Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study(364 views) Imbriaco M, Pisani A, Spinelli L, Cuocolo A, Messalli G, Capuano E, Marmo M, Liuzzi R, Visciano B, Cianciaruso B, Salvatore M
Keywords: Acetylsalicylic Acid, Agalsidase Beta, Antilipemic Agent, Calcium Antagonist, Dipeptidyl Carboxypeptidase Inhibitor, Erythropoietin, Adult, Anderson Fabry Disease, Article, Clinical Article, Controlled Study, Disorders Of Lipid And Lipoprotein Metabolism, Drug Tolerability, Enzyme Replacement, Female, Heart Left Ventricle Function, Heart Left Ventricle Mass, Heart Left Ventricle Volume, Heart Ventricle Septum, Human, Long Term Care, Physical Examination, Priority Journal, Relaxation Time, Treatment Duration, X Chromosome Linked Disorder, Alpha-Galactosidase, Drug Administration Schedule, Hypertrophy, Left Ventricular, Isoenzymes, Magnetic Resonance Imaging, Middle Aged, Prospective Studies, Stroke Volume, Ventricular Function, Young Adult, Fluorodeoxyglucose F 18, Body Posture, Breast Cancer, Cancer Diagnosis, Computer Assisted Emission Tomography, Follow Up, Intermethod Comparison, Letter, Nuclear Magnetic Resonance Imaging,
Affiliations: *** IBB - CNR ***
Department of Biomorphological and Functional Sciences, University Federico II, Naples, Italy
Department of Nephrology, University Federico II, Naples, Italy
Department of Clinical Medicine, Cardiovascular and Immunological Sciences, University Federico II, Naples, Italy
Department of National Research Council (IBB-CNR), University Federico II, Naples, Italy
Via Posillipo 196, 80123, Naples, Italy
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Hughes, D. A., Elliott, P. M., Shah, J., Zuckerman, J., Coghlan, G., Brookes, J., Mehta, A. B., Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: A randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa (2008) Heart, 94 (2), pp. 153-158. , DOI 10. 1136/hrt. 2006. 104026
Vedder, A. C., Breunig, F., Donker-Koopman, W. E., Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3 (2008) Mol Genet Metab, 94, pp. 319-325
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Goerres, G.W., Michel, S.C., Fehr, M.K., Follow up of women with breast cancer: Comparison between MRI and FDG PET (2003) Eur Radiol, 13, pp. 1635-1644
Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study
Background: Anderson-Fabry disease is a multisystem X linked disorder of lipid metabolism frequently associated with cardiac symptoms, including left ventricular (LV) hypertrophy gradually impairing cardiac function. Evidence showing that enzyme-replacement therapy (ERT) can be effective in reducing LV hypertrophy and improving myocardial function in the long term is limited. Objective: This study aimed to assess the long-term effects of ERT with recombinant alpha-galactosidase A (agalsidase beta, Fabrazyme) on LV function and myocardial signal intensity in 11 patients with Anderson Fabry disease. Patients: Eleven patients (eight males, three females) with varying stages of genetically confirmed Anderson Fabry disease were examined by means of physical examination and magnetic resonance imaging before ERT with agalsidase beta at 1 mg/kg every other week (study 1) and after a mean treatment duration of 45 months (study 2). Results: At 45 months of treatment, LV mass and LV wall thickness had significantly reduced: 188 (SD 60) g versus 153 (47) g, and 16 (4) mm versus 14 (4) mm, respectively. Furthermore, a significant reduction in myocardial T2 relaxation times was noted in all myocardial regions, that is, interventricular septum 80 (5) ms versus 66 (8) ms, apex 79 (10) ms versus 64 (10) ms, and lateral wall 80 (8) ms versus 65 (16) ms. Changes in LV ejection fraction were not significant. Amelioration of clinical symptoms was observed in all patients. Conclusions: Long-term therapy with agalsidase beta at 1 mg/kg every 2 weeks was effective in significantly reducing LV hypertrophy, improving overall cardiac performance and ameliorating clinical symptoms in patients with Anderson-Fabry disease.
Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study
No results.
Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study
Hesse B, Tagil K, Cuocolo A, Anagnostopoulos C, Bardies M, Bax J, Bengel F, Busemann Sokole E, Davies G, Dondi M, Edenbrandt L, Franken P, Kjaer A, Knuuti J, Lassmann M, Ljungberg M, Marcassa C, Marie PY, Mckiddie F, O'connor M, Prvuolovich E, Underwood R * 3. 0 T perfusion MR imaging(694 views) Rivista Di Neuroradiologia (ISSN: 1120-9976), 2004; 17(6): 807-812. Impact Factor:0.023 ViewExport to BibTeXExport to EndNote