Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α

Genetics in Medicine

Volumn 16, Issue 10, 2014, Pages 766-772

  Tsuboi, Kazuya ^a   Yamamoto, Hiroshi ^a  

^a NAGOYA CENTRAL HOSPITAL   (Japan)

Author keywords

agalsidase ; agalsidase ; enzyme replacement therapy; Fabry disease

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE; CREATININE; ISOENZYME;

ADULT; AGED; DRUG EFFECTS; ENZYME REPLACEMENT; FABRY DISEASE; FEMALE; FOLLOW UP; GLOMERULUS FILTRATION RATE; HUMAN; MALE; MIDDLE AGED; PROCEDURES; PROTEINURIA; TIME; TREATMENT OUTCOME; URINE;

ADULT; AGED; ALPHA-GALACTOSIDASE; CREATININE; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FEMALE; FOLLOW-UP STUDIES; GLOMERULAR FILTRATION RATE; HUMANS; ISOENZYMES; MALE; MIDDLE AGED; PROTEINURIA; TIME FACTORS; TREATMENT OUTCOME;

EID: 84920512558     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2014.28     Document Type: Article

References (29)

1. Germain, D.P., Fabry disease. 10.1186/1750-1172-5-30 Orphanet J Rare Dis, 5, 2010, 30.
2. Mehta, A., Beck, M., Eyskens, F., Fabry disease: a review of current management strategies. 1:STN:280:DC%2BC3cjlslOmuw%3D%3D 10.1093/qjmed/hcq117 QJM 103 (2010), 641–659.
3. Ramaswami, U., Update on role of agalsidase alfa in management of Fabry disease. 1:CAS:528:DC%2BC3MXktlCgur0%3D 10.2147/DDDT.S11985 Drug Des Devel Ther 5 (2011), 155–173.
4. Mehta, A., West, M.L., Pintos-Morell, G., Therapeutic goals in the treatment of Fabry disease. 1:CAS:528:DC%2BC3cXhsVWhur3F 10.1097/GIM.0b013e3181f6e676 Genet Med 12 (2010), 713–720.
5. Lidove, O., West, M.L., Pintos-Morell, G., Effects of enzyme replacement therapy in Fabry disease–a comprehensive review of the medical literature. 1:CAS:528:DC%2BC3cXhsVWhur3L 10.1097/GIM.0b013e3181f13b75 Genet Med 12 (2010), 668–679.
6. Keating, G.M., Agalsidase alfa: a review of its use in the management of Fabry disease. 1:CAS:528:DC%2BC38Xhs1Kmt7rL 10.1007/BF03261891 BioDrugs 26 (2012), 335–354.
7. West, M.L., LeMoine, K.M., Bichet, D.G., A randomized controlled trial of enzyme replacement therapy in Fabry nephropathy: the Canadian Fabry Disease Initiative at year two [Abstract SA-PO2780]. 10.1681/ASN.2008080870 J Am Soc Neph, 20, 2009, 747A.
8. Lee, K., Jin, X., Zhang, K., A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease. 10.1093/glycob/cwg034 Glycobiology 13 (2003), 305–313.
9. Sakuraba, H., Murata-Ohsawa, M., Kawashima, I., Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice. 1:CAS:528:DC%2BD28Xis1Oktr8%3D 10.1007/s10038-005-0342-9 J Hum Genet 51 (2006), 180–188.
10. Huby, R.D., Dearman, R.J., Kimber, I., Why are some proteins allergens?. 1:CAS:528:DC%2BD3cXjsl2qs78%3D 10.1093/toxsci/55.2.235 Toxicol Sci 55 (2000), 235–246.
11. Bekri, S., Mehta, A., Beck, M., Sunder-Plassmann, G., Importance of glycosylation in enzyme replacement therapy. Fabry Disease: Perspectives from 5 Years of FOS., 2006, Oxford PharmaGenesis.
12. European Medicines Agency. Assessment report on the shortage of Fabrazyme. Overview of shortage period: spontaneous reports from June 2009 through 15 September 2010 and registry data from June 2009 through 05 August 2010. EMEA/H/C/000370. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/11/WC500099241.pdf Accessed December 2012.
13. European Medicines Agency. European Medicines Agency reviews treatment recommendations for Fabrazyme. http://www.ema.europa.eu/docs/en_GB/document_library/Press_release/2010/10/WC500098370.pdf Accessed December 2012.
14. European Medicines Agency, Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT). 1:CAS:528:DC%2BC3MXjvFyntA%3D%3D 10.1016/j.ymgme.2010.11.155 Mol Genet Metab 102 (2011), 99–102.
15. Tsuboi, K., Yamamoto, H., Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagal). 1:CAS:528:DC%2BC38XhtlWqtLnO 10.1038/gim.2012.39 Genet Med 14 (2012), 779–786.
16. CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration), A new equation to estimate glomerular filtration rate. 10.7326/0003-4819-150-9-200905050-00006 Ann Intern Med 150 (2009), 604–612.
17. Devereux, R.B., Alonso, D.R., Lutas, E.M., Echocardiographic assessment of left ventricular hypertrophy: comparison to necropsy findings. 1:STN:280:DyaL287islSkug%3D%3D 10.1016/0002-9149(86)90771-X Am J Cardiol 57 (1986), 450–458.
18. Chamber Quantification Writing Group; American Society of Echocardiography's Guidelines and Standards Committee; European Association of Echocardiography, Recommendations for chamber quantification: a report from the American Society of Echocardiography's Guidelines and Standards Committee and the Chamber Quantification Writing Group, developed in conjunction with the European Association of Echocardiography, a branch of the European Society of Cardiology. 10.1016/j.echo.2005.10.005 J Am Soc Echocardiogr 18 (2005), 1440–1463.
19. Tan, G., Jensen, M.P., Thornby, J.I., Shanti, B.F., Validation of the Brief Pain Inventory for chronic nonmalignant pain. 10.1016/j.jpain.2003.12.005 J Pain 5 (2004), 133–137.
20. The EuroQoL Group., EuroQoL-a new facility for the measurement of health-related quality of life. 10.1016/0168-8510(90)90421-9 Health Policy 16 (1990), 199–208.
21. Whybra, C., Kampmann, C., Krummenauer, F., The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy. 1:STN:280:DC%2BD2c7jtlGqtQ%3D%3D 10.1111/j.1399-0004.2004.00219.x Clin Genet 65 (2004), 299–307.
22. Linthorst, G.E., Burlina, A.P., Cecchi, F., Recommendations on reintroduction of agalsidase Beta for patients with Fabry disease in Europe, following a period of shortage. 10.1007/8904_2012_160 JIMD Rep 8 (2013), 51–56.
23. Smid, B.E., Rombach, S.M., Aerts, J.M., Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients. 10.1186/1750-1172-6-69 Orphanet J Rare Dis, 6, 2011, 69.
24. Pisani, A., Spinelli, L., Visciano, B., Effects of switching from agalsidase Beta to agalsidase alfa in 10 patients with Anderson-Fabry disease. 1:STN:280:DC%2BC3svgtlyqsw%3D%3D 10.1007/8904_2012_177 JIMD Rep 9 (2013), 41–48.
25. van Breemen, M.J., Rombach, S.M., Dekker, N., Reduction of elevated plasma globotriaosylsphingosine in patients with classic Fabry disease following enzyme replacement therapy. 1:CAS:528:DC%2BC3cXhsVGnsbzP 10.1016/j.bbadis.2010.09.007 Biochim Biophys Acta 1812 (2011), 70–76.
26. Maisel, A., B-type natriuretic peptide levels: diagnostic and prognostic in congestive heart failure: what's next?. 10.1161/01.CIR.0000019121.91548.C2 Circulation 105 (2002), 2328–2331.
27. Yasuda, K., Kimura, T., Sasaki, K., Plasma B-type natriuretic peptide level predicts kidney prognosis in patients with predialysis chronic kidney disease. 1:CAS:528:DC%2BC38Xhs1CqsLjE 10.1093/ndt/gfs365 Nephrol Dial Transplant 27 (2012), 3885–3891.
28. Linthorst, G.E., Hollak, C.E., Donker-Koopman, W.E., Strijland, A., Aerts, J.M., Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta. 1:CAS:528:DC%2BD2cXpsVSgs7w%3D 10.1111/j.1523-1755.2004.00924.x Kidney Int 66 (2004), 1589–1595.
29. Tanaka, A., Takeda, T., Hoshina, T., Enzyme replacement therapy in a patient with Fabry disease and the development of IgE antibodies against agalsidase beta but not agalsidase alpha. J Inherit Metab Dis 33:suppl 3 (2010), S245–S252.