resources
- MeSH
- alpha-Galactosidase therapeutic use MeSH
- Adult MeSH
- Dyspnea epidemiology etiology MeSH
- Echocardiography MeSH
- Fabry Disease complications drug therapy MeSH
- Glomerular Filtration Rate MeSH
- Hypertrophy, Left Ventricular complications epidemiology MeSH
- Isoenzymes therapeutic use MeSH
- Middle Aged MeSH
- Humans MeSH
- Heart Diseases * complications epidemiology MeSH
- Prevalence MeSH
- Recombinant Proteins MeSH
- Risk Factors MeSH
- Syncope epidemiology etiology MeSH
- Treatment Outcome MeSH
- Check Tag
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Male MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Multicenter Study MeSH
- Research Support, Non-U.S. Gov't MeSH
- Geographicals
- Europe epidemiology MeSH
- Names of Substances
- agalsidase alfa MeSH Browser
- alpha-Galactosidase MeSH
- Isoenzymes MeSH
- Recombinant Proteins MeSH
AIMS: Anderson-Fabry disease (AFD) is an uncommon X-linked disorder caused by deficient activity of the lysosomal enzyme alpha-galactosidase A. The Fabry Outcome Survey is a European database designed to monitor the long-term efficacy and safety of enzyme replacement therapy (ERT) with agalsidase alfa. The aim of this study was to determine the prevalence and characteristics of cardiac disease in AFD patients. METHODS AND RESULTS: Clinical and laboratory data were available in 714 patients from 11 countries (mean age 35 +/- 17 years, 369 women, 336 treated). The prevalence of angina was 23 vs. 22%; palpitations and arrhythmias 27 vs. 26%; exertional dyspnoea 23 vs. 23%; and syncope 2 vs. 4%, in women and men, respectively (all P = NS). The frequency of all cardiac symptoms was significantly higher in treated than in untreated patients. Gender, age, and glomerular filtration rate were independent determinants of echocardiographically assessed left ventricular hypertrophy (LVH). CONCLUSION: This study confirms the high prevalence of cardiac morbidity associated with AFD. The disease burden in treated women exceeds that of untreated men, suggesting that most women selected for ERT have advanced disease. The presence of LVH is associated with higher frequency of cardiac signs and symptoms and relates independently to gender, age, and renal function.
2nd Department of Internal Medicine 1st Faculty of Medicine Charles University Prague Czech Republic
References provided by Crossref.org
Nationwide screening of Fabry disease in patients with hypertrophic cardiomyopathy in Czech Republic
Narrative review on Morbus Fabry: diagnosis and management of cardiac manifestations
Ventricular tachycardia: a presentation of Fabry disease case report
Uric Acid as a Marker of Mortality and Morbidity in Fabry Disease
Intravascular ultrasound assessment of coronary artery involvement in Fabry disease
