Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study (364 views)
Heart (ISSN: 1355-6037, 1468-201x), 2009 Jul; 95(13): 1103-1107.
Export to BibTeX Export to EndNote
Paper type: Letter, Journal Article,
Impact factor: 5.385, 5-year impact factor: 4.838
Url: http://www.scopus.com/inward/record.url?eid=2-s2.0-67649583702&partnerID=40&md5=a963c7aeaff474a0a34efb4bfa2c9cbd
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
References:
Brady, R.O., Gal, A.E., Bradley, R.M., Enzymatic defect in Fabry's disease. Ceramidetrihexosidase deficiency (1967) New Engl J Med, 276, pp. 1163-116
Desnick, R., Ioannou, Y., Eng, C., Alpha-galactosidase A deficiency: Fabry disease (2001) The Metabolic and Molecular Bases of Inherited Disease. 8th Edn., pp. 3733-3774. , Scriver CR, Beaudet AL, Sly WS, et al, eds. New York: McGraw-Hill
Linhart, A., Magage, S., Palecek, T., Bultas, J., Cardiac involvement in Fabry disease (2002) Acta Paediatrica, International Journal of Paediatrics, Supplement, 91 (439), pp. 15-20
Wilcox, W.R., Oliveira, J.P., Hopkin, R.J., Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry (2008) Mol Genet Metab, 93, pp. 112-128
Hopkin, R.J., Bissler, J., Banikazemi, M., Characterization of Fabry disease in 352 paediatric patients in the Fabry Registry (2008) Pediatr Res, 64, pp. 550-555
Kampmann, C., Linhart, A., Baehner, F., Onset and progression of the Anderson-Fabry disease related cardiomyopathy (2008) Int J Cardiol, 130, pp. 367-373
Weidemann, F., Breunig, F., Beer, M., Sandstede, J., Stork, S., Voelker, W., Ertl, G., Strotmann, J.M., The variation of morphological and functional cardiac manifestation in Fabry disease: Potential implications for the time course of the disease (2005) European Heart Journal, 26 (12), pp. 1221-1227. , DOI 10.1093/eurheartj/ehi143
Imbriaco, M., Spinelli, L., Cuocolo, A., Maurea, S., Sica, G., Quarantelli, M., Pisani, A., Salvatore, M., MRI characterization of myocardial tissue in patients with Fabry's disease (2007) American Journal of Roentgenology, 188 (3), pp. 850-853. , DOI 10.2214/AJR.05.0442
Eng, C.M., Guffon, N., Wilcox, W.R., Germain, D.P., Lee, P., Waldek, S., Caplan, L., Desnick, R.J., Safety and efficacy of recombinant human alpha-galactosidase a replacement therapy in Fabry's disease (2001) New England Journal of Medicine, 345 (1), pp. 9-16. , DOI 10.1056/NEJM200107053450102
Schiffmann, R., Kopp, J.B., Austin III, H.A., Sabnis, S., Moore, D.F., Weibel, T., Balow, J.E., Brady, R.O., Enzyme replacement therapy in fabry disease a randomized controlled trial (2001) Journal of the American Medical Association, 285 (21), pp. 2743-2749
Wilcox, W.R., Banikazemi, M., Guffon, N., Long term safety and efficacy of enzyme replacement therapy for Fabry disease (2004) Am J Hum Genet, 75, pp. 65-74
Koskenvuo, J.W., Hartiala, J.J., Nuutila, P., Twenty-four-month a-galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters (2008) J Inherit Metab Dis, 31, pp. 432-441
Whybra, C., Kampmann, C., Krummenauer, F., Ries, M., Mengel, E., Miebach, E., Baehner, F., Beck, M., The Mainz Severity Score Index: A new instrument for quantifying the Anderson - Fabry disease phenotype, and the response of patients to enzyme replacement therapy (2004) Clinical Genetics, 65 (4), pp. 299-307. , DOI 10.1111/j.1399-0004.2004.00219.x
Branton, M., Schiffmann, R., Kopp, J.B., Natural history and treatment of renal involvement in Fabry disease (2002) Journal of the American Society of Nephrology, 13 (SUPPL. 2), pp. S139-S143
Desnick, R.J., Brady, R.O., Fabry disease in childhood (2004) Journal of Pediatrics, 144 (5 SUPPL.), pp. S20-S26. , DOI 10.1016/j.jpeds.2004.01.051
Moller, A.T., Jensen, T.S., Neurological manifestations in Fabry's disease (2007) Nature Clinical Practice Neurology, 3 (2), pp. 95-106. , DOI 10.1038/ncpneuro0407, PII NCPNEURO0407
Linhart, A., Elliott, P.M., The heart in Anderson-Fabry disease and other lysosomal storage disorders (2007) Heart, 93 (4), pp. 528-535. , DOI 10.1136/hrt.2005.063818
Linhart, A., Treatment of Anderson-Fabry disease (2008) Heart, 94 (2), pp. 138-139. , DOI 10.1136/hrt.2006.113886
Von Scheidt, W., Eng, C.M., Fitzmaurice, T.F., An atypical variant of Fabry's disease with manifestations confined to the myocardium (1991) New Engl J Med, 324, pp. 395-399
Higgins, C.B., Herfkens, R., Lipton, M.J., Nuclear magnetic resonance imaging of acute myocardial infarction in dogs: Alterations in magnetic relaxation times (1983) Am J Cardiol, 52, pp. 184-188
Germain, D.P., Waldek, S., Banikazemi, M., Bushinsky, D.A., Charrow, J., Desnick, R.J., Lee, P., Guffon, N., Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with fabry disease (2007) Journal of the American Society of Nephrology, 18 (5), pp. 1547-1557. , DOI 10.1681/ASN.2006080816
Breunig, F., Weidemann, F., Strotmann, J., Clinical benefit of enzyme replacement therapy in Fabry disease (2006) Kidney Int, 69, pp. 1216-1221
Banikazemi, M., Bultas, J., Waldek, S., Wilcox, W.R., Whitley, C.B., McDonald, M., Finkel, R., Desnick, R.J., Agalsidase-beta therapy for advanced fabry disease: A randomized trial (2007) Annals of Internal Medicine, 146 (2), pp. 77-86. , http://www.annals.org/cgi/reprint/146/2/77.pdf
Beer, M., Weidemann, F., Breunig, F., Impact of enzyme replace therapy on cardiac morphology and function and late enhancement in Fabry's cardiomiopathy (2006) Am J Cardiol, 97, pp. 1515-1518
Spinelli, L., Pisani, A., Sabbatini, M., Petretta, M., Andreucci, M.V., Procaccini, D., Lo Surdo, N., Cianciaruso, B., Enzyme replacement therapy with agalsidase beta improves cardiac involvement in Fabry's disease (2004) Clinical Genetics, 66 (2), pp. 158-165. , DOI 10.1111/j.1399-0004.2004.00284.x
Kovacevic-Preradovic, T., Zuber, M., Attenhofer Jost, C.H., Anderson-Fabry disease: Long-term echocardiographic follow-up under enzyme replacement therapy (2008) Eur J Echocardiogr, 9, pp. 729-735
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
Ioannou, Y.A., Zeidner, K.M., Gordon, R.E., Desnick, R.J., Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase a replacement in enzyme-deficient mice (2001) American Journal of Human Genetics, 68 (1), pp. 14-25. , DOI 10.1086/316953
Beck, M., Ricci, R., Widmer, U., Dehout, F., Garcia De Lorenzo, A., Kampmann, C., Linhart, A., Mehta, A., Fabry disease: Overall effects of agalsidase alfa treatment (2004) European Journal of Clinical Investigation, 34 (12), pp. 838-844. , DOI 10.1111/j.1365-2362.2004.01424.x
Ohashi, T., Iizuka, S., Ida, H., Reduced alpha-Gal A enzyme activity in Fabry fibroblast cells and Fabry mice tissues induced by serum from antibody positive patients with Fabry disease (2008) Mol Genet Metab, 94, pp. 313-318
Zarate, Y.A., Hopkin, R.J., Fabry's disease (2008) Lancet, 372, pp. 1427-1435
Maguire Jr., G. Q., Noz, M. E., Ponzo, F., Prone breast dual-time-point PET/CT compared with MRI for determining breast cancer (2009) AJR, 193, pp. W77. , (letter) [web
Imbriaco, M., Caprio, M. G., Limite, G., Dual-time-point 18F-FDG PET/CT versus dynamic breast MRI of suspicious breast lesions (2008) AJR, 191, pp. 1323-1330
Moy, L., Ponzo, F., Noz, M. E., Improving specificity of breast MRI using prone PET and fused MRI and PET 3D volume datasets (2007) J Nucl Med, 48, pp. 528-537
Moy, L., Noz, M. E., Maguire Jr., G. Q., Prone mammoPET acquisition improves the ability to fuse MRI and PET breast scans (2007) Clin Nucl Med, 32, pp. 194-198
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
Brady, R. O., Gal, A. E., Bradley, R. M., Enzymatic defect in Fabry's disease. Ceramidetrihexosidase deficiency (1967) New Engl J Med, 276, pp. 1163-116
Wilcox, W. R., Oliveira, J. P., Hopkin, R. J., Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry (2008) Mol Genet Metab, 93, pp. 112-128
Hopkin, R. J., Bissler, J., Banikazemi, M., Characterization of Fabry disease in 352 paediatric patients in the Fabry Registry (2008) Pediatr Res, 64, pp. 550-555
Eng, C. M., Guffon, N., Wilcox, W. R., Germain, D. P., Lee, P., Waldek, S., Caplan, L., Desnick, R. J., Safety and efficacy of recombinant human alpha-galactosidase a replacement therapy in Fabry's disease (2001) New England Journal of Medicine, 345 (1), pp. 9-16. , DOI 10. 1056/NEJM200107053450102
Wilcox, W. R., Banikazemi, M., Guffon, N., Long term safety and efficacy of enzyme replacement therapy for Fabry disease (2004) Am J Hum Genet, 75, pp. 65-74
Koskenvuo, J. W., Hartiala, J. J., Nuutila, P., Twenty-four-month -galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters (2008) J Inherit Metab Dis, 31, pp. 432-441
Desnick, R. J., Brady, R. O., Fabry disease in childhood (2004) Journal of Pediatrics, 144 (5 SUPPL.), pp. S20-S26. , DOI 10. 1016/j. jpeds. 2004. 01. 051
Moller, A. T., Jensen, T. S., Neurological manifestations in Fabry's disease (2007) Nature Clinical Practice Neurology, 3 (2), pp. 95-106. , DOI 10. 1038/ncpneuro0407, PII NCPNEURO0407
Higgins, C. B., Herfkens, R., Lipton, M. J., Nuclear magnetic resonance imaging of acute myocardial infarction in dogs: Alterations in magnetic relaxation times (1983) Am J Cardiol, 52, pp. 184-188
Germain, D. P., Waldek, S., Banikazemi, M., Bushinsky, D. A., Charrow, J., Desnick, R. J., Lee, P., Guffon, N., Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with fabry disease (2007) Journal of the American Society of Nephrology, 18 (5), pp. 1547-1557. , DOI 10. 1681/ASN. 2006080816
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
Ioannou, Y. A., Zeidner, K. M., Gordon, R. E., Desnick, R. J., Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase a replacement in enzyme-deficient mice (2001) American Journal of Human Genetics, 68 (1), pp. 14-25. , DOI 10. 1086/316953
Zarate, Y. A., Hopkin, R. J., Fabry's disease (2008) Lancet, 372, pp. 1427-1435
Maguire Jr., G.Q., Noz, M.E., Ponzo, F., Prone breast dual-time-point PET/CT compared with MRI for determining breast cancer (2009) AJR, 193, pp. W77. , (letter) [web
Moy, L., Noz, M.E., Maguire Jr., G.Q., Prone mammoPET acquisition improves the ability to fuse MRI and PET breast scans (2007) Clin Nucl Med, 32, pp. 194-198
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
Heart (ISSN: 1355-6037, 1468-201x), 2009 Jul; 95(13): 1103-1107.
Export to BibTeX Export to EndNote
Paper type: Letter, Journal Article,
Impact factor: 5.385, 5-year impact factor: 4.838
Url: http://www.scopus.com/inward/record.url?eid=2-s2.0-67649583702&partnerID=40&md5=a963c7aeaff474a0a34efb4bfa2c9cbd
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
References:
Brady, R.O., Gal, A.E., Bradley, R.M., Enzymatic defect in Fabry's disease. Ceramidetrihexosidase deficiency (1967) New Engl J Med, 276, pp. 1163-116
Desnick, R., Ioannou, Y., Eng, C., Alpha-galactosidase A deficiency: Fabry disease (2001) The Metabolic and Molecular Bases of Inherited Disease. 8th Edn., pp. 3733-3774. , Scriver CR, Beaudet AL, Sly WS, et al, eds. New York: McGraw-Hill
Linhart, A., Magage, S., Palecek, T., Bultas, J., Cardiac involvement in Fabry disease (2002) Acta Paediatrica, International Journal of Paediatrics, Supplement, 91 (439), pp. 15-20
Wilcox, W.R., Oliveira, J.P., Hopkin, R.J., Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry (2008) Mol Genet Metab, 93, pp. 112-128
Hopkin, R.J., Bissler, J., Banikazemi, M., Characterization of Fabry disease in 352 paediatric patients in the Fabry Registry (2008) Pediatr Res, 64, pp. 550-555
Kampmann, C., Linhart, A., Baehner, F., Onset and progression of the Anderson-Fabry disease related cardiomyopathy (2008) Int J Cardiol, 130, pp. 367-373
Weidemann, F., Breunig, F., Beer, M., Sandstede, J., Stork, S., Voelker, W., Ertl, G., Strotmann, J.M., The variation of morphological and functional cardiac manifestation in Fabry disease: Potential implications for the time course of the disease (2005) European Heart Journal, 26 (12), pp. 1221-1227. , DOI 10.1093/eurheartj/ehi143
Imbriaco, M., Spinelli, L., Cuocolo, A., Maurea, S., Sica, G., Quarantelli, M., Pisani, A., Salvatore, M., MRI characterization of myocardial tissue in patients with Fabry's disease (2007) American Journal of Roentgenology, 188 (3), pp. 850-853. , DOI 10.2214/AJR.05.0442
Eng, C.M., Guffon, N., Wilcox, W.R., Germain, D.P., Lee, P., Waldek, S., Caplan, L., Desnick, R.J., Safety and efficacy of recombinant human alpha-galactosidase a replacement therapy in Fabry's disease (2001) New England Journal of Medicine, 345 (1), pp. 9-16. , DOI 10.1056/NEJM200107053450102
Schiffmann, R., Kopp, J.B., Austin III, H.A., Sabnis, S., Moore, D.F., Weibel, T., Balow, J.E., Brady, R.O., Enzyme replacement therapy in fabry disease a randomized controlled trial (2001) Journal of the American Medical Association, 285 (21), pp. 2743-2749
Wilcox, W.R., Banikazemi, M., Guffon, N., Long term safety and efficacy of enzyme replacement therapy for Fabry disease (2004) Am J Hum Genet, 75, pp. 65-74
Koskenvuo, J.W., Hartiala, J.J., Nuutila, P., Twenty-four-month a-galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters (2008) J Inherit Metab Dis, 31, pp. 432-441
Whybra, C., Kampmann, C., Krummenauer, F., Ries, M., Mengel, E., Miebach, E., Baehner, F., Beck, M., The Mainz Severity Score Index: A new instrument for quantifying the Anderson - Fabry disease phenotype, and the response of patients to enzyme replacement therapy (2004) Clinical Genetics, 65 (4), pp. 299-307. , DOI 10.1111/j.1399-0004.2004.00219.x
Branton, M., Schiffmann, R., Kopp, J.B., Natural history and treatment of renal involvement in Fabry disease (2002) Journal of the American Society of Nephrology, 13 (SUPPL. 2), pp. S139-S143
Desnick, R.J., Brady, R.O., Fabry disease in childhood (2004) Journal of Pediatrics, 144 (5 SUPPL.), pp. S20-S26. , DOI 10.1016/j.jpeds.2004.01.051
Moller, A.T., Jensen, T.S., Neurological manifestations in Fabry's disease (2007) Nature Clinical Practice Neurology, 3 (2), pp. 95-106. , DOI 10.1038/ncpneuro0407, PII NCPNEURO0407
Linhart, A., Elliott, P.M., The heart in Anderson-Fabry disease and other lysosomal storage disorders (2007) Heart, 93 (4), pp. 528-535. , DOI 10.1136/hrt.2005.063818
Linhart, A., Treatment of Anderson-Fabry disease (2008) Heart, 94 (2), pp. 138-139. , DOI 10.1136/hrt.2006.113886
Von Scheidt, W., Eng, C.M., Fitzmaurice, T.F., An atypical variant of Fabry's disease with manifestations confined to the myocardium (1991) New Engl J Med, 324, pp. 395-399
Higgins, C.B., Herfkens, R., Lipton, M.J., Nuclear magnetic resonance imaging of acute myocardial infarction in dogs: Alterations in magnetic relaxation times (1983) Am J Cardiol, 52, pp. 184-188
Germain, D.P., Waldek, S., Banikazemi, M., Bushinsky, D.A., Charrow, J., Desnick, R.J., Lee, P., Guffon, N., Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with fabry disease (2007) Journal of the American Society of Nephrology, 18 (5), pp. 1547-1557. , DOI 10.1681/ASN.2006080816
Breunig, F., Weidemann, F., Strotmann, J., Clinical benefit of enzyme replacement therapy in Fabry disease (2006) Kidney Int, 69, pp. 1216-1221
Banikazemi, M., Bultas, J., Waldek, S., Wilcox, W.R., Whitley, C.B., McDonald, M., Finkel, R., Desnick, R.J., Agalsidase-beta therapy for advanced fabry disease: A randomized trial (2007) Annals of Internal Medicine, 146 (2), pp. 77-86. , http://www.annals.org/cgi/reprint/146/2/77.pdf
Beer, M., Weidemann, F., Breunig, F., Impact of enzyme replace therapy on cardiac morphology and function and late enhancement in Fabry's cardiomiopathy (2006) Am J Cardiol, 97, pp. 1515-1518
Spinelli, L., Pisani, A., Sabbatini, M., Petretta, M., Andreucci, M.V., Procaccini, D., Lo Surdo, N., Cianciaruso, B., Enzyme replacement therapy with agalsidase beta improves cardiac involvement in Fabry's disease (2004) Clinical Genetics, 66 (2), pp. 158-165. , DOI 10.1111/j.1399-0004.2004.00284.x
Kovacevic-Preradovic, T., Zuber, M., Attenhofer Jost, C.H., Anderson-Fabry disease: Long-term echocardiographic follow-up under enzyme replacement therapy (2008) Eur J Echocardiogr, 9, pp. 729-735
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
Ioannou, Y.A., Zeidner, K.M., Gordon, R.E., Desnick, R.J., Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase a replacement in enzyme-deficient mice (2001) American Journal of Human Genetics, 68 (1), pp. 14-25. , DOI 10.1086/316953
Beck, M., Ricci, R., Widmer, U., Dehout, F., Garcia De Lorenzo, A., Kampmann, C., Linhart, A., Mehta, A., Fabry disease: Overall effects of agalsidase alfa treatment (2004) European Journal of Clinical Investigation, 34 (12), pp. 838-844. , DOI 10.1111/j.1365-2362.2004.01424.x
Ohashi, T., Iizuka, S., Ida, H., Reduced alpha-Gal A enzyme activity in Fabry fibroblast cells and Fabry mice tissues induced by serum from antibody positive patients with Fabry disease (2008) Mol Genet Metab, 94, pp. 313-318
Zarate, Y.A., Hopkin, R.J., Fabry's disease (2008) Lancet, 372, pp. 1427-1435
Maguire Jr., G. Q., Noz, M. E., Ponzo, F., Prone breast dual-time-point PET/CT compared with MRI for determining breast cancer (2009) AJR, 193, pp. W77. , (letter) [web
Imbriaco, M., Caprio, M. G., Limite, G., Dual-time-point 18F-FDG PET/CT versus dynamic breast MRI of suspicious breast lesions (2008) AJR, 191, pp. 1323-1330
Moy, L., Ponzo, F., Noz, M. E., Improving specificity of breast MRI using prone PET and fused MRI and PET 3D volume datasets (2007) J Nucl Med, 48, pp. 528-537
Moy, L., Noz, M. E., Maguire Jr., G. Q., Prone mammoPET acquisition improves the ability to fuse MRI and PET breast scans (2007) Clin Nucl Med, 32, pp. 194-198
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
Brady, R. O., Gal, A. E., Bradley, R. M., Enzymatic defect in Fabry's disease. Ceramidetrihexosidase deficiency (1967) New Engl J Med, 276, pp. 1163-116
Wilcox, W. R., Oliveira, J. P., Hopkin, R. J., Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry (2008) Mol Genet Metab, 93, pp. 112-128
Hopkin, R. J., Bissler, J., Banikazemi, M., Characterization of Fabry disease in 352 paediatric patients in the Fabry Registry (2008) Pediatr Res, 64, pp. 550-555
Eng, C. M., Guffon, N., Wilcox, W. R., Germain, D. P., Lee, P., Waldek, S., Caplan, L., Desnick, R. J., Safety and efficacy of recombinant human alpha-galactosidase a replacement therapy in Fabry's disease (2001) New England Journal of Medicine, 345 (1), pp. 9-16. , DOI 10. 1056/NEJM200107053450102
Wilcox, W. R., Banikazemi, M., Guffon, N., Long term safety and efficacy of enzyme replacement therapy for Fabry disease (2004) Am J Hum Genet, 75, pp. 65-74
Koskenvuo, J. W., Hartiala, J. J., Nuutila, P., Twenty-four-month -galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters (2008) J Inherit Metab Dis, 31, pp. 432-441
Desnick, R. J., Brady, R. O., Fabry disease in childhood (2004) Journal of Pediatrics, 144 (5 SUPPL.), pp. S20-S26. , DOI 10. 1016/j. jpeds. 2004. 01. 051
Moller, A. T., Jensen, T. S., Neurological manifestations in Fabry's disease (2007) Nature Clinical Practice Neurology, 3 (2), pp. 95-106. , DOI 10. 1038/ncpneuro0407, PII NCPNEURO0407
Higgins, C. B., Herfkens, R., Lipton, M. J., Nuclear magnetic resonance imaging of acute myocardial infarction in dogs: Alterations in magnetic relaxation times (1983) Am J Cardiol, 52, pp. 184-188
Germain, D. P., Waldek, S., Banikazemi, M., Bushinsky, D. A., Charrow, J., Desnick, R. J., Lee, P., Guffon, N., Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with fabry disease (2007) Journal of the American Society of Nephrology, 18 (5), pp. 1547-1557. , DOI 10. 1681/ASN. 2006080816
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
Ioannou, Y. A., Zeidner, K. M., Gordon, R. E., Desnick, R. J., Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase a replacement in enzyme-deficient mice (2001) American Journal of Human Genetics, 68 (1), pp. 14-25. , DOI 10. 1086/316953
Zarate, Y. A., Hopkin, R. J., Fabry's disease (2008) Lancet, 372, pp. 1427-1435
Maguire Jr., G.Q., Noz, M.E., Ponzo, F., Prone breast dual-time-point PET/CT compared with MRI for determining breast cancer (2009) AJR, 193, pp. W77. , (letter) [web
Moy, L., Noz, M.E., Maguire Jr., G.Q., Prone mammoPET acquisition improves the ability to fuse MRI and PET breast scans (2007) Clin Nucl Med, 32, pp. 194-198
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.
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
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Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study
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Petretta M, Cuocolo A
Letter by Petretta and Cuocolo regarding article, "Four-variable risk model in men and women with heart failure" (299 views)
Circ-Heart Fail (ISSN: 1941-3289), 2014 Mar 1; 7(2): 380-380.
Impact Factor: 5.867
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Andreotti G, Citro V, Correra A, Cubellis MV
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Paolillo S, Rengo G, Pagano G, Pellegrino T, Savarese G, Femminella GD, Tuccillo M, Boemio A, Attena E, Formisano R, Petraglia L, Scopacasa F, Galasso G, Leosco D, Trimarco B, Cuocolo A, Perrone-Filardi P
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Berisio R
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De Simone G
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Messalli G, Imbriaco M, Avitabile G, Russo R, Iodice D, Spinelli L, Dellegrottaglie S, Cademartiri F, Salvatore M, Pisani A
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Perrone-Filardi P, Paolillo S, Dellegrottaglie S, Gargiulo P, Savarese G, Marciano C, Casaretti L, Cecere M, Musella F, Pirozzi E, Parente A, Cuocolo A
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Manganelli F, Spadafora M, Varrella P, Peluso G, Sauro R, Di Lorenzo E, Rosato G, Daniele S, Cuocolo A
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Imbriaco M, Messalli G, Avitabile G, Cuocolo A, Maurea S, Soscia F, Pisani A
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Br J Radiol (ISSN: 0007-1285, 1748-880xelectronic, 0007-1285linking), 2010 Dec; 83(996): e249-e249.
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Pedone C, Di Pasquale G, Greco C, Gonzini L, Maggioni AP, Pavesi PC, Sabini A, Boccanelli A; In-Acs Outcome Investigators
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Storto G, Pellegrino T, Sorrentino AR, Luongo L, Petretta M, Cuocolo A
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J Nucl Cardiol (ISSN: 1532-6551, 1071-3581, 1532-6551electronic), 2007 Apr; 14(2): 194-199.
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Maestri R, Pinna G, Accardo A, Allegrini P, Balocchi R, D'ADDIO G, Ferrario M, Menicucci D, Porta A, Sassi R
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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
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Pauciullo P, Giannino A, De Michele M, Gentile M, Liguori R, Argiriou A, Carlotto A, Faccenda F, Mancini M, Bond MG, De Simone V, Rubba P
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Metab Clin Exp (ISSN: 0026-0495, 1532-8600), 2003; 52(11): 1433-1438.
Impact Factor: 2.013
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Morisco C, Argenziano L, Sarno D, Cuocolo A, Romano M, Trimarco B
The practical aspects of treatment in heart failure (343 views)
Ann Ital Med Int (ISSN: 0393-9340), 1993 Oct; 8Suppl: 14S-19S.
Impact Factor: 0
View Export to BibTeX Export to EndNote
Letter by Petretta and Cuocolo regarding article, "Four-variable risk model in men and women with heart failure" (299 views)
Circ-Heart Fail (ISSN: 1941-3289), 2014 Mar 1; 7(2): 380-380.
Impact Factor: 5.867
View Export to BibTeX Export to EndNote
Andreotti G, Citro V, Correra A, Cubellis MV
* A thermodynamic assay to test pharmacological chaperones for Fabry disease (394 views)
Bba-Gen Subjects (ISSN: 0925-4439, 0006-3002, 1570-9639), 2014 Mar; 1840(3): 1214-1224.
Impact Factor: 4.882
View Export to BibTeX Export to EndNote
Paolillo S, Rengo G, Pagano G, Pellegrino T, Savarese G, Femminella GD, Tuccillo M, Boemio A, Attena E, Formisano R, Petraglia L, Scopacasa F, Galasso G, Leosco D, Trimarco B, Cuocolo A, Perrone-Filardi P
* Impact of diabetes on cardiac sympathetic innervation in patients with heart failure: a 123I meta-iodobenzylguanidine (123I MIBG) scintigraphic study (313 views)
Diabetes Care (ISSN: 1935-5548, 0149-5992), 2013 Sep; 36(8): 2395-2401.
Impact Factor: 8.57
View Export to BibTeX Export to EndNote
Berisio R
* Editorial: cell processes in bacterial diseases (409 views)
Protein Pept Lett (ISSN: 0929-8665, 1875-5305), 2012 Oct; 19(10): 1011-1012.
Impact Factor: 1.994
View Export to BibTeX Export to EndNote
De Simone G
* Editorial: Zinc Metallo-Enzymes As Target For Drug Design (423 views)
Curr Med Chem (ISSN: 0929-8673, 1875-533x, 1875-533xelectronic), 2012 Feb; 19(6): 820-1012.
Impact Factor: 4.07
View Export to BibTeX Export to EndNote
Messalli G, Imbriaco M, Avitabile G, Russo R, Iodice D, Spinelli L, Dellegrottaglie S, Cademartiri F, Salvatore M, Pisani A
* Role of cardiac MRI in evaluating patients with Anderson-Fabry disease: Assessing cardiac effects of long-term enzyme replacement therapy (333 views)
Radiol Med (ISSN: 0033-8362, 1826-6983, 1826-6983electronic), 2012 Feb; 117(1): 19-28.
Impact Factor: 1.461
View Export to BibTeX Export to EndNote
Perrone-Filardi P, Paolillo S, Dellegrottaglie S, Gargiulo P, Savarese G, Marciano C, Casaretti L, Cecere M, Musella F, Pirozzi E, Parente A, Cuocolo A
* Assessment of cardiac sympathetic activity by MIBG imaging in patients with heart failure: a clinical appraisal (319 views)
Heart (ISSN: 1468-201x, 1355-6037), 2011 Nov; 97(22): 1828-1833.
Impact Factor: 4.223
View Export to BibTeX Export to EndNote
Manganelli F, Spadafora M, Varrella P, Peluso G, Sauro R, Di Lorenzo E, Rosato G, Daniele S, Cuocolo A
* Addition of atropine to submaximal exercise stress testing in patients evaluated for suspected ischaemia with SPECT imaging: a randomized, placebo-controlled trial (357 views)
Eur J Nucl Med (ISSN: 1619-7089, 1619-7070, 0340-6997), 2011 Feb; 38(2): 245-251.
Impact Factor: 4.991
View Export to BibTeX Export to EndNote
Imbriaco M, Messalli G, Avitabile G, Cuocolo A, Maurea S, Soscia F, Pisani A
* Cardiac magnetic resonance imaging illustrating Anderson-Fabry disease progression (283 views)
Br J Radiol (ISSN: 0007-1285, 1748-880xelectronic, 0007-1285linking), 2010 Dec; 83(996): e249-e249.
Impact Factor: 2.062
View Export to BibTeX Export to EndNote
Pedone C, Di Pasquale G, Greco C, Gonzini L, Maggioni AP, Pavesi PC, Sabini A, Boccanelli A; In-Acs Outcome Investigators
* Prescription at discharge of recommended treatments for secondary prevention in patients with ST-segment elevation myocardial infarction according to reperfusion strategies. Results from the IN-ACS outcome study (449 views)
Acute Cardiac Care (ISSN: 1748-295x, 1748-2941), 2009; 11(4): 222-228.
Impact Factor: 0
View Export to BibTeX Export to EndNote
Storto G, Pellegrino T, Sorrentino AR, Luongo L, Petretta M, Cuocolo A
* Estimation of coronary flow reserve by sestamibi imaging in type 2 diabetic patients with normal coronary arteries (322 views)
J Nucl Cardiol (ISSN: 1532-6551, 1071-3581, 1532-6551electronic), 2007 Apr; 14(2): 194-199.
Impact Factor: 2.359
View Export to BibTeX Export to EndNote
Maestri R, Pinna G, Accardo A, Allegrini P, Balocchi R, D'ADDIO G, Ferrario M, Menicucci D, Porta A, Sassi R
* Nonlinear indices of heart rate variability in chronic heart failure patients: Redundancy and comparative clinical value (373 views)
J Cardiovasc Electrophysiol (ISSN: 1045-3873), 2007; 18(4): 425-433.
Impact Factor: 3.475
View Export to BibTeX Export to EndNote
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
View Export to BibTeX Export to EndNote
Pauciullo P, Giannino A, De Michele M, Gentile M, Liguori R, Argiriou A, Carlotto A, Faccenda F, Mancini M, Bond MG, De Simone V, Rubba P
* Increased carotid artery intima-media thickness is associated with a novel mutation of low-density lipoprotein receptor independently of major cardiovascular risk factors (566 views)
Metab Clin Exp (ISSN: 0026-0495, 1532-8600), 2003; 52(11): 1433-1438.
Impact Factor: 2.013
View Export to BibTeX Export to EndNote
Morisco C, Argenziano L, Sarno D, Cuocolo A, Romano M, Trimarco B
The practical aspects of treatment in heart failure (343 views)
Ann Ital Med Int (ISSN: 0393-9340), 1993 Oct; 8Suppl: 14S-19S.
Impact Factor: 0
View Export to BibTeX Export to EndNote
15 Records (14 excluding Abstracts).
Total impact factor: 45.99 (43.928 excluding Abstracts).
Total 5 year impact factor: 50.819 (48.464 excluding Abstracts).
Total impact factor: 45.99 (43.928 excluding Abstracts).
Total 5 year impact factor: 50.819 (48.464 excluding Abstracts).
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